JP2020164529A - 遺伝子編集用のcas多様体 - Google Patents
遺伝子編集用のcas多様体 Download PDFInfo
- Publication number
- JP2020164529A JP2020164529A JP2020082201A JP2020082201A JP2020164529A JP 2020164529 A JP2020164529 A JP 2020164529A JP 2020082201 A JP2020082201 A JP 2020082201A JP 2020082201 A JP2020082201 A JP 2020082201A JP 2020164529 A JP2020164529 A JP 2020164529A
- Authority
- JP
- Japan
- Prior art keywords
- cas9
- deaminase
- nucleic acid
- sequence
- domain
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 238000010362 genome editing Methods 0.000 title description 26
- 108091033409 CRISPR Proteins 0.000 claims abstract description 243
- 108020001507 fusion proteins Proteins 0.000 claims abstract description 162
- 102000037865 fusion proteins Human genes 0.000 claims abstract description 158
- 150000007523 nucleic acids Chemical class 0.000 claims abstract description 153
- 102000039446 nucleic acids Human genes 0.000 claims abstract description 145
- 108020004707 nucleic acids Proteins 0.000 claims abstract description 145
- 108090000623 proteins and genes Proteins 0.000 claims abstract description 139
- 102000004169 proteins and genes Human genes 0.000 claims abstract description 102
- 238000000034 method Methods 0.000 claims abstract description 87
- 102000004190 Enzymes Human genes 0.000 claims abstract description 75
- 108090000790 Enzymes Proteins 0.000 claims abstract description 75
- 239000003153 chemical reaction reagent Substances 0.000 claims abstract description 7
- 230000035772 mutation Effects 0.000 claims description 186
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 97
- 201000010099 disease Diseases 0.000 claims description 64
- 239000002773 nucleotide Substances 0.000 claims description 60
- 108020004414 DNA Proteins 0.000 claims description 59
- 125000003729 nucleotide group Chemical group 0.000 claims description 57
- 108020004705 Codon Proteins 0.000 claims description 38
- 238000006481 deamination reaction Methods 0.000 claims description 34
- 208000035475 disorder Diseases 0.000 claims description 33
- 230000000694 effects Effects 0.000 claims description 32
- 230000009615 deamination Effects 0.000 claims description 31
- 101710143275 Single-stranded DNA cytosine deaminase Proteins 0.000 claims description 28
- 102100022433 Single-stranded DNA cytosine deaminase Human genes 0.000 claims description 28
- 230000014509 gene expression Effects 0.000 claims description 25
- 102000053602 DNA Human genes 0.000 claims description 22
- 238000010442 DNA editing Methods 0.000 claims description 21
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 20
- 238000006467 substitution reaction Methods 0.000 claims description 19
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 18
- 108700008625 Reporter Genes Proteins 0.000 claims description 17
- 102200085789 rs121913279 Human genes 0.000 claims description 17
- 108090000566 Caspase-9 Proteins 0.000 claims description 16
- 239000002753 trypsin inhibitor Substances 0.000 claims description 14
- 108010047303 von Willebrand Factor Proteins 0.000 claims description 14
- 108010079649 APOBEC-1 Deaminase Proteins 0.000 claims description 13
- 238000010367 cloning Methods 0.000 claims description 13
- 108060001084 Luciferase Proteins 0.000 claims description 12
- 108091027544 Subgenomic mRNA Proteins 0.000 claims description 12
- 102100036537 von Willebrand factor Human genes 0.000 claims description 12
- 102000004039 Caspase-9 Human genes 0.000 claims description 10
- 206010029260 Neuroblastoma Diseases 0.000 claims description 10
- UHDGCWIWMRVCDJ-ZAKLUEHWSA-N cytidine Chemical group O=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-ZAKLUEHWSA-N 0.000 claims description 9
- 230000008685 targeting Effects 0.000 claims description 9
- 102000055025 Adenosine deaminases Human genes 0.000 claims description 8
- 239000005089 Luciferase Substances 0.000 claims description 8
- 102100022033 Presenilin-1 Human genes 0.000 claims description 8
- 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 claims description 7
- 208000027276 Von Willebrand disease Diseases 0.000 claims description 7
- 208000012137 von Willebrand disease (hereditary or acquired) Diseases 0.000 claims description 7
- 101710095342 Apolipoprotein B Proteins 0.000 claims description 6
- 102100040202 Apolipoprotein B-100 Human genes 0.000 claims description 6
- 206010056370 Congestive cardiomyopathy Diseases 0.000 claims description 6
- 108010031325 Cytidine deaminase Proteins 0.000 claims description 6
- 201000010046 Dilated cardiomyopathy Diseases 0.000 claims description 6
- 101000617536 Homo sapiens Presenilin-1 Proteins 0.000 claims description 6
- 238000001727 in vivo Methods 0.000 claims description 6
- 102100027310 Bromodomain adjacent to zinc finger domain protein 1A Human genes 0.000 claims description 5
- 208000006545 Chronic Obstructive Pulmonary Disease Diseases 0.000 claims description 5
- 101000937778 Homo sapiens Bromodomain adjacent to zinc finger domain protein 1A Proteins 0.000 claims description 5
- 108091007960 PI3Ks Proteins 0.000 claims description 5
- 101150035190 PSEN1 gene Proteins 0.000 claims description 5
- 230000017156 mRNA modification Effects 0.000 claims description 5
- 230000001613 neoplastic effect Effects 0.000 claims description 5
- 239000000872 buffer Substances 0.000 claims description 4
- 208000025688 early-onset autosomal dominant Alzheimer disease Diseases 0.000 claims description 4
- 208000015756 familial Alzheimer disease Diseases 0.000 claims description 4
- 208000024827 Alzheimer disease Diseases 0.000 claims description 3
- 208000031229 Cardiomyopathies Diseases 0.000 claims description 3
- 201000003883 Cystic fibrosis Diseases 0.000 claims description 3
- 102100036912 Desmin Human genes 0.000 claims description 3
- 108010044052 Desmin Proteins 0.000 claims description 3
- 208000032838 Hereditary amyloidosis with primary renal involvement Diseases 0.000 claims description 3
- 208000001126 Keratosis Diseases 0.000 claims description 3
- 201000011252 Phenylketonuria Diseases 0.000 claims description 3
- 208000024777 Prion disease Diseases 0.000 claims description 3
- 125000000539 amino acid group Chemical group 0.000 claims description 3
- 230000001684 chronic effect Effects 0.000 claims description 3
- 210000005045 desmin Anatomy 0.000 claims description 3
- 238000001514 detection method Methods 0.000 claims description 3
- 201000007891 familial visceral amyloidosis Diseases 0.000 claims description 3
- 201000006716 hereditary lymphedema Diseases 0.000 claims description 3
- 208000032300 lymphatic malformation Diseases 0.000 claims description 3
- 208000002502 lymphedema Diseases 0.000 claims description 3
- 210000003205 muscle Anatomy 0.000 claims description 3
- 208000011580 syndromic disease Diseases 0.000 claims description 3
- 230000003115 biocidal effect Effects 0.000 claims description 2
- 108091006047 fluorescent proteins Proteins 0.000 claims description 2
- 102000034287 fluorescent proteins Human genes 0.000 claims description 2
- 239000003550 marker Substances 0.000 claims description 2
- 102200049725 rs182983506 Human genes 0.000 claims description 2
- 238000004904 shortening Methods 0.000 claims description 2
- 102100040397 C->U-editing enzyme APOBEC-1 Human genes 0.000 claims 2
- 102100026846 Cytidine deaminase Human genes 0.000 claims 2
- 101150045640 VWF gene Proteins 0.000 claims 2
- 102000010400 1-phosphatidylinositol-3-kinase activity proteins Human genes 0.000 claims 1
- 206010010356 Congenital anomaly Diseases 0.000 claims 1
- 241001323319 Psen Species 0.000 claims 1
- 208000010641 Tooth disease Diseases 0.000 claims 1
- 238000005576 amination reaction Methods 0.000 claims 1
- -1 e.g. Proteins 0.000 abstract description 9
- 235000018102 proteins Nutrition 0.000 description 87
- 101710163270 Nuclease Proteins 0.000 description 54
- 210000004027 cell Anatomy 0.000 description 44
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 33
- 108020005004 Guide RNA Proteins 0.000 description 31
- 230000004927 fusion Effects 0.000 description 31
- 235000001014 amino acid Nutrition 0.000 description 29
- 229940024606 amino acid Drugs 0.000 description 22
- 150000001413 amino acids Chemical class 0.000 description 22
- 108090000765 processed proteins & peptides Proteins 0.000 description 21
- 239000012634 fragment Substances 0.000 description 19
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 description 18
- 230000004048 modification Effects 0.000 description 16
- 238000012986 modification Methods 0.000 description 16
- 238000012408 PCR amplification Methods 0.000 description 15
- 239000004475 Arginine Substances 0.000 description 14
- 108010077850 Nuclear Localization Signals Proteins 0.000 description 14
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 description 13
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 description 12
- 102000012758 APOBEC-1 Deaminase Human genes 0.000 description 11
- 102000000311 Cytosine Deaminase Human genes 0.000 description 11
- 108010080611 Cytosine Deaminase Proteins 0.000 description 11
- 102220605874 Cytosolic arginine sensor for mTORC1 subunit 2_D10A_mutation Human genes 0.000 description 11
- 230000027455 binding Effects 0.000 description 11
- 238000012937 correction Methods 0.000 description 10
- 102000004196 processed proteins & peptides Human genes 0.000 description 10
- 238000012163 sequencing technique Methods 0.000 description 10
- 230000007018 DNA scission Effects 0.000 description 9
- 108091028113 Trans-activating crRNA Proteins 0.000 description 9
- 238000003776 cleavage reaction Methods 0.000 description 9
- 229940104302 cytosine Drugs 0.000 description 9
- 229920001184 polypeptide Polymers 0.000 description 9
- 230000007017 scission Effects 0.000 description 9
- 208000024891 symptom Diseases 0.000 description 9
- 108091079001 CRISPR RNA Proteins 0.000 description 8
- 101000799160 Homo sapiens tRNA-specific adenosine deaminase 1 Proteins 0.000 description 8
- 206010028980 Neoplasm Diseases 0.000 description 8
- 241000193996 Streptococcus pyogenes Species 0.000 description 8
- 210000004899 c-terminal region Anatomy 0.000 description 8
- 201000011510 cancer Diseases 0.000 description 8
- 230000000295 complement effect Effects 0.000 description 8
- 230000006870 function Effects 0.000 description 8
- 125000000896 monocarboxylic acid group Chemical group 0.000 description 8
- 102100034113 tRNA-specific adenosine deaminase 1 Human genes 0.000 description 8
- 108091081024 Start codon Proteins 0.000 description 7
- 230000003197 catalytic effect Effects 0.000 description 7
- 229940045145 uridine Drugs 0.000 description 7
- ONIBWKKTOPOVIA-BYPYZUCNSA-N L-Proline Chemical compound OC(=O)[C@@H]1CCCN1 ONIBWKKTOPOVIA-BYPYZUCNSA-N 0.000 description 6
- ONIBWKKTOPOVIA-UHFFFAOYSA-N Proline Natural products OC(=O)C1CCCN1 ONIBWKKTOPOVIA-UHFFFAOYSA-N 0.000 description 6
- DRTQHJPVMGBUCF-PSQAKQOGSA-N beta-L-uridine Natural products O[C@H]1[C@@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-PSQAKQOGSA-N 0.000 description 6
- 230000009395 genetic defect Effects 0.000 description 6
- 239000000203 mixture Substances 0.000 description 6
- 238000011160 research Methods 0.000 description 6
- 241000894007 species Species 0.000 description 6
- 108091006106 transcriptional activators Proteins 0.000 description 6
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 description 6
- UHDGCWIWMRVCDJ-UHFFFAOYSA-N 1-beta-D-Xylofuranosyl-NH-Cytosine Natural products O=C1N=C(N)C=CN1C1C(O)C(O)C(CO)O1 UHDGCWIWMRVCDJ-UHFFFAOYSA-N 0.000 description 5
- 108010004483 APOBEC-3G Deaminase Proteins 0.000 description 5
- UHDGCWIWMRVCDJ-PSQAKQOGSA-N Cytidine Natural products O=C1N=C(N)C=CN1[C@@H]1[C@@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-PSQAKQOGSA-N 0.000 description 5
- 101100490452 Drosophila melanogaster Adat1 gene Proteins 0.000 description 5
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 description 5
- 101000755690 Homo sapiens Single-stranded DNA cytosine deaminase Proteins 0.000 description 5
- 208000026350 Inborn Genetic disease Diseases 0.000 description 5
- ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical compound CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 description 5
- 108091034117 Oligonucleotide Proteins 0.000 description 5
- 230000003321 amplification Effects 0.000 description 5
- 238000003556 assay Methods 0.000 description 5
- 235000018417 cysteine Nutrition 0.000 description 5
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 description 5
- 230000007711 cytoplasmic localization Effects 0.000 description 5
- 238000013461 design Methods 0.000 description 5
- 229940079593 drug Drugs 0.000 description 5
- 239000003814 drug Substances 0.000 description 5
- 238000000338 in vitro Methods 0.000 description 5
- 238000003199 nucleic acid amplification method Methods 0.000 description 5
- 239000002777 nucleoside Substances 0.000 description 5
- 230000002062 proliferating effect Effects 0.000 description 5
- 229960001134 von willebrand factor Drugs 0.000 description 5
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 4
- 208000001593 Bernard-Soulier syndrome Diseases 0.000 description 4
- 208000028702 Congenital thrombocyte disease Diseases 0.000 description 4
- 102000005381 Cytidine Deaminase Human genes 0.000 description 4
- AGPKZVBTJJNPAG-WHFBIAKZSA-N L-isoleucine Chemical compound CC[C@H](C)[C@H](N)C(O)=O AGPKZVBTJJNPAG-WHFBIAKZSA-N 0.000 description 4
- KZSNJWFQEVHDMF-BYPYZUCNSA-N L-valine Chemical compound CC(C)[C@H](N)C(O)=O KZSNJWFQEVHDMF-BYPYZUCNSA-N 0.000 description 4
- ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Natural products CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 description 4
- 101000755751 Mus musculus Single-stranded DNA cytosine deaminase Proteins 0.000 description 4
- 102000038030 PI3Ks Human genes 0.000 description 4
- IQFYYKKMVGJFEH-XLPZGREQSA-N Thymidine Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 description 4
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 description 4
- KZSNJWFQEVHDMF-UHFFFAOYSA-N Valine Natural products CC(C)C(N)C(O)=O KZSNJWFQEVHDMF-UHFFFAOYSA-N 0.000 description 4
- 108020001778 catalytic domains Proteins 0.000 description 4
- 239000013078 crystal Substances 0.000 description 4
- 230000002068 genetic effect Effects 0.000 description 4
- 210000005260 human cell Anatomy 0.000 description 4
- 229960000310 isoleucine Drugs 0.000 description 4
- 230000001404 mediated effect Effects 0.000 description 4
- 230000006780 non-homologous end joining Effects 0.000 description 4
- 230000025308 nuclear transport Effects 0.000 description 4
- 150000003833 nucleoside derivatives Chemical class 0.000 description 4
- 239000013612 plasmid Substances 0.000 description 4
- 229920000642 polymer Polymers 0.000 description 4
- 238000000746 purification Methods 0.000 description 4
- 239000000758 substrate Substances 0.000 description 4
- 238000002560 therapeutic procedure Methods 0.000 description 4
- 238000013519 translation Methods 0.000 description 4
- 239000004474 valine Substances 0.000 description 4
- 108700028369 Alleles Proteins 0.000 description 3
- 101710081722 Antitrypsin Proteins 0.000 description 3
- 241000894006 Bacteria Species 0.000 description 3
- 108091032955 Bacterial small RNA Proteins 0.000 description 3
- 102100038076 DNA dC->dU-editing enzyme APOBEC-3G Human genes 0.000 description 3
- 241000724791 Filamentous phage Species 0.000 description 3
- 229940123611 Genome editing Drugs 0.000 description 3
- FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 description 3
- COLNVLDHVKWLRT-QMMMGPOBSA-N L-phenylalanine Chemical compound OC(=O)[C@@H](N)CC1=CC=CC=C1 COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 description 3
- 208000024556 Mendelian disease Diseases 0.000 description 3
- 108091081021 Sense strand Proteins 0.000 description 3
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 description 3
- HEMHJVSKTPXQMS-UHFFFAOYSA-M Sodium hydroxide Chemical compound [OH-].[Na+] HEMHJVSKTPXQMS-UHFFFAOYSA-M 0.000 description 3
- 239000002253 acid Substances 0.000 description 3
- 238000013459 approach Methods 0.000 description 3
- 230000008901 benefit Effects 0.000 description 3
- 239000003795 chemical substances by application Substances 0.000 description 3
- 238000012217 deletion Methods 0.000 description 3
- 230000037430 deletion Effects 0.000 description 3
- 230000005782 double-strand break Effects 0.000 description 3
- 238000002474 experimental method Methods 0.000 description 3
- 238000012239 gene modification Methods 0.000 description 3
- 238000001415 gene therapy Methods 0.000 description 3
- 230000000415 inactivating effect Effects 0.000 description 3
- AGPKZVBTJJNPAG-UHFFFAOYSA-N isoleucine Natural products CCC(C)C(N)C(O)=O AGPKZVBTJJNPAG-UHFFFAOYSA-N 0.000 description 3
- 230000004807 localization Effects 0.000 description 3
- 238000004519 manufacturing process Methods 0.000 description 3
- 208000030159 metabolic disease Diseases 0.000 description 3
- 229930182817 methionine Natural products 0.000 description 3
- 239000003471 mutagenic agent Substances 0.000 description 3
- 230000009437 off-target effect Effects 0.000 description 3
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Natural products OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 description 3
- 102000040430 polynucleotide Human genes 0.000 description 3
- 108091033319 polynucleotide Proteins 0.000 description 3
- 239000002157 polynucleotide Substances 0.000 description 3
- 230000008569 process Effects 0.000 description 3
- 230000010076 replication Effects 0.000 description 3
- 210000001519 tissue Anatomy 0.000 description 3
- 238000013518 transcription Methods 0.000 description 3
- 230000035897 transcription Effects 0.000 description 3
- 230000002103 transcriptional effect Effects 0.000 description 3
- 239000011701 zinc Substances 0.000 description 3
- ZDTFMPXQUSBYRL-FTWQFJAYSA-N (2r,3r,4r,5r)-2-(2,6-diaminopurin-9-yl)-5-(hydroxymethyl)oxolane-3,4-diol Chemical compound C12=NC(N)=NC(N)=C2N=CN1[C@@H]1O[C@H](CO)[C@H](O)[C@H]1O ZDTFMPXQUSBYRL-FTWQFJAYSA-N 0.000 description 2
- ZAYHVCMSTBRABG-JXOAFFINSA-N 5-methylcytidine Chemical compound O=C1N=C(N)C(C)=CN1[C@H]1[C@H](O)[C@H](O)[C@@H](CO)O1 ZAYHVCMSTBRABG-JXOAFFINSA-N 0.000 description 2
- 102000002797 APOBEC-3G Deaminase Human genes 0.000 description 2
- 102000009081 Apolipoprotein A-II Human genes 0.000 description 2
- 108010087614 Apolipoprotein A-II Proteins 0.000 description 2
- 241000203069 Archaea Species 0.000 description 2
- 238000010446 CRISPR interference Methods 0.000 description 2
- 238000010356 CRISPR-Cas9 genome editing Methods 0.000 description 2
- 108010062745 Chloride Channels Proteins 0.000 description 2
- 102100023457 Chloride channel protein 1 Human genes 0.000 description 2
- 208000029323 Congenital myotonia Diseases 0.000 description 2
- 230000004568 DNA-binding Effects 0.000 description 2
- 241000196324 Embryophyta Species 0.000 description 2
- 101710191360 Eosinophil cationic protein Proteins 0.000 description 2
- 241000206602 Eukaryota Species 0.000 description 2
- 108010070675 Glutathione transferase Proteins 0.000 description 2
- 239000004471 Glycine Substances 0.000 description 2
- 108010043121 Green Fluorescent Proteins Proteins 0.000 description 2
- 102000004144 Green Fluorescent Proteins Human genes 0.000 description 2
- NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 description 2
- 102000029812 HNH nuclease Human genes 0.000 description 2
- 108060003760 HNH nuclease Proteins 0.000 description 2
- 102100029100 Hematopoietic prostaglandin D synthase Human genes 0.000 description 2
- 208000028782 Hereditary disease Diseases 0.000 description 2
- 101100490453 Homo sapiens ADAT1 gene Proteins 0.000 description 2
- 101000964330 Homo sapiens C->U-editing enzyme APOBEC-1 Proteins 0.000 description 2
- 101000906651 Homo sapiens Chloride channel protein 1 Proteins 0.000 description 2
- 101000742736 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3G Proteins 0.000 description 2
- 101001029302 Homo sapiens Forkhead box protein D4 Proteins 0.000 description 2
- 101000827703 Homo sapiens Polyphosphoinositide phosphatase Proteins 0.000 description 2
- 101000851030 Homo sapiens Vascular endothelial growth factor receptor 3 Proteins 0.000 description 2
- 206010020649 Hyperkeratosis Diseases 0.000 description 2
- 108010015268 Integration Host Factors Proteins 0.000 description 2
- 102100022905 Keratin, type II cytoskeletal 1 Human genes 0.000 description 2
- 108010070514 Keratin-1 Proteins 0.000 description 2
- 241000186781 Listeria Species 0.000 description 2
- 208000015439 Lysosomal storage disease Diseases 0.000 description 2
- 101710175625 Maltose/maltodextrin-binding periplasmic protein Proteins 0.000 description 2
- 241000124008 Mammalia Species 0.000 description 2
- 241001465754 Metazoa Species 0.000 description 2
- 208000010316 Myotonia congenita Diseases 0.000 description 2
- 208000009869 Neu-Laxova syndrome Diseases 0.000 description 2
- 108010066154 Nuclear Export Signals Proteins 0.000 description 2
- 108091007494 Nucleic acid- binding domains Proteins 0.000 description 2
- 108010069013 Phenylalanine Hydroxylase Proteins 0.000 description 2
- 102000023159 Platelet Glycoprotein GPIb-IX Complex Human genes 0.000 description 2
- 108010045766 Platelet Glycoprotein GPIb-IX Complex Proteins 0.000 description 2
- 102100023591 Polyphosphoinositide phosphatase Human genes 0.000 description 2
- 108010036933 Presenilin-1 Proteins 0.000 description 2
- 241001135221 Prevotella intermedia Species 0.000 description 2
- 102000029797 Prion Human genes 0.000 description 2
- 108091000054 Prion Proteins 0.000 description 2
- 230000009948 RNA mutation Effects 0.000 description 2
- 108010008281 Recombinant Fusion Proteins Proteins 0.000 description 2
- 102000007056 Recombinant Fusion Proteins Human genes 0.000 description 2
- 102000018120 Recombinases Human genes 0.000 description 2
- 108010091086 Recombinases Proteins 0.000 description 2
- 102100036007 Ribonuclease 3 Human genes 0.000 description 2
- 101710192197 Ribonuclease 3 Proteins 0.000 description 2
- 102000003661 Ribonuclease III Human genes 0.000 description 2
- 108010057163 Ribonuclease III Proteins 0.000 description 2
- 108020004682 Single-Stranded DNA Proteins 0.000 description 2
- 241000194020 Streptococcus thermophilus Species 0.000 description 2
- AYFVYJQAPQTCCC-UHFFFAOYSA-N Threonine Natural products CC(O)C(N)C(O)=O AYFVYJQAPQTCCC-UHFFFAOYSA-N 0.000 description 2
- 239000004473 Threonine Substances 0.000 description 2
- 102000006943 Uracil-DNA Glycosidase Human genes 0.000 description 2
- 108010072685 Uracil-DNA Glycosidase Proteins 0.000 description 2
- 108010017070 Zinc Finger Nucleases Proteins 0.000 description 2
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 description 2
- 230000004913 activation Effects 0.000 description 2
- 230000003044 adaptive effect Effects 0.000 description 2
- 210000005006 adaptive immune system Anatomy 0.000 description 2
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 description 2
- 102000013640 alpha-Crystallin B Chain Human genes 0.000 description 2
- 108010051585 alpha-Crystallin B Chain Proteins 0.000 description 2
- 230000000692 anti-sense effect Effects 0.000 description 2
- 230000001580 bacterial effect Effects 0.000 description 2
- 230000008512 biological response Effects 0.000 description 2
- 108700023293 biotin carboxyl carrier Proteins 0.000 description 2
- 230000000711 cancerogenic effect Effects 0.000 description 2
- 238000004113 cell culture Methods 0.000 description 2
- 230000022131 cell cycle Effects 0.000 description 2
- 210000000349 chromosome Anatomy 0.000 description 2
- 230000004186 co-expression Effects 0.000 description 2
- 150000001875 compounds Chemical class 0.000 description 2
- 230000007123 defense Effects 0.000 description 2
- 238000011161 development Methods 0.000 description 2
- 208000016097 disease of metabolism Diseases 0.000 description 2
- 208000037765 diseases and disorders Diseases 0.000 description 2
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 description 2
- 239000012636 effector Substances 0.000 description 2
- 208000016361 genetic disease Diseases 0.000 description 2
- 230000005017 genetic modification Effects 0.000 description 2
- 235000013617 genetically modified food Nutrition 0.000 description 2
- 239000005090 green fluorescent protein Substances 0.000 description 2
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 description 2
- 238000002744 homologous recombination Methods 0.000 description 2
- 230000006801 homologous recombination Effects 0.000 description 2
- 102000046390 human APOBEC1 Human genes 0.000 description 2
- 102000054962 human APOBEC3G Human genes 0.000 description 2
- 238000009396 hybridization Methods 0.000 description 2
- 230000036039 immunity Effects 0.000 description 2
- 230000002401 inhibitory effect Effects 0.000 description 2
- 238000003780 insertion Methods 0.000 description 2
- 230000037431 insertion Effects 0.000 description 2
- 230000000670 limiting effect Effects 0.000 description 2
- 238000003670 luciferase enzyme activity assay Methods 0.000 description 2
- 108020004999 messenger RNA Proteins 0.000 description 2
- 238000010369 molecular cloning Methods 0.000 description 2
- 230000003505 mutagenic effect Effects 0.000 description 2
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 description 2
- 230000029279 positive regulation of transcription, DNA-dependent Effects 0.000 description 2
- 238000012545 processing Methods 0.000 description 2
- 239000013636 protein dimer Substances 0.000 description 2
- 230000037425 regulation of transcription Effects 0.000 description 2
- 230000008439 repair process Effects 0.000 description 2
- 238000012216 screening Methods 0.000 description 2
- 238000001228 spectrum Methods 0.000 description 2
- 210000000130 stem cell Anatomy 0.000 description 2
- 239000000126 substance Substances 0.000 description 2
- 235000000346 sugar Nutrition 0.000 description 2
- 150000008163 sugars Chemical class 0.000 description 2
- 108091006107 transcriptional repressors Proteins 0.000 description 2
- 229940035893 uracil Drugs 0.000 description 2
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 2
- RIFDKYBNWNPCQK-IOSLPCCCSA-N (2r,3s,4r,5r)-2-(hydroxymethyl)-5-(6-imino-3-methylpurin-9-yl)oxolane-3,4-diol Chemical compound C1=2N(C)C=NC(=N)C=2N=CN1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O RIFDKYBNWNPCQK-IOSLPCCCSA-N 0.000 description 1
- RKSLVDIXBGWPIS-UAKXSSHOSA-N 1-[(2r,3r,4s,5r)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-5-iodopyrimidine-2,4-dione Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C(I)=C1 RKSLVDIXBGWPIS-UAKXSSHOSA-N 0.000 description 1
- PISWNSOQFZRVJK-XLPZGREQSA-N 1-[(2r,4s,5r)-4-hydroxy-5-(hydroxymethyl)oxolan-2-yl]-5-methyl-2-sulfanylidenepyrimidin-4-one Chemical compound S=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 PISWNSOQFZRVJK-XLPZGREQSA-N 0.000 description 1
- YKBGVTZYEHREMT-KVQBGUIXSA-N 2'-deoxyguanosine Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@H]1C[C@H](O)[C@@H](CO)O1 YKBGVTZYEHREMT-KVQBGUIXSA-N 0.000 description 1
- CKTSBUTUHBMZGZ-SHYZEUOFSA-N 2'‐deoxycytidine Chemical compound O=C1N=C(N)C=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 CKTSBUTUHBMZGZ-SHYZEUOFSA-N 0.000 description 1
- JTBBWRKSUYCPFY-UHFFFAOYSA-N 2,3-dihydro-1h-pyrimidin-4-one Chemical compound O=C1NCNC=C1 JTBBWRKSUYCPFY-UHFFFAOYSA-N 0.000 description 1
- RHFUOMFWUGWKKO-XVFCMESISA-N 2-thiocytidine Chemical compound S=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@H](O)[C@@H](CO)O1 RHFUOMFWUGWKKO-XVFCMESISA-N 0.000 description 1
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 description 1
- ZAYHVCMSTBRABG-UHFFFAOYSA-N 5-Methylcytidine Natural products O=C1N=C(N)C(C)=CN1C1C(O)C(O)C(CO)O1 ZAYHVCMSTBRABG-UHFFFAOYSA-N 0.000 description 1
- AGFIRQJZCNVMCW-UAKXSSHOSA-N 5-bromouridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C(Br)=C1 AGFIRQJZCNVMCW-UAKXSSHOSA-N 0.000 description 1
- KDOPAZIWBAHVJB-UHFFFAOYSA-N 5h-pyrrolo[3,2-d]pyrimidine Chemical compound C1=NC=C2NC=CC2=N1 KDOPAZIWBAHVJB-UHFFFAOYSA-N 0.000 description 1
- BXJHWYVXLGLDMZ-UHFFFAOYSA-N 6-O-methylguanine Chemical compound COC1=NC(N)=NC2=C1NC=N2 BXJHWYVXLGLDMZ-UHFFFAOYSA-N 0.000 description 1
- UEHOMUNTZPIBIL-UUOKFMHZSA-N 6-amino-9-[(2r,3r,4s,5r)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-7h-purin-8-one Chemical compound O=C1NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O UEHOMUNTZPIBIL-UUOKFMHZSA-N 0.000 description 1
- HCAJQHYUCKICQH-VPENINKCSA-N 8-Oxo-7,8-dihydro-2'-deoxyguanosine Chemical compound C1=2NC(N)=NC(=O)C=2NC(=O)N1[C@H]1C[C@H](O)[C@@H](CO)O1 HCAJQHYUCKICQH-VPENINKCSA-N 0.000 description 1
- HDZZVAMISRMYHH-UHFFFAOYSA-N 9beta-Ribofuranosyl-7-deazaadenin Natural products C1=CC=2C(N)=NC=NC=2N1C1OC(CO)C(O)C1O HDZZVAMISRMYHH-UHFFFAOYSA-N 0.000 description 1
- 241000251468 Actinopterygii Species 0.000 description 1
- 102100022524 Alpha-1-antichymotrypsin Human genes 0.000 description 1
- 244000063299 Bacillus subtilis Species 0.000 description 1
- 235000014469 Bacillus subtilis Nutrition 0.000 description 1
- DWRXFEITVBNRMK-UHFFFAOYSA-N Beta-D-1-Arabinofuranosylthymine Natural products O=C1NC(=O)C(C)=CN1C1C(O)C(O)C(CO)O1 DWRXFEITVBNRMK-UHFFFAOYSA-N 0.000 description 1
- 241000283690 Bos taurus Species 0.000 description 1
- 208000026310 Breast neoplasm Diseases 0.000 description 1
- 239000002126 C01EB10 - Adenosine Substances 0.000 description 1
- 241000589875 Campylobacter jejuni Species 0.000 description 1
- 101000755689 Canis lupus familiaris Single-stranded DNA cytosine deaminase Proteins 0.000 description 1
- 241000283707 Capra Species 0.000 description 1
- 201000009030 Carcinoma Diseases 0.000 description 1
- 102000014914 Carrier Proteins Human genes 0.000 description 1
- 102000011727 Caspases Human genes 0.000 description 1
- 108010076667 Caspases Proteins 0.000 description 1
- 201000008890 Charcot-Marie-Tooth disease type 4J Diseases 0.000 description 1
- 241000867607 Chlorocebus sabaeus Species 0.000 description 1
- 108010077544 Chromatin Proteins 0.000 description 1
- 108091026890 Coding region Proteins 0.000 description 1
- 241000186216 Corynebacterium Species 0.000 description 1
- 241000918600 Corynebacterium ulcerans Species 0.000 description 1
- MIKUYHXYGGJMLM-GIMIYPNGSA-N Crotonoside Natural products C1=NC2=C(N)NC(=O)N=C2N1[C@H]1O[C@@H](CO)[C@H](O)[C@@H]1O MIKUYHXYGGJMLM-GIMIYPNGSA-N 0.000 description 1
- NYHBQMYGNKIUIF-UHFFFAOYSA-N D-guanosine Natural products C1=2NC(N)=NC(=O)C=2N=CN1C1OC(CO)C(O)C1O NYHBQMYGNKIUIF-UHFFFAOYSA-N 0.000 description 1
- HMFHBZSHGGEWLO-SOOFDHNKSA-N D-ribofuranose Chemical compound OC[C@H]1OC(O)[C@H](O)[C@@H]1O HMFHBZSHGGEWLO-SOOFDHNKSA-N 0.000 description 1
- 108091062167 DNA cytosine Proteins 0.000 description 1
- 230000009946 DNA mutation Effects 0.000 description 1
- 238000012270 DNA recombination Methods 0.000 description 1
- 230000033616 DNA repair Effects 0.000 description 1
- 241000252212 Danio rerio Species 0.000 description 1
- CKTSBUTUHBMZGZ-UHFFFAOYSA-N Deoxycytidine Natural products O=C1N=C(N)C=CN1C1OC(CO)C(O)C1 CKTSBUTUHBMZGZ-UHFFFAOYSA-N 0.000 description 1
- 108010008532 Deoxyribonuclease I Proteins 0.000 description 1
- 102000007260 Deoxyribonuclease I Human genes 0.000 description 1
- 108010042407 Endonucleases Proteins 0.000 description 1
- 102000004533 Endonucleases Human genes 0.000 description 1
- 108700024394 Exon Proteins 0.000 description 1
- 241000282326 Felis catus Species 0.000 description 1
- 101150106478 GPS1 gene Proteins 0.000 description 1
- 206010064571 Gene mutation Diseases 0.000 description 1
- 241000238631 Hexapoda Species 0.000 description 1
- 101000964378 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3A Proteins 0.000 description 1
- 101000964385 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3B Proteins 0.000 description 1
- 101000964383 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3C Proteins 0.000 description 1
- 101000964382 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3D Proteins 0.000 description 1
- 101000964377 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3F Proteins 0.000 description 1
- 101000742769 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3H Proteins 0.000 description 1
- 101000605639 Homo sapiens Phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit alpha isoform Proteins 0.000 description 1
- 241000713772 Human immunodeficiency virus 1 Species 0.000 description 1
- UGQMRVRMYYASKQ-KQYNXXCUSA-N Inosine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(O)=C2N=C1 UGQMRVRMYYASKQ-KQYNXXCUSA-N 0.000 description 1
- 102000012330 Integrases Human genes 0.000 description 1
- 108010061833 Integrases Proteins 0.000 description 1
- QIVBCDIJIAJPQS-VIFPVBQESA-N L-tryptophane Chemical compound C1=CC=C2C(C[C@H](N)C(O)=O)=CNC2=C1 QIVBCDIJIAJPQS-VIFPVBQESA-N 0.000 description 1
- OUYCCCASQSFEME-QMMMGPOBSA-N L-tyrosine Chemical compound OC(=O)[C@@H](N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-QMMMGPOBSA-N 0.000 description 1
- 241000270322 Lepidosauria Species 0.000 description 1
- 108090000364 Ligases Proteins 0.000 description 1
- 102000003960 Ligases Human genes 0.000 description 1
- 241000282560 Macaca mulatta Species 0.000 description 1
- 102100025169 Max-binding protein MNT Human genes 0.000 description 1
- 101100377883 Mus musculus Apobec1 gene Proteins 0.000 description 1
- 101100489911 Mus musculus Apobec3 gene Proteins 0.000 description 1
- 108010021466 Mutant Proteins Proteins 0.000 description 1
- 102000008300 Mutant Proteins Human genes 0.000 description 1
- 102100022691 NACHT, LRR and PYD domains-containing protein 3 Human genes 0.000 description 1
- 101710126825 NACHT, LRR and PYD domains-containing protein 3 Proteins 0.000 description 1
- 241000588653 Neisseria Species 0.000 description 1
- 241000244206 Nematoda Species 0.000 description 1
- 108700019961 Neoplasm Genes Proteins 0.000 description 1
- 102000048850 Neoplasm Genes Human genes 0.000 description 1
- 101710149004 Nuclease P1 Proteins 0.000 description 1
- 108700020796 Oncogene Proteins 0.000 description 1
- 208000012868 Overgrowth Diseases 0.000 description 1
- 101100214779 Pan troglodytes APOBEC3G gene Proteins 0.000 description 1
- 241001494479 Pecora Species 0.000 description 1
- 241000009328 Perro Species 0.000 description 1
- 102100038223 Phenylalanine-4-hydroxylase Human genes 0.000 description 1
- 108090000430 Phosphatidylinositol 3-kinases Proteins 0.000 description 1
- 102100038332 Phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit alpha isoform Human genes 0.000 description 1
- 101150063858 Pik3ca gene Proteins 0.000 description 1
- 108700001094 Plant Genes Proteins 0.000 description 1
- 241000350158 Prioria balsamifera Species 0.000 description 1
- 108010076504 Protein Sorting Signals Proteins 0.000 description 1
- 241001647888 Psychroflexus Species 0.000 description 1
- 238000010357 RNA editing Methods 0.000 description 1
- 230000026279 RNA modification Effects 0.000 description 1
- 108020004511 Recombinant DNA Proteins 0.000 description 1
- PYMYPHUHKUWMLA-LMVFSUKVSA-N Ribose Natural products OC[C@@H](O)[C@@H](O)[C@@H](O)C=O PYMYPHUHKUWMLA-LMVFSUKVSA-N 0.000 description 1
- 241000283984 Rodentia Species 0.000 description 1
- 230000018199 S phase Effects 0.000 description 1
- 240000004808 Saccharomyces cerevisiae Species 0.000 description 1
- 235000014680 Saccharomyces cerevisiae Nutrition 0.000 description 1
- 108020004688 Small Nuclear RNA Proteins 0.000 description 1
- 102000039471 Small Nuclear RNA Human genes 0.000 description 1
- 108020004459 Small interfering RNA Proteins 0.000 description 1
- 102220497176 Small vasohibin-binding protein_T47D_mutation Human genes 0.000 description 1
- 241000202917 Spiroplasma Species 0.000 description 1
- 241000194056 Streptococcus iniae Species 0.000 description 1
- 238000010459 TALEN Methods 0.000 description 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical class OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 description 1
- 108010043645 Transcription Activator-Like Effector Nucleases Proteins 0.000 description 1
- 108091023040 Transcription factor Proteins 0.000 description 1
- 102000040945 Transcription factor Human genes 0.000 description 1
- 108020004566 Transfer RNA Proteins 0.000 description 1
- QIVBCDIJIAJPQS-UHFFFAOYSA-N Tryptophan Natural products C1=CC=C2C(CC(N)C(O)=O)=CNC2=C1 QIVBCDIJIAJPQS-UHFFFAOYSA-N 0.000 description 1
- 206010067584 Type 1 diabetes mellitus Diseases 0.000 description 1
- 241000251539 Vertebrata <Metazoa> Species 0.000 description 1
- 241000700605 Viruses Species 0.000 description 1
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 description 1
- 230000002159 abnormal effect Effects 0.000 description 1
- 238000009825 accumulation Methods 0.000 description 1
- 230000002378 acidificating effect Effects 0.000 description 1
- 150000007513 acids Chemical class 0.000 description 1
- 239000012190 activator Substances 0.000 description 1
- 239000013543 active substance Substances 0.000 description 1
- 101150063416 add gene Proteins 0.000 description 1
- 229960005305 adenosine Drugs 0.000 description 1
- 230000002411 adverse Effects 0.000 description 1
- 108010091628 alpha 1-Antichymotrypsin Proteins 0.000 description 1
- HMFHBZSHGGEWLO-UHFFFAOYSA-N alpha-D-Furanose-Ribose Natural products OCC1OC(O)C(O)C1O HMFHBZSHGGEWLO-UHFFFAOYSA-N 0.000 description 1
- 150000001408 amides Chemical group 0.000 description 1
- 238000004458 analytical method Methods 0.000 description 1
- 230000001475 anti-trypsic effect Effects 0.000 description 1
- 230000002155 anti-virotic effect Effects 0.000 description 1
- 239000000427 antigen Substances 0.000 description 1
- 108091007433 antigens Proteins 0.000 description 1
- 102000036639 antigens Human genes 0.000 description 1
- PYMYPHUHKUWMLA-WDCZJNDASA-N arabinose Chemical compound OC[C@@H](O)[C@@H](O)[C@H](O)C=O PYMYPHUHKUWMLA-WDCZJNDASA-N 0.000 description 1
- PYMYPHUHKUWMLA-UHFFFAOYSA-N arabinose Natural products OCC(O)C(O)C(O)C=O PYMYPHUHKUWMLA-UHFFFAOYSA-N 0.000 description 1
- 125000000637 arginyl group Chemical group N[C@@H](CCCNC(N)=N)C(=O)* 0.000 description 1
- 210000004507 artificial chromosome Anatomy 0.000 description 1
- 208000005980 beta thalassemia Diseases 0.000 description 1
- SRBFZHDQGSBBOR-UHFFFAOYSA-N beta-D-Pyranose-Lyxose Natural products OC1COC(O)C(O)C1O SRBFZHDQGSBBOR-UHFFFAOYSA-N 0.000 description 1
- IQFYYKKMVGJFEH-UHFFFAOYSA-N beta-L-thymidine Natural products O=C1NC(=O)C(C)=CN1C1OC(CO)C(O)C1 IQFYYKKMVGJFEH-UHFFFAOYSA-N 0.000 description 1
- 108091008324 binding proteins Proteins 0.000 description 1
- 229960002685 biotin Drugs 0.000 description 1
- YBJHBAHKTGYVGT-ZKWXMUAHSA-N biotin Natural products N1C(=O)N[C@@H]2[C@H](CCCCC(=O)O)SC[C@@H]21 YBJHBAHKTGYVGT-ZKWXMUAHSA-N 0.000 description 1
- 235000020958 biotin Nutrition 0.000 description 1
- 239000011616 biotin Substances 0.000 description 1
- 239000008280 blood Substances 0.000 description 1
- 210000004369 blood Anatomy 0.000 description 1
- 125000000837 carbohydrate group Chemical group 0.000 description 1
- 231100000357 carcinogen Toxicity 0.000 description 1
- 231100000315 carcinogenic Toxicity 0.000 description 1
- 239000003183 carcinogenic agent Substances 0.000 description 1
- 239000003054 catalyst Substances 0.000 description 1
- 238000000423 cell based assay Methods 0.000 description 1
- 230000008859 change Effects 0.000 description 1
- 238000012512 characterization method Methods 0.000 description 1
- 238000006243 chemical reaction Methods 0.000 description 1
- 210000003483 chromatin Anatomy 0.000 description 1
- 238000010276 construction Methods 0.000 description 1
- 125000004122 cyclic group Chemical group 0.000 description 1
- 230000001086 cytosolic effect Effects 0.000 description 1
- 230000001419 dependent effect Effects 0.000 description 1
- 206010013023 diphtheria Diseases 0.000 description 1
- 210000002257 embryonic structure Anatomy 0.000 description 1
- 238000005516 engineering process Methods 0.000 description 1
- 230000002255 enzymatic effect Effects 0.000 description 1
- 108010052305 exodeoxyribonuclease III Proteins 0.000 description 1
- 239000013604 expression vector Substances 0.000 description 1
- 125000004030 farnesyl group Chemical group [H]C([*])([H])C([H])=C(C([H])([H])[H])C([H])([H])C([H])([H])C([H])=C(C([H])([H])[H])C([H])([H])C([H])([H])C([H])=C(C([H])([H])[H])C([H])([H])[H] 0.000 description 1
- 125000005313 fatty acid group Chemical group 0.000 description 1
- 238000007306 functionalization reaction Methods 0.000 description 1
- 238000003209 gene knockout Methods 0.000 description 1
- 230000030279 gene silencing Effects 0.000 description 1
- 238000012226 gene silencing method Methods 0.000 description 1
- 238000010363 gene targeting Methods 0.000 description 1
- 230000012010 growth Effects 0.000 description 1
- 229940029575 guanosine Drugs 0.000 description 1
- 230000036541 health Effects 0.000 description 1
- 108010049074 hemoglobin B Proteins 0.000 description 1
- 206010073071 hepatocellular carcinoma Diseases 0.000 description 1
- 231100000844 hepatocellular carcinoma Toxicity 0.000 description 1
- 150000002402 hexoses Chemical class 0.000 description 1
- 230000013632 homeostatic process Effects 0.000 description 1
- 102000048646 human APOBEC3A Human genes 0.000 description 1
- 102000048415 human APOBEC3B Human genes 0.000 description 1
- 102000048419 human APOBEC3C Human genes 0.000 description 1
- 102000043429 human APOBEC3D Human genes 0.000 description 1
- 102000049338 human APOBEC3F Human genes 0.000 description 1
- 102000044839 human APOBEC3H Human genes 0.000 description 1
- 125000002887 hydroxy group Chemical group [H]O* 0.000 description 1
- 206010020718 hyperplasia Diseases 0.000 description 1
- 230000002390 hyperplastic effect Effects 0.000 description 1
- 230000003463 hyperproliferative effect Effects 0.000 description 1
- 230000001900 immune effect Effects 0.000 description 1
- 210000000987 immune system Anatomy 0.000 description 1
- 208000015181 infectious disease Diseases 0.000 description 1
- 230000000977 initiatory effect Effects 0.000 description 1
- CDAISMWEOUEBRE-GPIVLXJGSA-N inositol Chemical group O[C@H]1[C@H](O)[C@@H](O)[C@H](O)[C@H](O)[C@@H]1O CDAISMWEOUEBRE-GPIVLXJGSA-N 0.000 description 1
- 210000004966 intestinal stem cell Anatomy 0.000 description 1
- 210000003734 kidney Anatomy 0.000 description 1
- 239000006166 lysate Substances 0.000 description 1
- 230000002132 lysosomal effect Effects 0.000 description 1
- 230000035800 maturation Effects 0.000 description 1
- 230000007246 mechanism Effects 0.000 description 1
- 108091005763 multidomain proteins Proteins 0.000 description 1
- 231100000707 mutagenic chemical Toxicity 0.000 description 1
- 231100000243 mutagenic effect Toxicity 0.000 description 1
- 230000000869 mutational effect Effects 0.000 description 1
- 239000013642 negative control Substances 0.000 description 1
- 230000030147 nuclear export Effects 0.000 description 1
- 230000000269 nucleophilic effect Effects 0.000 description 1
- 125000003835 nucleoside group Chemical group 0.000 description 1
- 238000005457 optimization Methods 0.000 description 1
- 150000002894 organic compounds Chemical class 0.000 description 1
- 210000002220 organoid Anatomy 0.000 description 1
- 230000002018 overexpression Effects 0.000 description 1
- 150000003905 phosphatidylinositols Chemical class 0.000 description 1
- 150000004713 phosphodiesters Chemical group 0.000 description 1
- 230000035479 physiological effects, processes and functions Effects 0.000 description 1
- 238000002264 polyacrylamide gel electrophoresis Methods 0.000 description 1
- 229920002704 polyhistidine Polymers 0.000 description 1
- 102000054765 polymorphisms of proteins Human genes 0.000 description 1
- 239000013641 positive control Substances 0.000 description 1
- 230000003389 potentiating effect Effects 0.000 description 1
- 239000002243 precursor Substances 0.000 description 1
- 230000001855 preneoplastic effect Effects 0.000 description 1
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 description 1
- MWWATHDPGQKSAR-UHFFFAOYSA-N propyne Chemical compound CC#C MWWATHDPGQKSAR-UHFFFAOYSA-N 0.000 description 1
- 108020001580 protein domains Proteins 0.000 description 1
- 238000002818 protein evolution Methods 0.000 description 1
- 230000004853 protein function Effects 0.000 description 1
- 238000011002 quantification Methods 0.000 description 1
- 238000003259 recombinant expression Methods 0.000 description 1
- 230000002441 reversible effect Effects 0.000 description 1
- RHFUOMFWUGWKKO-UHFFFAOYSA-N s2C Natural products S=C1N=C(N)C=CN1C1C(O)C(O)C(CO)O1 RHFUOMFWUGWKKO-UHFFFAOYSA-N 0.000 description 1
- 238000002864 sequence alignment Methods 0.000 description 1
- 125000003607 serino group Chemical group [H]N([H])[C@]([H])(C(=O)[*])C(O[H])([H])[H] 0.000 description 1
- 208000007056 sickle cell anemia Diseases 0.000 description 1
- 238000002415 sodium dodecyl sulfate polyacrylamide gel electrophoresis Methods 0.000 description 1
- 125000006850 spacer group Chemical group 0.000 description 1
- 238000005309 stochastic process Methods 0.000 description 1
- 108010018381 streptavidin-binding peptide Proteins 0.000 description 1
- 230000002194 synthesizing effect Effects 0.000 description 1
- 230000009897 systematic effect Effects 0.000 description 1
- 238000012360 testing method Methods 0.000 description 1
- 230000001225 therapeutic effect Effects 0.000 description 1
- 229940104230 thymidine Drugs 0.000 description 1
- 230000032258 transport Effects 0.000 description 1
- HDZZVAMISRMYHH-KCGFPETGSA-N tubercidin Chemical compound C1=CC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O HDZZVAMISRMYHH-KCGFPETGSA-N 0.000 description 1
- 241001515965 unidentified phage Species 0.000 description 1
- 239000013598 vector Substances 0.000 description 1
- 210000002845 virion Anatomy 0.000 description 1
- 230000003612 virological effect Effects 0.000 description 1
- 229910052725 zinc Inorganic materials 0.000 description 1
- UGZADUVQMDAIAO-UHFFFAOYSA-L zinc hydroxide Chemical compound [OH-].[OH-].[Zn+2] UGZADUVQMDAIAO-UHFFFAOYSA-L 0.000 description 1
- 229940007718 zinc hydroxide Drugs 0.000 description 1
- 229910021511 zinc hydroxide Inorganic materials 0.000 description 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
- A61K47/61—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule the organic macromolecular compound being a polysaccharide or a derivative thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6472—Cysteine endopeptidases (3.4.22)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
- C12P19/00—Preparation of compounds containing saccharide radicals
- C12P19/26—Preparation of nitrogen-containing carbohydrates
- C12P19/28—N-glycosides
- C12P19/30—Nucleotides
- C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
- C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/22—Cysteine endopeptidases (3.4.22)
- C12Y304/22062—Caspase-9 (3.4.22.62)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04001—Cytosine deaminase (3.5.4.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04004—Adenosine deaminase (3.5.4.4)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04005—Cytidine deaminase (3.5.4.5)
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/156—Polymorphic or mutational markers
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/22—Endodeoxyribonucleases producing 3'-phosphomonoesters (3.1.22)
-
- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
- Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
- Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
- Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Analytical Chemistry (AREA)
- Crystallography & Structural Chemistry (AREA)
- Physical Education & Sports Medicine (AREA)
- Urology & Nephrology (AREA)
- Pathology (AREA)
- Psychiatry (AREA)
- Obesity (AREA)
Abstract
Description
本出願は、35U.S.C.§119(e)の下で、2013年12月12日に出願された米国仮特許出願第61/915,386号明細書および2014年4月16日に出願された米国仮特許出願第61/980,333号明細書に対する優先権を主張し、35U.S.C.§120の下で、米国特許出願第14/325,815号明細書、米国特許出願第14/326,109号明細書、米国特許出願第14/326,140号明細書、米国特許出願第14/326,269号明細書、米国特許出願第14/326,290号明細書、米国特許出願第14/326,318号明細書および米国特許出願第14/326,303号明細書(全て2014年7月8日に出願されている)に対する優先権も主張し、これらはそれぞれ参照により本明細書に援用される。
本発明は、国防高等研究計画局(DARPA)により付与された助成金HR0011−11−2−0003、国立衛生研究所(NIH)により付与された助成金GM095501、ならびに宇宙および海軍戦闘システムセンター(Space and Naval Warfare Systems Center)(SPAWAR)により付与された助成金N66001−12−C−4207の下で米国政府の支援によってなされた。米国政府は本発明においてある程度の権利を有する。
本明細書および特許請求の範囲で使用する場合、単数形「1つの(a)」、「1つの(an)」および「その(the)」は、文脈が別途明確に示さない限り単数への言及および複数への言及を含む。そのため、例えば「薬剤」への言及は、単一の薬剤および複数のそのような薬剤を含む。
dCas9(D10AおよびH840A):
本開示のいくつかの態様は、(i)ヌクレアーゼ不活性型Cas9酵素またはヌクレアーゼ不活性型Cas9ドメインと、(ii)核酸編集酵素または核酸編集ドメインとを含む融合タンパク質を提供する。いくつかの実施形態では、この核酸編集酵素または核酸編集ドメインは、DNA編集酵素またはDNA編集ドメインである。いくつかの実施形態では、この核酸編集酵素はデアミナーゼ活性を有する。いくつかの実施形態では、この核酸編集酵素または核酸編集ドメインは、デアミナーゼドメインを含む、またはデアミナーゼドメインである。いくつかの実施形態では、このデアミナーゼはシチジンデアミナーゼである。いくつかの実施形態では、このデアミナーゼは、アポリポタンパク質B mRNA編集複合体(APOBEC)ファミリのデアミナーゼである。いくつかの実施形態では、このデアミナーゼはAPOBEC1ファミリのデアミナーゼである。いくつかの実施形態では、このデアミナーゼは活性化誘導シチジンデアミナーゼ(AID)である。いくつかの実施形態では、このデアミナーゼはACF1/ASEデアミナーゼである。いくつかの実施形態では、このデアミナーゼはアデノシンデアミナーゼである。いくつかの実施形態では、このデアミナーゼはADATファミリのデアミナーゼである。数種類の核酸編集酵素および核酸編集ドメイン、ならびにそのような酵素またはドメインを含むCas9融合タンパク質を本明細書において詳細に説明する。追加の好適な核酸編集酵素または核酸編集ドメインが、本開示をベースとして当業者に明らかであろう。
[NH2]−[核酸編集酵素または核酸編集ドメイン]−[Cas9]−[COOH]、または
[NH2]−[Cas9]−[核酸編集酵素または核酸編集ドメイン]−[COOH]
(上記構造中、NH2は融合タンパク質のN末端であり、COOHは融合タンパク質のC末端である)。
[NH2]−[NLS]−[Cas9]−[デアミナーゼ]−[COOH]、
[NH2]−[NLS]−[デアミナーゼ]−[Cas9]−−[COOH]、
[NH2]−[Cas9]−[デアミナーゼ]−[COOH]、または
[NH2]−[デアミナーゼ]−[Cas9]−[COOH]
(上記構造中、NLSは核局在化シグナルであり、NH2は融合タンパク質のN末端であり、COOHは融合タンパク質のC末端である)。いくつかの実施形態では、Cas9とデアミナーゼとの間にリンカーが挿入されている。いくつかの実施形態では、NLSはデアミナーゼおよび/またはCas9ドメインのC末端に位置している。いくつかの実施形態では、NLSはデアミナーゼとCas9ドメインとの間に位置している。配列タグ等の追加の特徴も存在し得る。
マウスAID:
イヌAID:
ウシAID:
マウスAPOBEC−3:
ラットAPOBEC−3:
アカゲザルAPOBEC−3G:
チンパンジーAPOBEC−3G:
ミドリザルAPOBEC−3G:
ヒトAPOBEC−3G:
ヒトAPOBEC−3F:
ヒトAPOBEC−3B:
ヒトAPOBEC−3C:
ヒトAPOBEC−3A:
ヒトAPOBEC−3H:
ヒトAPOBEC−3D:
ヒトAPOBEC−1:
ヒトADAT−1:
いくつかの実施形態は、本明細書に記載したCas9 DNA編集融合タンパク質の使用方法を提供する。いくつかの実施形態では、この融合タンパク質を使用して、標的核酸塩基、例えばC残基を脱アミノ化させることにより核酸に点変異を導入する。いくつかの実施形態では、標的核酸塩基の脱アミノ化により遺伝的欠陥が修正され、例えば遺伝子産物の機能の喪失を引き起こす点変異が修正される。いくつかの実施形態では、遺伝的欠陥は疾患または障害に関連しており、例えばI型糖尿病等のリソソーム蓄積障害または代謝性疾患に関連している。いくつかの実施形態では、本明細書に記載した方法を使用して、疾患または障害に関連する遺伝子産物をコードする遺伝子またはアレルに不活性型の点変異を導入する。例えば、いくつかの実施形態では、本明細書において、(例えば増殖性疾患の処置で)Cas9 DNA編集融合タンパク質を利用して発がん遺伝子に不活性型の点変異を導入する方法が提供される。いくつかの実施形態では、不活性型の変異によりコート配列中に未成熟終止コドンが生成され得、これにより短縮遺伝子産物が発現され、例えば完全長タンパク質の機能を欠く短縮タンパク質が発現され得る。
ベルナール・スーリエ症候群(BSS)− 血小板膜糖タンパク質IXにおける残基24でのシステインからアルギニン(T>C変異):
表皮剥離性角化症(EHK)− ケラチン1における残基161でのロイシンからプロリンへの変異(T>C変異):
慢性閉塞性肺疾患(COPD)− α1−抗トリプシンにおける残基54でのロイシンからプロリンへの変異(T>C変異):
慢性閉塞性肺疾患(COPD)− α1−抗キモトリプシンにおける残基78でのロイシンからプロリンへの変異(T>C変異):
神経芽細胞腫(NB)− カスパーゼ−9における残基197でのロイシンからプロリンへの変異(T>C変異):
シャルコー・マリー・トゥース病4J型 − FIG4における残基41でのイソロイシンからトレオニンへの変異(T>C変異):
フォン・ウィルブランド病(vWD)− フォン・ウィルブランド因子における残基1272でのシステインからアルギニンへの変異(T>C変異):
先天性筋強直症 − 筋肉の塩化物チャンネル遺伝子CLCN1における277位でのシステインからアルギニンへの変異(T>C変異):
遺伝性腎アミロイドーシス − アポリポタンパク質AIIにおける残基111での終止コドンからアルギニンへの変異(T>C変異):
拡張型心筋症(DCM)− FOXD4遺伝子における148位でのトリプトファンからアルギニンへの変異(T>C変異):
遺伝性リンパ水腫 − VEGFR3チロシンキナーゼにおける残基1035でのヒスチジンからアルギニンへの変異(A>G変異):
家族性アルツハイマー病 − プレセニリン1における残基143でのイソロイシンからバリンへの変異(A>G変異):
プリオン病 − プリオンタンパク質における残基129でのメチオニンからバリンへの変異(A>G変異):
慢性乳児神経皮膚関節症候群(CINCA)− クリオピリンにおける残基570でのチロシンからシステインへの変異(A>G変異):
デスミン関連心筋症(DRM)− αBクリスタリンにおける残基120でのアルギニンからグリシンへの変異(A>G変異):
上記に記載した配列は例示であり、本開示の範囲を限定することは意図されていないことを理解しなければならない。疾患と関連しているおよびCas9:核酸編集酵素/ドメイン融合タンパク質による修正に適している点変異の好適な追加の配列、ならびに好適なガイドRNA配列が、本開示をベースとして当業者に明らかであろう。
本開示のいくつかの態様は、本明細書に記載した融合タンパク質のデアミナーゼ活性の検出に使用し得るレポーターシステムを提供する。いくつかの実施形態では、このレポーターシステムは、デアミナーゼ活性がルシフェラーゼの発現をもたらすルシフェラーゼベースのアッセイである。デアミナーゼドメイン(例えばAIDドメイン)の潜在的な基質無差別性の影響を最小化するために、非意図的に脱アミノ化の標的となる可能性がある残基(例えば、レポーターシステム内のssDNA上に潜在的に存在する可能性があるオフターゲットC残基)の数を最小化する。いくつかの実施形態では、目的の標的残基は、翻訳を開始することができないルシフェラーゼ遺伝子のACG変異した開始コドンに位置し得る。所望のデアミナーゼ活性によりACG>AUG修飾が生じ、そのため、ルシフェラーゼの翻訳ならびにデアミナーゼ活性の検出および定量化が可能になる。
例示的なCas9:デアミナーゼ融合タンパク質を下記に記載する。
Cas9:ヒトAID融合体(C末端)
Cas9:ヒトAID融合体(N末端)
Cas9:マウスAID融合体(C末端)
Cas9:ヒトAPOBEC−3G融合体(N末端)
Cas9:ヒトAPOBEC−1融合体(N末端)
Cas9:ヒトADAT1融合体(N末端)
Cas9:ヒトADAT1融合体(末端)
結果としてPI3Kタンパク質においてH1047Rアミノ酸置換が生じる、PI3KCA遺伝子のエクソン20におけるA3140G点変異を、この変異タンパク質をコードする核酸と、Cas9:AID(配列番号30)融合タンパク質またはCas9:APOBEC1(配列番号92)融合タンパク質およびこの融合タンパク質の標的をこのコーディングPI3KCA遺伝子中の変異部位に定める適切に設計したsgRNAとを接触させることにより修正する。各エクソン20配列のゲノムPCR、例えば3000〜3250個のヌクレオチドのPCR増幅産物の生成、およびその後のPCT増幅産物の配列決定により、A3140G点変異を確認する。
結果としてPSEN1タンパク質においてI143Vアミノ酸置換が生じる、プレセニリン1(PSEN1)遺伝子のコドン143におけるA−>G点変異を、この変異PSEN1タンパク質をコードする核酸と、Cas9:AID(配列番号30)融合タンパク質またはCas9:APOBEC1(配列番号92)融合タンパク質およびこの融合タンパク質の標的をこのコーディングPSEN1遺伝子中の変異部位に定める適切に設計したsgRNAとを接触させることにより修正する。家族性アルツハイマー病に関連する例示的なPSEN1 I143V変異の説明に関して例えばGallo et.al.,J.Alzheimer’s disease.2011;25:425−431を参照されたい。各PSEN1配列のゲノムPCR、例えばエクソン143を中心とする約100〜250個のヌクレオチドのPCR増幅産物の生成、およびその後のPCT増幅産物の配列決定により、A−>G点変異を確認する。
結果としてα1−抗トリプシンタンパク質においてL55Pアミノ酸置換が生じる、α1−抗トリプシン遺伝子のコドン55におけるT−>C点変異を、この変異α1−抗トリプシンタンパク質をコードする核酸と、Cas9:ADAT1融合タンパク質(配列番号35または配列番号36)およびこの融合タンパク質の標的をこのコーディングα1−抗トリプシン遺伝子中の変異部位に定める適切に設計したsgRNAとを接触させることにより修正する。慢性閉塞性肺疾患(COPD)に関連する例示的なコドン55 T−>C変異のより詳細な説明に関して例えばPoller et al.,Genomics.1993;17:740−743を参照されたい。コドン55をコードする各α1−抗トリプシン配列のゲノムPCR、例えば約100〜250個のヌクレオチドのPCR増幅産物の生成、およびその後のPCT増幅産物の配列決定により、T−>C点変異を確認する。
結果としてフォン・ウィルブランド因子タンパク質においてC509Aアミノ酸置換が生じる、フォン・ウィルブランド因子遺伝子のコドン509におけるT−>C点変異を、この変異フォン・ウィルブランド因子タンパク質をコードする核酸と、Cas9:ADAT1融合タンパク質(配列番号35または配列番号36)およびこの融合タンパク質の標的をこのコーディングフォン・ウィルブランド因子遺伝子のセンス鎖中の変異部位に定める適切に設計したsgRNAとを接触させることにより修正する。フォン・ウィルブランド病(vWD)に関連する例示的なフォン・ウィルブランド因子C509A変異の説明に関して例えばLavergne et al.,Br.J.Haematol.1992;82:66−7を参照されたい。各フォン・ウィルブランド因子ゲノム配列のゲノムPCR、例えばエクソン509を中心とする約100〜250個のヌクレオチドのPCR増幅産物の生成、およびその後のPCT増幅産物の配列決定により、T−>C点変異を確認する。
結果としてカスパーゼ−9タンパク質においてL197Pアミノ酸置換が生じる、カスパーゼ−9遺伝子のコドン197におけるT−>C点変異を、この変異カスパーゼ−9タンパク質をコードする核酸と、Cas9:ADAT1融合タンパク質(配列番号35または配列番号36)およびこの融合タンパク質の標的をこのコーディングカスパーゼ−9遺伝子のセンス鎖中の変異部位に定める適切に設計したsgRNAとを接触させることにより修正する。神経芽細胞腫(NB)に関連する例示的なカスパーゼ−9 L197P変異の説明に関して例えばLenk et al.,PLoS Genetics.2011;7:e1002104を参照されたい。各カスパーゼ−9ゲノム配列のゲノムPCR、例えばエクソン197を中心とする約100〜250個のヌクレオチドのPCR増幅産物の生成、およびその後のPCT増幅産物の配列決定により、T−>C点変異を確認する。
下記の異なるリンカーを有する2種のdCas9−APOBEC1融合タンパク質を生成した。
rAPOBEC1_GGS_dCas9:
rAPOBEC1_(GGS)3_dCas9:
1.Humbert O,Davis L,Maizels N.Targeted gene therapies:tools,applications,optimization.Crit Rev Biochem Mol.2012;47(3):264−81.PMID:22530743.
2.Perez−Pinera P,Ousterout DG,Gersbach CA.Advances in targeted genome editing.Curr Opin Chem Biol.2012;16(3−4):268−77.PMID:22819644.
3.Urnov FD,Rebar EJ,Holmes MC,Zhang HS,Gregory PD.Genome editing with engineered zinc finger nucleases.Nat Rev Genet.2010;11(9):636−46.PMID:20717154.
4.Joung JK,Sander JD.TALENs:a widely applicable technology for targeted genome editing.Nat Rev Mol Cell Biol.2013;14(1):49−55.PMID:23169466.
5.Charpentier E,Doudna JA.Biotechnology:Rewriting a genome.Nature.2013;495,(7439):50−1.PMID:23467164.
6.Pan Y,Xia L,Li AS,Zhang X,Sirois P,Zhang J,Li K.Biological and biomedical applications of engineered nucleases.Mol Biotechnol.2013;55(1):54−62.PMID:23089945.
7.De Souza,N.Primer:genome editing with engineered nucleases.Nat Methods.2012;9(1):27.PMID:22312638.
8.Santiago Y,Chan E,Liu PQ,Orlando S,Zhang L,Urnov FD,Holmes MC,Guschin D,Waite A,Miller JC,Rebar EJ,Gregory PD,Klug A,Collingwood TN.Targeted gene knockout in mammalian cells by using engineered zinc−finger nucleases.Proc Natl Acad Sci USA.2008;105(15):5809−14.PMID:18359850.
9.Cargill M,Altshuler D,Ireland J,Sklar P,Ardlie K,Patil N,Lane CR,Lim EP,Kalyanaraman N,Nemesh J,Ziaugra L,Friedland L,Rolfe A,Warrington J,Lipshutz R,Daley GQ,Lander ES.Characterization of single−nucleotide polymorphisms in coding regions of human genes.Nat Genet.1999;22(3):231−8.PMID:10391209.
10.Jansen R,van Embden JD,Gaastra W,Schouls LM.Identification of genes that are associated with DNA repeats in prokaryotes.Mol Microbiol.2002;43(6):1565−75.PMID:11952905.
11.Mali P,Esvelt KM,Church GM.Cas9 as a versatile tool for engineering biology.Nat Methods.2013;10(10):957−63.PMID:24076990.
12.Jore MM,Lundgren M,van Duijin E,Bultema JB,Westra ER,Waghmare SP,Wiedenheft B,Pul U,Wurm R,Wagner R,Beijer MR,Barendregt A,Shou K,Snijders AP,Dickman MJ,Doudna JA,Boekema EJ,Heck AJ,van der Oost J,Brouns SJ.Structural basis for CRISPR RNA−guided DNA recognition by Cascade.Nat Struct Mol Biol.2011;18(5):529−36.PMID:21460843.
13.Horvath P,Barrangou R.CRISPR/Cas,the immune system of bacteria and archaea.Science.2010;327(5962):167−70.PMID:20056882.
14.Wiedenheft B,Sternberg SH,Doudna JA.RNA−guided genetic silencing systems in bacteria and archaea.Nature.2012;482(7385):331−8.PMID:22337052.
15.Gasiunas G,Siksnys V.RNA−dependent DNA endonuclease Cas9 of the CRISPR system:Holy Grail of genome editing?Trends Microbiol.2013;21(11):562−7.PMID:24095303.
16.Qi LS,Larson MH,Gilbert LA,Doudna JA,Weissman JS,Arkin AP,Lim WA.Repurposing CRISPR as an RNA−guided platform for sequence−specific control of gene expression.Cell.2013;152(5):1173−83.PMID:23452860.
17.Perez−Pinera P,Kocak DD,Vockley CM,Adler AF,Kabadi AM,Polstein LR,Thakore PI,Glass KA,Ousterout DG,Leong KW,Guilak F,Crawford GE,Reddy TE,Gersbach CA.RNA−guided gene activation by CRISPR−Cas9−based transcription factors.Nat Methods.2013;10(10):973−6.PMID:23892895.
18.Mali P,Aach J,Stranges PB,Esvelt KM,Moosburner M,Kosuri S,Yang L,Church GM.CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering.Nat Biotechnol.2013;31(9):833−8.PMID:23907171.
19.Gilbert LA,Larson MH,Morsut L,Liu Z,Brar GA,Torres SE,Stern−Ginossar N,Brandman O,Whitehead EH,Doudna JA,Lim WA,Weissman JS,Qi LS.CRISPR−mediated modular RNA−guided regulation of transcription in eukaryotes.Cell.2013;154(2):442−51.PMID:23849981.
20.Larson MH,Gilbert LA,Wang X,Lim WA,Weissman JS,Qi LS.CRISPR interference(CRISPRi) for sequence−specific control of gene expression.Nat Protoc.2013;8(11):2180−96.PMID:24136345.
21.Mali P,Yang L,Esvelt KM,Aach J,Guell M,DiCarlo JE,Norville JE,Church GM.RNA−guided human genome engineering via Cas9.Science.2013;339(6121):823−6.PMID:23287722.
22.Cole−Strauss A,Yoon K,Xiang Y,Byrne BC,Rice MC,Gryn J,Holloman WK,Kmiec EB.Correction of the mutation responsible for sickle cell anemia by an RNA−DNA oligonucleotide.Science.1996;273(5280):1386−9.PMID:8703073.
23.Tagalakis AD,Owen JS,Simons JP.Lack of RNA−DNA oligonucleotide(chimeraplast) mutagenic activity in mouse embryos.Mol Reprod Dev.2005;71(2):140−4.PMID:15791601.
24.Ray A,Langer M.Homologous recombination:ends as the means.Trends Plant Sci.2002;7(10):435−40.PMID 12399177.
25.Britt AB,May GD.Re−engineering plant gene targeting.Trends Plant Sci.2003;8(2):90−5.PMID:12597876.
26.Vagner V,Ehrlich SD.Efficiency of homologous DNA recombination varies along the Bacillus subtilis chromosome.J Bacteriol.1988;170(9):3978−82.PMID:3137211.
27.Saleh−Gohari N,Helleday T.Conservative homologous recombination preferentially repairs DNA double−strand breaks in the S phase of the cell cycle in human cells.Nucleic Acids Res.2004;32(12):3683−8.PMID:15252152.
28.Lombardo A,Genovese P,Beausejour CM,Colleoni S,Lee YL,Kim KA,Ando D,Urnov FD,Galli C,Gregory PD,Holmes MC,Naldini L.Gene editing in human stem cells using zince finger nucleases and integrase−defective lentiviral vector delivery.Nat Biotechnol.2007;25(11):1298−306.PMID:17965707.
29.Conticello SG.The AID/APOBEC family of nucleic acid mutators.Genome Biol.2008;9(6):229.PMID:18598372.
30.Reynaud CA,Aoufouchi S,Faili A,Weill JC.What role for AID:mutator,or assembler of the immunoglobulin mutasome?Nat Immunol.2003;4(7):631−8.
31.Bhagwat AS.DNA−cytosine deaminases:from antibody maturation to antiviral defense.DNA Repair(Amst).2004;3(1):85−9.PMID:14697763.
32.Navaratnam N,Sarwar R.An overview of cytidine deaminases.Int J Hematol.2006;83(3):195−200.PMID:16720547.
33.Holden LG,Prochnow C,Chang YP,Bransteitter R,Chelico L,Sen U,Stevens RC,Goodman MF,Chen XS.Crystal structure of the anti−viral APOBEC3G catalytic domain and functional implications.Nature.2008;456(7218):121−4.PMID:18849968.
34.Chelico L,Pham P,Petruska J,Goodman MF.Biochemical basis of immunological and retroviral responses to DNA−targeted cytosine deamination by activation−induced cytidine deaminase and APOBEC3G.J Biol Chem.2009;284(41).27761−5.PMID:19684020.
35.Pham P,Bransteitter R,Goodman MF.Reward versus risk:DNA cytidine deaminases triggering immunity and disease.Biochemistry.2005;44(8):2703−15.PMID 15723516.
36.Barbas CF,Kim DH.Cytidine deaminase fusions and related methods.PCT Int Appl.2010;WO2010132092A2 20101118.
37.Chen X,Zaro JL,Shen WC.Fusion protein linkers:property,design and functionality.Adv Drug Deliv Rev.2013;65(10):1357−69.PMID:23026637.
38.Gerber AP,Keller W.RNA editing by base deamination:more enzymes,more targets,new mysteries.Trends Biochem Sci.2001;26(6):376−84.PMID:11406411.
39.Yuan L,Kurek I,English J,Keenan R.Laboratory−directed protein evolution.Microbiol Mol Biol Rev.2005;69(3):373−92.PMID:16148303.
40.Cobb RE,Sun N,Zhao H.Directed evolution as a powerful synthetic biology tool.Methods.2013;60(1):81−90.PMID:22465795.
41.Bershtein S,Tawfik DS.Advances in laboratory evolution of enzymes.Curr Opin Chem Biol.2008;12(2):151−8.PMID:18284924.
42.Hida K,Hanes J,Ostermeier M.Directed evolution for drug and nucleic acid delivery.Adv Drug Deliv Rev.2007;59(15):1562−78.PMID:17933418.
43.Esvelt KM,Carlson JC,Liu DR.A system for the continuous directed evolution of biomolecules.Nature.2011;472(7344):499−503.PMID:21478873.
44.Husimi Y.Selection and evolution of bacteriophages in cellstat.Adv Biophys.1989;25:1−43.PMID:2696338.
45.Riechmann L,Holliger P.The C−terminal domain of TolA is the coreceptor for filamentous phage infection of E.coli.Cell.1997;90(2):351−60.PMID:9244308.
46.Nelson FK,Friedman SM,Smith GP.Filamentous phage DNA cloning vectors:a noninfective mutant with a nonpolar deletion in gene III.Virology.1981;108(2):338−50.PMID:6258292.
47.Rakonjac J,Model P.Roles of pIII in filamentous phage assembly.J Mol Biol.1998;282(1):25−41.
48.Smith GP.Filamentous fusion phage:novel expression vectors that display cloned antigens on the virion surface.Science.1985;228(4705):1315−7.PMID:4001944.
49.Sheridan C.Gene therapy finds its niche.Nat Biotechnol.2011;29(2):121−8.PMID:21301435.
50.Lee JW,Soung YH,Kim SY,Lee HW,Park WS,Nam SW,Kim SH,Lee JY,Yoo NJ,Lee SH.PIK3CA gene is frequently mutated in breast carcinomas and hepatocellular carcinomas.Oncogene.2005;24(8):1477−80.PMID:15608678.
51.Ikediobi ON,Davies H,Bignell G,Edkins S,Stevens C,O’Meara S,Santarius T,Avis T,Barthorpe S,Brackenbury L,Buck G,Butler A,Clements J,Cole J,Dicks E,Forbes S,Gray K,Halliday K,Harrison R,Hills K,Hinton J,Hunter C,Jenkinson A,Jones D,Kosmidou V,Lugg R,Menzies A,Mironenko T,Parker A,Perry J,Raine K,Richardson D,Shepherd R,Small A,Smith R,Solomon H,Stephens P,Teaque J,Tofts C,Varian J,Webb T,West S,Widaa S,Yates A,Reinhold W,Weinstein JN,Stratton MR,Futreal PA,Wooster R.Mutation analysis of 24 known cancer genes in the NCI−60 cell line set.Mol Cancer Ther.2006;5(11):2606−12.PMID:17088437.
当業者は、本明細書に記載した実施形態の多くの均等物を認識し得るか、または通常の実験のみを使用して確認することができる。本開示の範囲は上記の説明に限定されることを意図されておらず、特許請求の範囲で規定されている通りである。
Claims (62)
- (i)ヌクレアーゼ不活性型Cas9ドメインと、
(ii)核酸編集ドメインと
を含む融合タンパク質。 - 前記核酸編集ドメインがDNA編集ドメインである、請求項1に記載の融合タンパク質。
- 前記核酸編集ドメインがデアミナーゼドメインである、請求項1に記載の融合タンパク質。
- デアミナーゼがシチジンデアミナーゼである、請求項1に記載の融合タンパク質。
- デアミナーゼがアポリポタンパク質B mRNA編集複合体(APOBEC)ファミリのデアミナーゼである、請求項1に記載の融合タンパク質。
- デアミナーゼがAPOBEC1ファミリのデアミナーゼである、請求項5に記載の融合タンパク質。
- デアミナーゼが活性化誘導シチジンデアミナーゼ(AID)である、請求項5に記載の融合タンパク質。
- デアミナーゼがACF1/ASEデアミナーゼである、請求項1に記載の融合タンパク質。
- デアミナーゼがアデノシンデアミナーゼである、請求項1に記載の融合タンパク質。
- デアミナーゼがADATファミリのデアミナーゼである、請求項9に記載の融合タンパク質。
- 前記核酸編集ドメインが前記Cas9ドメインのN末端に融合している、請求項1に記載の融合タンパク質。
- 前記核酸編集ドメインが前記Cas9ドメインのC末端に融合している、請求項1に記載の融合タンパク質。
- 前記Cas9ドメインおよび前記核酸編集ドメインがリンカーを介して融合している、請求項1に記載の融合タンパク質。
- リンカーが、(GGGGS)n(配列番号91)、(G)n、(EAAAK)n(配列番号5)、(GGS)n、SGSETPGTSESATPES(配列番号93)もしくは(XP)nモチーフまたはこれらのうちのいずれかの組み合わせを含み、nが独立して1〜30の整数である、請求項1に記載の融合タンパク質。
- DNA編集の方法であって、DNA分子と、
(a)ヌクレアーゼ不活性型Cas9ドメインおよび核酸編集ドメインを含む融合タンパク質、および
(b)前記(a)の融合タンパク質の標的を前記DNA分子の標的DNA配列に定めるsgRNA
とを接触させることを含み、
前記DNA分子が、前記DNA分子のヌクレオチド塩基の脱アミノ化に有効な量で、および前記脱アミノ化に適した条件下で前記融合タンパク質および前記sgRNAと接触させられる、方法。 - 前記核酸編集ドメインがデアミナーゼドメインである、請求項15に記載の方法。
- デアミナーゼがシチジンデアミナーゼである、請求項16に記載の方法。
- 前記デアミナーゼがアポリポタンパク質B mRNA編集複合体(APOBEC)ファミリのデアミナーゼである、請求項16または17に記載の方法。
- デアミナーゼがAPOBEC1ファミリのデアミナーゼである、請求項16に記載の方法。
- デアミナーゼが活性化誘導シチジンデアミナーゼ(AID)である、請求項16に記載の方法。
- デアミナーゼがACF1/ASEデアミナーゼである、請求項16に記載の方法。
- デアミナーゼがアデノシンデアミナーゼである、請求項16に記載の方法。
- デアミナーゼがADATファミリのデアミナーゼである、請求項16に記載の方法。
- 前記核酸編集ドメインが前記Cas9ドメインのN末端に融合している、請求項15〜23のいずれか一項に記載の方法。
- 前記核酸編集ドメインが前記Cas9ドメインのC末端に融合している、請求項15〜23のいずれか一項に記載の方法。
- 前記Cas9ドメインおよび前記核酸編集ドメインがリンカーを介して融合している、請求項15〜25のいずれか一項に記載の方法。
- 前記リンカーが、(GGGGS)n(配列番号91)、(G)n、(EAAAK)n(配列番号5)、(GGS)n、SGSETPGTSESATPES(配列番号93)もしくは(XP)nモチーフまたはこれらのうちのいずれかの組み合わせを含み、nが独立して1〜30の整数である、請求項26に記載の方法。
- 前記標的DNA配列が疾患または障害に関連する配列を含み、前記ヌクレオチド塩基の前記脱アミノ化により、疾患または障害に関連しない配列が生じる、請求項15〜27のいずれか一項に記載の方法。
- 前記標的DNA配列が疾患または障害に関連する点変異を含み、前記脱アミノ化により前記点変異が修正される、請求項28に記載の方法。
- 前記標的DNA配列が、疾患または障害に関連するT→CまたはA→G点変異を含み、変異CまたはG塩基の脱アミノ化により、疾患または障害に関連しない配列が生じる、請求項15〜28のいずれか一項に記載の方法。
- 前記疾患または障害に関連する前記配列がタンパク質をコードし、前記脱アミノ化により、前記疾患または障害に関連する前記配列に終止コドンが導入され、その結果、前記コードされたタンパク質が短縮される、請求項28〜30のいずれか一項に記載の方法。
- 前記接触させることが、疾患もしくは障害を有するか、または疾患もしくは障害と診断されている対象中におけるインビボである、請求項28〜31のいずれか一項に記載の方法。
- 前記疾患または障害が、嚢胞性線維症、フェニルケトン尿症、表皮剥離性角化症(EHK)、シャルコー・マリー・トゥース病4J型、神経芽細胞腫(NB)、フォン・ウィルブランド病(vWD)、先天性筋強直症、遺伝性腎アミロイドーシス、拡張型心筋症(DCM)、遺伝性リンパ水腫、家族性アルツハイマー病、プリオン病、慢性乳児神経皮膚関節症候群(CINCA)、デスミン関連心筋症(DRM)または変異PI3KCAタンパク質に関連する腫瘍性疾患である、請求項28〜32のいずれか一項に記載の方法。
- 前記標的DNA配列が、野生型PI3Kタンパク質と比較してPI3KCAタンパク質においてアミノ酸配列変異が生じるT→Cおよび/またはA→G点変異を含み、前記方法により変異CまたはG塩基が脱アミノ化される、請求項15〜33のいずれか一項に記載の方法。
- 前記点変異が、H1047R置換が生じるA3140G変異である、請求項34に記載の方法。
- 前記標的DNA配列が、野生型PSEN1タンパク質と比較してPSEN1タンパク質においてアミノ酸配列変異が生じるT→Cおよび/またはA→G点変異を含み、前記方法により変異CまたはG塩基が脱アミノ化される、請求項15〜33のいずれか一項に記載の方法。
- 前記PSEN1タンパク質がPSEN1遺伝子のコドン143におけるA→G点変異に起因するI143V置換を含む、請求項36に記載の方法。
- 前記PSEN1点変異がアルツハイマー病に関連している、請求項36または37に記載の方法。
- 前記接触させることにより、前記PSEN1遺伝子のコドン143における変異シチジン残基が脱アミノ化され、そのため前記A→G点変異が修正される、請求項36〜38のいずれか一項に記載の方法。
- 前記標的DNA配列が、野生型α−抗トリプシンタンパク質と比較してα−抗トリプシンタンパク質においてアミノ酸配列変異が生じるT→Cおよび/またはA→G点変異を含み、前記方法により変異CまたはG塩基が脱アミノ化される、請求項15〜33のいずれか一項に記載の方法。
- 前記α−抗トリプシンタンパク質がα−抗トリプシン遺伝子のコドン55におけるT→C点変異に起因するL55P置換を含む、請求項40に記載の方法。
- 前記α−抗トリプシン点変異が慢性閉塞性肺疾患(COPD)に関連する、請求項40または41に記載の方法。
- 前記接触させることにより、前記α−抗トリプシン遺伝子のコドン55における変異シチジン残基が脱アミノ化され、そのため前記T→C点変異が修正される、請求項40〜42のいずれか一項に記載の方法。
- 前記標的DNA配列が、野生型vWFタンパク質と比較してvWFタンパク質においてアミノ酸配列変異が生じるT→Cおよび/またはA→G点変異を含み、前記方法により変異CまたはG塩基が脱アミノ化される、請求項15〜33のいずれか一項に記載の方法。
- 前記vWFタンパク質がvWF遺伝子のコドン509におけるT→C点変異に起因するC509A置換を含む、請求項44に記載の方法。
- 前記vWF点変異がフォン・ウィルブランド病に関連する、請求項45に記載の方法。
- 前記接触させることにより、前記vWF遺伝子のコドン509における変異シチジン残基が脱アミノ化され、そのため前記T→C点変異が補正される、請求項44または45に記載の方法。
- 前記標的DNA配列が、野生型カスパーゼ−9タンパク質と比較してカスパーゼ−9タンパク質においてアミノ酸配列変異が生じるT→Cおよび/またはA→G点変異を含み、前記方法により変異CまたはG塩基が脱アミノ化される、請求項15〜33のいずれか一項に記載の方法。
- 前記カスパーゼ−9タンパク質がカスパーゼ−9遺伝子のコドン197におけるT→C点変異に起因するL197P置換を含む、請求項48に記載の方法。
- 前記カスパーゼ−9点変異が神経芽細胞腫に関連する、請求項48または49に記載の方法。
- 前記接触させることにより、前記カスパーゼ−9遺伝子のコドン197における変異シチジン残基が脱アミノ化され、そのため前記T→C点変異が修正される、請求項48〜50のいずれか一項に記載の方法。
- 前記ヌクレオチド塩基の前記脱アミノ化を検出することを更に含む、請求項15〜51のいずれか一項に記載の方法。
- 前記検出することがPCRによる、請求項52に記載の方法。
- 前記融合タンパク質が請求項1〜14のいずれか一項に記載の融合タンパク質である、請求項15〜53のいずれか一項に記載の方法。
- Cas9 DNA編集タンパク質のDNA編集活性を検出するためのレポーター構築物であって、
(a)Cas9 DNA編集タンパク質用の標的部位を含むレポーター遺伝子であって、標的を定めたDNA編集により前記レポーター遺伝子の発現が増加する、レポーター遺伝子と、
(b)前記レポーター遺伝子の発現を制御するプロモーター配列と
を含むレポーター構築物。 - 前記構築物が、
(c)前記Cas9 DNA編集タンパク質の標的を前記レポーター遺伝子の標的部位に定めるsgRNAをコードする配列であって、前記sgRNAの発現が前記レポーター遺伝子の前記発現から独立している、配列
を更に含む、請求項55に記載のレポーター構築物。 - 前記レポーター遺伝子の前記標的部位が未成熟終止コドンを含み、前記Cas9 DNA編集タンパク質による鋳型鎖の標的を定めたDNA編集により、前記未成熟終止コドンがアミノ酸残基をコードするコドンに変換される、請求項55または56に記載のレポーター構築物。
- 前記レポーター遺伝子が、ルシフェラーゼ、蛍光タンパク質または抗生物質耐性マーカーをコードする、請求項55〜57のいずれか一項に記載のレポーター構築物。
- 核酸構築物であって、
ヌクレアーゼ不活性型Cas9配列をコードする配列、
前記Cas9をコードする配列と核酸編集酵素または核酸編集酵素ドメインをコードする配列とのインフレームでのクローニングを可能にするように位置するクローニング部位を含む配列、
任意選択的に、前記Cas9をコードする配列と前記クローニング部位との間に位置するリンカーをコードする配列
を含む核酸と、
Cas9核酸編集融合タンパク質を生成するための、核酸編集酵素または核酸編集酵素ドメインをコードする配列の前記核酸構築物中へのインフレームでのクローニングに好適な試薬、緩衝液および/または使用説明書と
を含むキット。 - 前記クローニング部位を含む前記配列が前記Cas9配列のN末端である、請求項59に記載のキット。
- 前記クローニング部位を含む前記配列が前記Cas9配列のC末端である、請求項59に記載のキット。
- 前記コードされたリンカーが、(GGGGS)n(配列番号91)、(G)n、(EAAAK)n(配列番号5)、(GGS)n、SGSETPGTSESATPES(配列番号93)もしくは(XP)nモチーフまたはこれらのうちのいずれかの組み合わせを含み、nが独立して1〜30の整数である、請求項59〜62のいずれか一項に記載のキット。
Priority Applications (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
JP2021188589A JP2022043042A (ja) | 2013-12-12 | 2021-11-19 | 遺伝子編集用のcas多様体 |
JP2024000252A JP2024061716A (ja) | 2013-12-12 | 2024-01-04 | 遺伝子編集用のcas多様体 |
Applications Claiming Priority (18)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201361915386P | 2013-12-12 | 2013-12-12 | |
US61/915,386 | 2013-12-12 | ||
US201461980333P | 2014-04-16 | 2014-04-16 | |
US61/980,333 | 2014-04-16 | ||
US14/326,290 | 2014-07-08 | ||
US14/325,815 US11053481B2 (en) | 2013-12-12 | 2014-07-08 | Fusions of Cas9 domains and nucleic acid-editing domains |
US14/326,269 US9068179B1 (en) | 2013-12-12 | 2014-07-08 | Methods for correcting presenilin point mutations |
US14/326,109 | 2014-07-08 | ||
US14/326,303 | 2014-07-08 | ||
US14/326,109 US9840699B2 (en) | 2013-12-12 | 2014-07-08 | Methods for nucleic acid editing |
US14/325,815 | 2014-07-08 | ||
US14/326,303 US20150165054A1 (en) | 2013-12-12 | 2014-07-08 | Methods for correcting caspase-9 point mutations |
US14/326,140 US20150166982A1 (en) | 2013-12-12 | 2014-07-08 | Methods for correcting pi3k point mutations |
US14/326,318 US20150166985A1 (en) | 2013-12-12 | 2014-07-08 | Methods for correcting von willebrand factor point mutations |
US14/326,269 | 2014-07-08 | ||
US14/326,140 | 2014-07-08 | ||
US14/326,290 US20150166984A1 (en) | 2013-12-12 | 2014-07-08 | Methods for correcting alpha-antitrypsin point mutations |
US14/326,318 | 2014-07-08 |
Related Parent Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP2016539218A Division JP2017500035A (ja) | 2013-12-12 | 2014-12-12 | 遺伝子編集用のcas多様体 |
Related Child Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP2021188589A Division JP2022043042A (ja) | 2013-12-12 | 2021-11-19 | 遺伝子編集用のcas多様体 |
Publications (1)
Publication Number | Publication Date |
---|---|
JP2020164529A true JP2020164529A (ja) | 2020-10-08 |
Family
ID=53367126
Family Applications (4)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP2016539218A Pending JP2017500035A (ja) | 2013-12-12 | 2014-12-12 | 遺伝子編集用のcas多様体 |
JP2020082201A Pending JP2020164529A (ja) | 2013-12-12 | 2020-05-07 | 遺伝子編集用のcas多様体 |
JP2021188589A Pending JP2022043042A (ja) | 2013-12-12 | 2021-11-19 | 遺伝子編集用のcas多様体 |
JP2024000252A Pending JP2024061716A (ja) | 2013-12-12 | 2024-01-04 | 遺伝子編集用のcas多様体 |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP2016539218A Pending JP2017500035A (ja) | 2013-12-12 | 2014-12-12 | 遺伝子編集用のcas多様体 |
Family Applications After (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP2021188589A Pending JP2022043042A (ja) | 2013-12-12 | 2021-11-19 | 遺伝子編集用のcas多様体 |
JP2024000252A Pending JP2024061716A (ja) | 2013-12-12 | 2024-01-04 | 遺伝子編集用のcas多様体 |
Country Status (12)
Country | Link |
---|---|
US (10) | US9840699B2 (ja) |
EP (3) | EP3604511B1 (ja) |
JP (4) | JP2017500035A (ja) |
CN (2) | CN114516920A (ja) |
AU (3) | AU2014362208B2 (ja) |
CA (1) | CA2933625C (ja) |
DK (1) | DK3080265T3 (ja) |
ES (1) | ES2754433T3 (ja) |
HU (1) | HUE046398T2 (ja) |
PL (1) | PL3080265T3 (ja) |
PT (1) | PT3080265T (ja) |
WO (1) | WO2015089406A1 (ja) |
Families Citing this family (279)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
ES2960803T3 (es) | 2012-05-25 | 2024-03-06 | Univ California | Métodos y composiciones para la modificación de ADN diana dirigida por RNA y para la modulación de la transcripción dirigida por RNA |
US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
AU2014308899B2 (en) | 2013-08-22 | 2020-11-19 | E. I. Du Pont De Nemours And Company | Methods for producing genetic modifications in a plant genome without incorporating a selectable transgene marker, and compositions thereof |
US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
US10584358B2 (en) | 2013-10-30 | 2020-03-10 | North Carolina State University | Compositions and methods related to a type-II CRISPR-Cas system in Lactobacillus buchneri |
WO2015070083A1 (en) | 2013-11-07 | 2015-05-14 | Editas Medicine,Inc. | CRISPR-RELATED METHODS AND COMPOSITIONS WITH GOVERNING gRNAS |
US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
US10787654B2 (en) | 2014-01-24 | 2020-09-29 | North Carolina State University | Methods and compositions for sequence guiding Cas9 targeting |
DK3102722T3 (da) | 2014-02-04 | 2020-11-16 | Jumpcode Genomics Inc | Genom fraktionering |
CN111471675A (zh) * | 2014-03-05 | 2020-07-31 | 国立大学法人神户大学 | 特异性转变靶向dna序列的核酸碱基的基因组序列的修饰方法、及其使用的分子复合体 |
EP3114227B1 (en) | 2014-03-05 | 2021-07-21 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
WO2015138510A1 (en) | 2014-03-10 | 2015-09-17 | Editas Medicine., Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
WO2015148863A2 (en) | 2014-03-26 | 2015-10-01 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
US11439712B2 (en) | 2014-04-08 | 2022-09-13 | North Carolina State University | Methods and compositions for RNA-directed repression of transcription using CRISPR-associated genes |
EP3140403A4 (en) | 2014-05-09 | 2017-12-20 | Université Laval | Prevention and treatment of alzheimer's disease by genome editing using the crispr/cas system |
EP3177718B1 (en) | 2014-07-30 | 2022-03-16 | President and Fellows of Harvard College | Cas9 proteins including ligand-dependent inteins |
CN113789317B (zh) * | 2014-08-06 | 2024-02-23 | 基因工具股份有限公司 | 使用空肠弯曲杆菌crispr/cas系统衍生的rna引导的工程化核酸酶的基因编辑 |
US10450584B2 (en) | 2014-08-28 | 2019-10-22 | North Carolina State University | Cas9 proteins and guiding features for DNA targeting and genome editing |
US10570418B2 (en) * | 2014-09-02 | 2020-02-25 | The Regents Of The University Of California | Methods and compositions for RNA-directed target DNA modification |
WO2016037163A1 (en) | 2014-09-07 | 2016-03-10 | Selecta Biosciences, Inc. | Methods and compositions for attenuating gene therapy anti-viral transfer vector immune responses |
US10920208B2 (en) | 2014-10-22 | 2021-02-16 | President And Fellows Of Harvard College | Evolution of proteases |
EP3739047A1 (en) * | 2014-11-04 | 2020-11-18 | National University Corporation Kobe University | Method for modifying genome sequence to introduce specific mutation to targeted dna sequence by base-removal reaction, and molecular complex used therein |
CN107250148B (zh) | 2014-12-03 | 2021-04-16 | 安捷伦科技有限公司 | 具有化学修饰的指导rna |
WO2016094679A1 (en) | 2014-12-10 | 2016-06-16 | Regents Of The University Of Minnesota | Genetically modified cells, tissues, and organs for treating disease |
US20210395729A1 (en) * | 2014-12-12 | 2021-12-23 | Tod M. Woolf | Compositions and methods for Editing Nucleic Acids in Cells Utilizing Oligonucleotides |
WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
KR102648489B1 (ko) | 2015-04-06 | 2024-03-15 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna |
SG11201708653RA (en) | 2015-04-24 | 2017-11-29 | Editas Medicine Inc | Evaluation of cas9 molecule/guide rna molecule complexes |
KR102451796B1 (ko) | 2015-05-29 | 2022-10-06 | 노쓰 캐롤라이나 스테이트 유니버시티 | 크리스퍼 핵산을 사용하여 박테리아, 고세균, 조류 및 효모를 스크리닝하는 방법 |
CA2983874C (en) | 2015-06-15 | 2022-06-21 | North Carolina State University | Methods and compositions for efficient delivery of nucleic acids and rna-based antimicrobials |
WO2017004261A1 (en) | 2015-06-29 | 2017-01-05 | Ionis Pharmaceuticals, Inc. | Modified crispr rna and modified single crispr rna and uses thereof |
CN108291218B (zh) | 2015-07-15 | 2022-08-19 | 新泽西鲁特格斯州立大学 | 核酸酶非依赖性靶向基因编辑平台及其用途 |
US10676735B2 (en) | 2015-07-22 | 2020-06-09 | Duke University | High-throughput screening of regulatory element function with epigenome editing technologies |
US10166255B2 (en) | 2015-07-31 | 2019-01-01 | Regents Of The University Of Minnesota | Intracellular genomic transplant and methods of therapy |
WO2017024047A1 (en) * | 2015-08-03 | 2017-02-09 | Emendobio Inc. | Compositions and methods for increasing nuclease induced recombination rate in cells |
CN107922953B (zh) * | 2015-08-20 | 2022-03-04 | 应用干细胞有限公司 | 提高基因编辑效率的核酸酶 |
ES2929110T3 (es) | 2015-08-25 | 2022-11-24 | Univ Duke | Composiciones y métodos para mejorar la especificidad en ingeniería genética usando endonucleasas guiadas por ARN |
ES2938623T3 (es) * | 2015-09-09 | 2023-04-13 | Univ Kobe Nat Univ Corp | Método para convertir una secuencia del genoma de una bacteria gram-positiva mediante una conversión específica de una base de ácido nucleico de una secuencia de ADN seleccionada como diana y el complejo molecular utilizado en el mismo |
CA2998087A1 (en) * | 2015-09-09 | 2017-03-16 | National University Corporation Kobe University | Method for modifying genome sequence that specifically converts nucleobase of targeted dna sequence, and molecular complex used in said method |
JP6940262B2 (ja) * | 2015-09-09 | 2021-09-22 | 株式会社日本触媒 | ゲノム配列が特異的に変換された遺伝子改変クロストリジウム・サッカロパーブチルアセトニカム種微生物、その製造方法およびその用途 |
US11286480B2 (en) | 2015-09-28 | 2022-03-29 | North Carolina State University | Methods and compositions for sequence specific antimicrobials |
EP4089175A1 (en) | 2015-10-13 | 2022-11-16 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
GB2559922A (en) * | 2015-10-23 | 2018-08-22 | Harvard College | Nucleobase editors and uses thereof |
EP3365356B1 (en) * | 2015-10-23 | 2023-06-28 | President and Fellows of Harvard College | Nucleobase editors and uses thereof |
WO2017091630A1 (en) | 2015-11-23 | 2017-06-01 | The Regents Of The University Of California | Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9 |
EP3382019B1 (en) * | 2015-11-27 | 2022-05-04 | National University Corporation Kobe University | Method for converting monocot plant genome sequence in which nucleic acid base in targeted dna sequence is specifically converted, and molecular complex used therein |
EP3967758A1 (en) * | 2015-12-01 | 2022-03-16 | CRISPR Therapeutics AG | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
WO2017112620A1 (en) | 2015-12-22 | 2017-06-29 | North Carolina State University | Methods and compositions for delivery of crispr based antimicrobials |
US11339427B2 (en) | 2016-02-12 | 2022-05-24 | Jumpcode Genomics, Inc. | Method for target specific RNA transcription of DNA sequences |
WO2017165862A1 (en) | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
KR102617874B1 (ko) | 2016-03-30 | 2023-12-22 | 인텔리아 테라퓨틱스, 인크. | Crispr/cas 성분을 위한 지질 나노입자 제제 |
US20190127713A1 (en) * | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
US20190300872A1 (en) * | 2016-05-06 | 2019-10-03 | Tod M. Woolf | Improved Methods of Genome Editing with and without Programmable Nucleases |
US10767175B2 (en) | 2016-06-08 | 2020-09-08 | Agilent Technologies, Inc. | High specificity genome editing using chemically modified guide RNAs |
CN114380922A (zh) * | 2016-06-15 | 2022-04-22 | 中国科学院上海营养与健康研究所 | 在细胞内产生点突变的融合蛋白、其制备及用途 |
AU2017281497B2 (en) * | 2016-06-22 | 2023-04-06 | Proqr Therapeutics Ii B.V. | Single-stranded RNA-editing oligonucleotides |
US11471462B2 (en) | 2016-06-27 | 2022-10-18 | The Broad Institute, Inc. | Compositions and methods for detecting and treating diabetes |
CN109477086A (zh) * | 2016-07-13 | 2019-03-15 | 陈奇涵 | 一种基因组dna特异性编辑方法和应用 |
US11566263B2 (en) | 2016-08-02 | 2023-01-31 | Editas Medicine, Inc. | Compositions and methods for treating CEP290 associated disease |
GB2568182A (en) | 2016-08-03 | 2019-05-08 | Harvard College | Adenosine nucleobase editors and uses thereof |
AU2017308889B2 (en) | 2016-08-09 | 2023-11-09 | President And Fellows Of Harvard College | Programmable Cas9-recombinase fusion proteins and uses thereof |
US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
KR102026421B1 (ko) | 2016-09-13 | 2019-09-27 | 주식회사 툴젠 | 시토신 디아미나제에 의한 dna에서의 염기 교정 확인 방법 |
US20190225974A1 (en) | 2016-09-23 | 2019-07-25 | BASF Agricultural Solutions Seed US LLC | Targeted genome optimization in plants |
KR102622411B1 (ko) | 2016-10-14 | 2024-01-10 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
CN110520530A (zh) | 2016-10-18 | 2019-11-29 | 明尼苏达大学董事会 | 肿瘤浸润性淋巴细胞和治疗方法 |
WO2018083606A1 (en) | 2016-11-01 | 2018-05-11 | Novartis Ag | Methods and compositions for enhancing gene editing |
JP2019523011A (ja) * | 2016-11-14 | 2019-08-22 | インスティテュート・オブ・ジェネティクス・アンド・ディヴェロプメンタル・バイオロジー、チャイニーズ・アカデミー・オブ・サイエンシズInstitute of Genetics and Developmental Biology, Chinese Academy of Sciences | 植物における塩基編集のための方法 |
CN107043779B (zh) * | 2016-12-01 | 2020-05-12 | 中国农业科学院作物科学研究所 | 一种CRISPR/nCas9介导的定点碱基替换在植物中的应用 |
CN106609282A (zh) * | 2016-12-02 | 2017-05-03 | 中国科学院上海生命科学研究院 | 一种用于植物基因组定点碱基替换的载体 |
US11192929B2 (en) | 2016-12-08 | 2021-12-07 | Regents Of The University Of Minnesota | Site-specific DNA base editing using modified APOBEC enzymes |
CN110582302A (zh) | 2016-12-14 | 2019-12-17 | 利甘达尔股份有限公司 | 用于核酸和/或蛋白有效负载递送的组合物和方法 |
NZ754739A (en) * | 2016-12-22 | 2024-01-26 | Intellia Therapeutics Inc | Compositions and methods for treating alpha-1 antitrypsin deficiency |
WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
KR102151065B1 (ko) * | 2016-12-23 | 2020-09-02 | 기초과학연구원 | 동물 배아의 염기 교정용 조성물 및 염기 교정 방법 |
GB2605925B (en) * | 2016-12-23 | 2023-02-22 | Harvard College | Gene editing of PCSK9 |
CN110520163A (zh) * | 2017-01-05 | 2019-11-29 | 新泽西鲁特格斯州立大学 | 独立于dna双链断裂的靶向基因编辑平台及其用途 |
US20190352634A1 (en) * | 2017-01-11 | 2019-11-21 | Oxford University Innovation Limited | Crispr rna |
WO2018135838A2 (ko) * | 2017-01-17 | 2018-07-26 | 기초과학연구원 | Dna 단일가닥 절단에 의한 염기 교정 비표적 위치 확인 방법 |
US10995333B2 (en) | 2017-02-06 | 2021-05-04 | 10X Genomics, Inc. | Systems and methods for nucleic acid preparation |
WO2018145041A1 (en) * | 2017-02-06 | 2018-08-09 | 10X Genomics, Inc. | Systems and methods for nucleic acid preparation |
TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
WO2018149915A1 (en) * | 2017-02-15 | 2018-08-23 | Keygene N.V. | Methods of targeted genetic alteration in plant cells |
CA3053861A1 (en) * | 2017-02-20 | 2018-08-23 | Institute Of Genetics And Developmental Biology, Chinese Academy Of Sciences | Genome editing method |
US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
CN110662556A (zh) * | 2017-03-09 | 2020-01-07 | 哈佛大学的校长及成员们 | 癌症疫苗 |
WO2018165629A1 (en) * | 2017-03-10 | 2018-09-13 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
EP3596217A1 (en) | 2017-03-14 | 2020-01-22 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
EP3601562A1 (en) | 2017-03-23 | 2020-02-05 | President and Fellows of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
CN110869498A (zh) | 2017-05-10 | 2020-03-06 | 加利福尼亚大学董事会 | 经由核递送crispr/cas9导向编辑细胞rna |
EP3622070A2 (en) | 2017-05-10 | 2020-03-18 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
WO2018213726A1 (en) * | 2017-05-18 | 2018-11-22 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
AU2018270088B2 (en) * | 2017-05-18 | 2024-05-16 | Massachusetts Institute Of Technology | Systems, methods, and compositions for targeted nucleic acid editing |
CN107177625B (zh) * | 2017-05-26 | 2021-05-25 | 中国农业科学院植物保护研究所 | 一种定点突变的人工载体系统及定点突变方法 |
CN111328290A (zh) * | 2017-06-26 | 2020-06-23 | 博德研究所 | 用于靶向核酸编辑的基于crispr/cas-腺嘌呤脱氨酶的组合物、系统和方法 |
EP3645721A1 (en) | 2017-06-30 | 2020-05-06 | Novartis AG | Methods for the treatment of disease with gene editing systems |
WO2019006418A2 (en) | 2017-06-30 | 2019-01-03 | Intima Bioscience, Inc. | ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY |
WO2019020007A1 (zh) * | 2017-07-25 | 2019-01-31 | 中国科学院上海生命科学研究院 | 通过诱导剪接位点碱基突变或多聚嘧啶区碱基置换调控rna剪接的方法 |
US11732274B2 (en) | 2017-07-28 | 2023-08-22 | President And Fellows Of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE) |
MX2020001178A (es) | 2017-07-31 | 2020-09-25 | Regeneron Pharma | Celulas madre embrionarias de raton transgenico con cas y ratones y usos de los mismos. |
SG11201912236YA (en) | 2017-07-31 | 2020-01-30 | Regeneron Pharma | Crispr reporter non-human animals and uses thereof |
RU2020111575A (ru) | 2017-08-22 | 2021-09-23 | Напиджен, Инк. | Модификация генома органелл с использованием направляемой полинуклеотидом эндонуклеазы |
EP3676376A2 (en) | 2017-08-30 | 2020-07-08 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
WO2019041296A1 (zh) * | 2017-09-01 | 2019-03-07 | 上海科技大学 | 一种碱基编辑系统及方法 |
WO2019051097A1 (en) * | 2017-09-08 | 2019-03-14 | The Regents Of The University Of California | RNA-GUIDED ENDONUCLEASE FUSION POLYPEPTIDES AND METHODS OF USING SAME |
CN107557373A (zh) * | 2017-09-19 | 2018-01-09 | 安徽大学 | 一种基于I‑B型CRISPR‑Cas系统基因cas3的基因编辑方法 |
IL311278A (en) | 2017-09-29 | 2024-05-01 | Intellia Therapeutics Inc | Polynucleotides, preparations, and methods for genome editing |
KR20200074134A (ko) | 2017-09-29 | 2020-06-24 | 인텔리아 테라퓨틱스, 인크. | 지질 나노파티클을 이용한 mRNA 전달의 체외 방법 |
MX2020003608A (es) | 2017-09-29 | 2020-09-25 | Intellia Therapeutics Inc | Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr. |
MX2020007148A (es) | 2017-09-29 | 2020-10-08 | Intellia Therapeutics Inc | Formulaciones. |
WO2019071048A1 (en) * | 2017-10-04 | 2019-04-11 | The Broad Institute, Inc. | SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITION |
AU2018347583A1 (en) | 2017-10-13 | 2020-05-21 | Selecta Biosciences, Inc. | Methods and compositions for attenuating anti-viral transfer vector IgM responses |
KR20200121782A (ko) * | 2017-10-16 | 2020-10-26 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
AU2018357926B2 (en) | 2017-10-31 | 2023-04-06 | Limagrain Europe | Wheat comprising male fertility restorer alleles |
EP3704245A1 (en) | 2017-11-01 | 2020-09-09 | Novartis AG | Synthetic rnas and methods of use |
WO2019104094A2 (en) | 2017-11-21 | 2019-05-31 | The Regents Of The University Of California | Fusion proteins and methods for site-directed genome editing |
US20200354413A1 (en) | 2017-12-15 | 2020-11-12 | Dana-Farber Cancer Institute, Inc. | Stabilized peptide-mediated targeted protein degradation |
CN110157727A (zh) | 2017-12-21 | 2019-08-23 | 中国科学院遗传与发育生物学研究所 | 植物碱基编辑方法 |
WO2019126716A1 (en) * | 2017-12-22 | 2019-06-27 | The Broad Institute, Inc. | Cas12b systems, methods, and compositions for targeted rna base editing |
US20230193242A1 (en) * | 2017-12-22 | 2023-06-22 | The Broad Institute, Inc. | Cas12b systems, methods, and compositions for targeted dna base editing |
CN108165543B (zh) * | 2017-12-26 | 2021-01-22 | 山东省农业科学院生物技术研究中心 | 水稻腺苷脱氨酶OsAD1及其编码基因在叶绿体基因RNA编辑中的应用 |
EP3737690A1 (en) | 2018-01-12 | 2020-11-18 | Basf Se | Gene underlying the number of spikelets per spike qtl in wheat on chromosome 7a |
JP7075170B2 (ja) * | 2018-01-23 | 2022-05-25 | インスティチュート フォー ベーシック サイエンス | 延長された単一ガイドrna及びその用途 |
WO2019147073A1 (ko) * | 2018-01-25 | 2019-08-01 | 주식회사 툴젠 | 아데노신 디아미나아제를 이용한 염기 교정 확인 방법 |
US20190233816A1 (en) | 2018-01-26 | 2019-08-01 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
EP3749764A1 (en) | 2018-02-08 | 2020-12-16 | Zymergen, Inc. | Genome editing using crispr in corynebacterium |
CN109021111B (zh) * | 2018-02-23 | 2021-12-07 | 上海科技大学 | 一种基因碱基编辑器 |
US20220010293A1 (en) * | 2018-02-23 | 2022-01-13 | Pioneer Hi-Bred International, Inc. | Novel cas9 orthologs |
CN112384204B (zh) | 2018-02-26 | 2023-03-14 | 安托瑞斯公司 | 致耐受性脂质体及其使用方法 |
CN112272516B (zh) | 2018-04-06 | 2023-05-30 | 儿童医疗中心有限公司 | 用于体细胞重新编程和调整印记的组合物和方法 |
GB201805865D0 (en) | 2018-04-09 | 2018-05-23 | Innes John Centre | Genes |
WO2019217941A1 (en) * | 2018-05-11 | 2019-11-14 | Beam Therapeutics Inc. | Methods of suppressing pathogenic mutations using programmable base editor systems |
EP3793590A4 (en) * | 2018-05-17 | 2022-02-16 | Regents of the University of Minnesota | DRUG RESISTANT IMMUNE CELLS AND METHODS OF USE |
US20210198330A1 (en) | 2018-05-23 | 2021-07-01 | The Broad Institute, Inc. | Base editors and uses thereof |
CA3102840A1 (en) | 2018-06-05 | 2019-12-12 | Lifeedit, Inc. | Rna-guided nucleases and active fragments and variants thereof and methods of use |
WO2019234132A1 (en) | 2018-06-05 | 2019-12-12 | KWS SAAT SE & Co. KGaA | Base editing in polymerase theta deficient plants |
US11608506B2 (en) | 2018-06-26 | 2023-03-21 | Regents Of The University Of Minnesota | Delivery of developmental regulators to plants for the induction of meristematic tissue with genetic alterations |
KR20210053898A (ko) * | 2018-07-31 | 2021-05-12 | 더 브로드 인스티튜트, 인코퍼레이티드 | 신규 crispr 효소 및 시스템 |
AU2019313348A1 (en) | 2018-07-31 | 2021-03-04 | Intellia Therapeutics, Inc. | Compositions and methods for hydroxyacid oxidase 1 (HAO1) gene editing for treating primary hyperoxaluria type 1 (PH1) |
EP3841203A4 (en) * | 2018-08-23 | 2022-11-02 | The Broad Institute Inc. | CAS9 VARIANTS WITH NON-CANONICAL PAM SPECIFICITIES AND USES OF THEM |
US20210332344A1 (en) * | 2018-08-31 | 2021-10-28 | The Regents Of The University Of California | Directed modification of rna |
WO2020051360A1 (en) | 2018-09-05 | 2020-03-12 | The Broad Institute, Inc. | Base editing for treating hutchinson-gilford progeria syndrome |
EP3847267A1 (en) * | 2018-09-08 | 2021-07-14 | Blueallele, LLC | Methods and compositions for modifying the von willebrand factor gene |
AU2019347517A1 (en) | 2018-09-28 | 2021-05-06 | Intellia Therapeutics, Inc. | Compositions and methods for lactate dehydrogenase (LDHA) gene editing |
WO2020072248A1 (en) | 2018-10-01 | 2020-04-09 | North Carolina State University | Recombinant type i crispr-cas system |
BR112021007123A2 (pt) | 2018-10-15 | 2021-08-10 | University Of Massachusetts | edição de base de dna programável pelas proteínas de fusão nme2cas9-desaminase |
CA3116132A1 (en) | 2018-10-16 | 2020-04-23 | Intellia Therapeutics, Inc. | Compositions and methods for immunotherapy |
US20210340562A1 (en) * | 2018-10-19 | 2021-11-04 | Board Of Regents, The University Of Texas System | Engineered long interspersed element (line) transposons and methods of use |
US20220389395A1 (en) | 2018-10-29 | 2022-12-08 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
EP3956443A1 (en) | 2018-11-01 | 2022-02-23 | Institute Of Genetics And Developmental Biology, Chinese Academy Of Sciences | Targeted mutagenesis using base editors |
US20220282275A1 (en) | 2018-11-15 | 2022-09-08 | The Broad Institute, Inc. | G-to-t base editors and uses thereof |
US10934536B2 (en) | 2018-12-14 | 2021-03-02 | Pioneer Hi-Bred International, Inc. | CRISPR-CAS systems for genome editing |
US20220145296A1 (en) | 2018-12-27 | 2022-05-12 | LifeEDIT Therapeutics, Inc. | Polypeptides useful for gene editing and methods of use |
CA3125009A1 (en) * | 2019-01-07 | 2020-07-16 | Crisp-Hr Therapeutics, Inc. | A non-toxic cas9 enzyme and application thereof |
WO2020157164A1 (en) | 2019-01-30 | 2020-08-06 | Enobraq | Modified plant with improved rubisco activity |
EP3918077A4 (en) * | 2019-01-31 | 2023-03-29 | Beam Therapeutics, Inc. | NUCLEOBASE EDITORS WITH REDUCED OFF-TARGET DEAMINATION AND METHODS OF USING THEM TO MODIFY A NUCLEOBASE TARGET SEQUENCE |
KR20210121113A (ko) * | 2019-01-31 | 2021-10-07 | 빔 테라퓨틱스, 인크. | 비-표적 탈아미노화가 감소된 핵염기 편집기 및 핵염기 편집기를 특성규명하기 위한 분석 |
CN116355966A (zh) * | 2019-02-02 | 2023-06-30 | 上海科技大学 | 一种融合蛋白在遗传编辑的用途 |
US20220145330A1 (en) | 2019-02-10 | 2022-05-12 | The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glads | Modified mitochondrion and methods of use thereof |
WO2020168133A1 (en) | 2019-02-13 | 2020-08-20 | Beam Therapeutics Inc. | Compositions and methods for treating hemoglobinopathies |
WO2020168132A1 (en) * | 2019-02-13 | 2020-08-20 | Beam Therapeutics Inc. | Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence |
WO2020181178A1 (en) | 2019-03-06 | 2020-09-10 | The Broad Institute, Inc. | T:a to a:t base editing through thymine alkylation |
WO2020181195A1 (en) | 2019-03-06 | 2020-09-10 | The Broad Institute, Inc. | T:a to a:t base editing through adenine excision |
WO2020181202A1 (en) | 2019-03-06 | 2020-09-10 | The Broad Institute, Inc. | A:t to t:a base editing through adenine deamination and oxidation |
US20220170013A1 (en) | 2019-03-06 | 2022-06-02 | The Broad Institute, Inc. | T:a to a:t base editing through adenosine methylation |
WO2020181180A1 (en) | 2019-03-06 | 2020-09-10 | The Broad Institute, Inc. | A:t to c:g base editors and uses thereof |
WO2020209959A1 (en) | 2019-03-08 | 2020-10-15 | Crispr Therapeutics Ag | Nucleobase-editing fusion protein systems, compositions, and uses thereof |
BR112021018606A2 (pt) | 2019-03-19 | 2021-11-23 | Harvard College | Métodos e composições para editar sequências de nucleotídeos |
MA55527A (fr) | 2019-03-28 | 2022-02-09 | Intellia Therapeutics Inc | Polynucléotides, compositions et procédés d'expression de polypeptides |
CA3134544A1 (en) | 2019-03-28 | 2020-10-01 | Intellia Therapeutics, Inc. | Compositions and methods for ttr gene editing and treating attr amyloidosis comprising a corticosteroid or use thereof |
EP3946598A1 (en) | 2019-03-28 | 2022-02-09 | Intellia Therapeutics, Inc. | Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent |
EP3947663A4 (en) * | 2019-04-05 | 2023-01-11 | The Broad Institute, Inc. | PSEUDO RANDOM DNA EDITOR FOR EFFICIENT AND CONTINUOUS NUCLEOTIDE DIVERSIFICATION IN HUMAN CELLS |
CN113748205A (zh) | 2019-04-12 | 2021-12-03 | 阿斯利康(瑞典)有限公司 | 用于改进的基因编辑的组合物和方法 |
WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
EP3966334A1 (en) | 2019-05-10 | 2022-03-16 | Basf Se | Regulatory nucleic acid molecules for enhancing gene expression in plants |
EP3973054A1 (en) | 2019-05-20 | 2022-03-30 | The Broad Institute Inc. | Aav delivery of nucleobase editors |
WO2020243261A1 (en) | 2019-05-28 | 2020-12-03 | Selecta Biosciences, Inc. | Methods and compositions for attenuated anti-viral transfer vector immune response |
WO2021025750A1 (en) | 2019-08-08 | 2021-02-11 | The Broad Institute, Inc. | Base editors with diversified targeting scope |
WO2021030344A1 (en) | 2019-08-12 | 2021-02-18 | Lifeedit, Inc. | Rna-guided nucleases and active fragments and variants thereof and methods of use |
WO2021030666A1 (en) | 2019-08-15 | 2021-02-18 | The Broad Institute, Inc. | Base editing by transglycosylation |
EP4022057A1 (en) | 2019-08-27 | 2022-07-06 | Vertex Pharmaceuticals Incorporated | Compositions and methods for treatment of disorders associated with repetitive dna |
CA3150334A1 (en) | 2019-09-12 | 2021-03-18 | Frank Meulewaeter | REGULATORY NUCLEIC ACID MOLECULES TO ENHANCE GENE EXPRESSION IN PLANTS |
WO2021069387A1 (en) | 2019-10-07 | 2021-04-15 | Basf Se | Regulatory nucleic acid molecules for enhancing gene expression in plants |
WO2021108717A2 (en) | 2019-11-26 | 2021-06-03 | The Broad Institute, Inc | Systems and methods for evaluating cas9-independent off-target editing of nucleic acids |
CN115038789A (zh) | 2019-12-02 | 2022-09-09 | 塑造治疗公司 | 治疗性编辑 |
AU2020396138A1 (en) | 2019-12-03 | 2022-06-16 | Basf Se | Regulatory nucleic acid molecules for enhancing gene expression in plants |
CN112979823B (zh) * | 2019-12-18 | 2022-04-08 | 华东师范大学 | 一种用于治疗和/或预防β血红蛋白病的产品及融合蛋白 |
US11584781B2 (en) | 2019-12-30 | 2023-02-21 | Eligo Bioscience | Chimeric receptor binding proteins resistant to proteolytic degradation |
US11746352B2 (en) | 2019-12-30 | 2023-09-05 | Eligo Bioscience | Microbiome modulation of a host by delivery of DNA payloads with minimized spread |
AU2020417760A1 (en) | 2019-12-30 | 2022-08-04 | LifeEDIT Therapeutics, Inc. | RNA-guided nucleases and active fragments and variants thereof and methods of use |
EP4090760A4 (en) * | 2020-01-17 | 2024-01-24 | Jumpcode Genomics Inc | METHOD FOR SAMPLE NORMALIZATION |
US20230073514A1 (en) | 2020-01-21 | 2023-03-09 | Limagrain Europe | Wheat haploid inducer plant and uses |
WO2021155065A1 (en) | 2020-01-28 | 2021-08-05 | The Broad Institute, Inc. | Base editors, compositions, and methods for modifying the mitochondrial genome |
EP4100519A2 (en) | 2020-02-05 | 2022-12-14 | The Broad Institute, Inc. | Adenine base editors and uses thereof |
WO2021158999A1 (en) | 2020-02-05 | 2021-08-12 | The Broad Institute, Inc. | Gene editing methods for treating spinal muscular atrophy |
US20230123669A1 (en) | 2020-02-05 | 2023-04-20 | The Broad Institute, Inc. | Base editor predictive algorithm and method of use |
EP4110913A2 (en) * | 2020-02-28 | 2023-01-04 | University of Massachusetts | Oligonucleotides for prnp modulation |
EP4118206A1 (en) | 2020-03-11 | 2023-01-18 | The Broad Institute Inc. | Stat3-targeted base editor therapeutics for the treatment of melanoma and other cancers |
EP4118195A1 (en) * | 2020-03-12 | 2023-01-18 | Novozymes A/S | Crispr-aid using catalytically inactive rna-guided endonuclease |
EP4121522A1 (en) | 2020-03-19 | 2023-01-25 | Intellia Therapeutics, Inc. | Methods and compositions for directed genome editing |
US11617773B2 (en) | 2020-04-08 | 2023-04-04 | Eligo Bioscience | Elimination of colonic bacterial driving lethal inflammatory cardiomyopathy |
TW202208626A (zh) | 2020-04-24 | 2022-03-01 | 美商生命編輯公司 | Rna引導核酸酶及其活性片段與變體,以及使用方法 |
US20230159913A1 (en) | 2020-04-28 | 2023-05-25 | The Broad Institute, Inc. | Targeted base editing of the ush2a gene |
MX2022013403A (es) | 2020-04-28 | 2023-01-11 | Intellia Therapeutics Inc | Metodos de administracion de celulas in vitro. |
EP4146797A1 (en) | 2020-05-06 | 2023-03-15 | Orchard Therapeutics (Europe) Limited | Treatment for neurodegenerative diseases |
DE112021002672T5 (de) | 2020-05-08 | 2023-04-13 | President And Fellows Of Harvard College | Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz |
CA3173882A1 (en) | 2020-05-11 | 2021-11-18 | Alexandra Briner CRAWLEY | Rna-guided nucleic acid binding proteins and active fragments and variants thereof and methods of use |
WO2021231603A2 (en) * | 2020-05-12 | 2021-11-18 | City Of Hope | Compositions and methods for base specific mitochondrial gene editing |
WO2022056000A1 (en) | 2020-09-09 | 2022-03-17 | Vertex Pharmaceuticals Incorporated | Compositions and methods for treatment of duchenne muscular dystrophy |
WO2022098933A1 (en) | 2020-11-06 | 2022-05-12 | Vertex Pharmaceuticals Incorporated | Compositions and methods for treatment of dm1 with slucas9 and sacas9 |
AU2021396403A1 (en) | 2020-12-11 | 2023-06-29 | Intellia Therapeutics, Inc. | Compositions and methods for reducing mhc class ii in a cell |
CR20230305A (es) | 2020-12-11 | 2023-11-10 | Intellia Therapeutics Inc | Polinucleótidos, composiciones y métodos para la edición del genoma que implican desaminación |
KR20230124664A (ko) | 2020-12-23 | 2023-08-25 | 인텔리아 테라퓨틱스, 인크. | 세포에서 hla-a를 감소시키기 위한 조성물 및 방법 |
EP4267723A1 (en) | 2020-12-23 | 2023-11-01 | Intellia Therapeutics, Inc. | Compositions and methods for genetically modifying ciita in a cell |
WO2022144381A1 (en) | 2020-12-30 | 2022-07-07 | Eligo Bioscience | Microbiome modulation of a host by delivery of dna payloads with minimized spread |
CN114686454B (zh) * | 2020-12-31 | 2024-04-26 | 北京市农林科学院 | Pe-p3引导编辑系统及其在基因组碱基编辑中的应用 |
WO2022170194A2 (en) | 2021-02-08 | 2022-08-11 | Intellia Therapeutics, Inc. | Lymphocyte activation gene 3 (lag3) compositions and methods for immunotherapy |
JP2024505672A (ja) | 2021-02-08 | 2024-02-07 | インテリア セラピューティクス,インコーポレイテッド | 免疫療法のためのナチュラルキラー細胞受容体2b4組成物及び方法 |
WO2022170193A2 (en) | 2021-02-08 | 2022-08-11 | Intellia Therapeutics, Inc. | T-cell immunoglobulin and mucin domain 3 (tim3) compositions and methods for immunotherapy |
TW202246510A (zh) | 2021-02-26 | 2022-12-01 | 美商維泰克斯製藥公司 | 以crispr/slucas9治療第1型肌強直性營養不良之組合物及方法 |
TW202302848A (zh) | 2021-02-26 | 2023-01-16 | 美商維泰克斯製藥公司 | 以crispr/sacas9治療第1型肌強直性營養不良之組合物及方法 |
EP4323360A1 (en) | 2021-04-17 | 2024-02-21 | Intellia Therapeutics, Inc. | Inhibitors of dna-dependent protein kinase and compositions and uses thereof |
AU2022258732A1 (en) | 2021-04-17 | 2023-11-30 | Intellia Therapeutics, Inc. | Lipid nanoparticle compositions |
KR20240017792A (ko) | 2021-04-17 | 2024-02-08 | 인텔리아 테라퓨틱스, 인크. | 지질 나노입자 조성물 |
WO2022229851A1 (en) | 2021-04-26 | 2022-11-03 | Crispr Therapeutics Ag | Compositions and methods for using slucas9 scaffold sequences |
WO2022234519A1 (en) | 2021-05-05 | 2022-11-10 | Crispr Therapeutics Ag | Compositions and methods for using sacas9 scaffold sequences |
KR20240021768A (ko) | 2021-05-12 | 2024-02-19 | 엘리고 바이오사이언스 | 용해성 파지의 제조 |
AU2022290278A1 (en) | 2021-06-11 | 2024-01-04 | LifeEDIT Therapeutics, Inc. | Rna polymerase iii promoters and methods of use |
WO2022261509A1 (en) | 2021-06-11 | 2022-12-15 | The Broad Institute, Inc. | Improved cytosine to guanine base editors |
WO2023018637A1 (en) | 2021-08-09 | 2023-02-16 | Vertex Pharmaceuticals Incorporated | Gene editing of regulatory elements |
CN117940153A (zh) | 2021-08-24 | 2024-04-26 | 因特利亚治疗公司 | 用于基于细胞的疗法的程序性细胞死亡蛋白1(pd1)组合物和方法 |
WO2023039444A2 (en) | 2021-09-08 | 2023-03-16 | Vertex Pharmaceuticals Incorporated | Precise excisions of portions of exon 51 for treatment of duchenne muscular dystrophy |
KR20240055811A (ko) | 2021-09-10 | 2024-04-29 | 애질런트 테크놀로지스, 인크. | 프라임 편집을 위한 화학적 변형을 갖는 가이드 rna |
WO2023064367A1 (en) | 2021-10-12 | 2023-04-20 | Selecta Biosciences, Inc. | Methods and compositions for attenuating anti-viral transfer vector igm responses |
CA3237300A1 (en) | 2021-11-01 | 2023-05-04 | Tome Biosciences, Inc. | Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo |
WO2023081070A1 (en) | 2021-11-02 | 2023-05-11 | University Of Massachusetts | Nme2Cas9 INLAID DOMAIN FUSION PROTEINS |
AU2022382975A1 (en) | 2021-11-03 | 2024-05-02 | Intellia Therapeutics, Inc. | Polynucleotides, compositions, and methods for genome editing |
WO2023081200A2 (en) | 2021-11-03 | 2023-05-11 | Intellia Therapeutics, Inc. | Cd38 compositions and methods for immunotherapy |
WO2023086847A1 (en) | 2021-11-10 | 2023-05-19 | Encodia, Inc. | Methods for barcoding macromolecules in individual cells |
WO2023086973A1 (en) * | 2021-11-12 | 2023-05-19 | Arbor Biotechnologies, Inc. | Type ii nucleases |
WO2023102537A2 (en) | 2021-12-03 | 2023-06-08 | The Broad Institute, Inc. | Self-assembling virus-like particles for delivery of nucleic acid programmable fusion proteins and methods of making and using same |
WO2023107902A1 (en) | 2021-12-06 | 2023-06-15 | Napigen, Inc. | Phosphite dehydrogenase as a selectable marker for mitochondrial transformation |
WO2023121965A1 (en) | 2021-12-20 | 2023-06-29 | Beam Therapeutics Inc. | Nanomaterial comprising diamines |
WO2023121971A1 (en) | 2021-12-20 | 2023-06-29 | Beam Therapeutics Inc. | Nanomaterials comprising tetravalent lipid compounds |
WO2023121964A1 (en) | 2021-12-20 | 2023-06-29 | Beam Therapeutics Inc. | Nanomaterials comprising disulfides |
WO2023121975A1 (en) | 2021-12-20 | 2023-06-29 | Beam Therapeutics Inc. | Ionizable amine lipids and lipid nanoparticles |
WO2023121970A1 (en) | 2021-12-20 | 2023-06-29 | Beam Therapeutics Inc. | Ionizable amine and ester lipids and lipid nanoparticles |
WO2023122764A1 (en) | 2021-12-22 | 2023-06-29 | Tome Biosciences, Inc. | Co-delivery of a gene editor construct and a donor template |
WO2023139557A1 (en) | 2022-01-24 | 2023-07-27 | LifeEDIT Therapeutics, Inc. | Rna-guided nucleases and active fragments and variants thereof and methods of use |
WO2023155901A1 (en) * | 2022-02-17 | 2023-08-24 | Correctsequence Therapeutics | Mutant cytidine deaminases with improved editing precision |
WO2023172926A1 (en) | 2022-03-08 | 2023-09-14 | Vertex Pharmaceuticals Incorporated | Precise excisions of portions of exons for treatment of duchenne muscular dystrophy |
TW202345911A (zh) | 2022-03-08 | 2023-12-01 | 美商維泰克斯製藥公司 | 用於治療杜興氏肌肉失養症(duchenne muscular dystrophy)之部分外顯子44、50及53之精確切除 |
WO2023172624A1 (en) | 2022-03-09 | 2023-09-14 | Selecta Biosciences, Inc. | Immunosuppressants in combination with anti-igm agents and related dosing |
WO2023196802A1 (en) | 2022-04-04 | 2023-10-12 | The Broad Institute, Inc. | Cas9 variants having non-canonical pam specificities and uses thereof |
WO2023205744A1 (en) | 2022-04-20 | 2023-10-26 | Tome Biosciences, Inc. | Programmable gene insertion compositions |
WO2023212715A1 (en) | 2022-04-28 | 2023-11-02 | The Broad Institute, Inc. | Aav vectors encoding base editors and uses thereof |
WO2023225670A2 (en) | 2022-05-20 | 2023-11-23 | Tome Biosciences, Inc. | Ex vivo programmable gene insertion |
WO2023230613A1 (en) | 2022-05-27 | 2023-11-30 | The Broad Institute, Inc. | Improved mitochondrial base editors and methods for editing mitochondrial dna |
WO2023240137A1 (en) | 2022-06-08 | 2023-12-14 | The Board Institute, Inc. | Evolved cas14a1 variants, compositions, and methods of making and using same in genome editing |
WO2023245113A1 (en) | 2022-06-16 | 2023-12-21 | Intellia Therapeutics, Inc. | Methods and compositions for genetically modifying a cell |
WO2024020346A2 (en) | 2022-07-18 | 2024-01-25 | Renagade Therapeutics Management Inc. | Gene editing components, systems, and methods of use |
WO2024020352A1 (en) | 2022-07-18 | 2024-01-25 | Vertex Pharmaceuticals Incorporated | Tandem guide rnas (tg-rnas) and their use in genome editing |
WO2024019936A1 (en) | 2022-07-20 | 2024-01-25 | Beam Therapeutics Inc. | Nanomaterials comprising triols |
WO2024020587A2 (en) | 2022-07-22 | 2024-01-25 | Tome Biosciences, Inc. | Pleiopluripotent stem cell programmable gene insertion |
WO2024033901A1 (en) | 2022-08-12 | 2024-02-15 | LifeEDIT Therapeutics, Inc. | Rna-guided nucleases and active fragments and variants thereof and methods of use |
WO2024040083A1 (en) | 2022-08-16 | 2024-02-22 | The Broad Institute, Inc. | Evolved cytosine deaminases and methods of editing dna using same |
WO2024044723A1 (en) | 2022-08-25 | 2024-02-29 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
WO2024052681A1 (en) | 2022-09-08 | 2024-03-14 | The University Court Of The University Of Edinburgh | Rett syndrome therapy |
WO2024077247A1 (en) | 2022-10-07 | 2024-04-11 | The Broad Institute, Inc. | Base editing methods and compositions for treating triplet repeat disorders |
WO2024083579A1 (en) | 2022-10-20 | 2024-04-25 | Basf Se | Regulatory nucleic acid molecules for enhancing gene expression in plants |
WO2024102434A1 (en) | 2022-11-10 | 2024-05-16 | Senda Biosciences, Inc. | Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs |
CN117821462B (zh) * | 2024-03-04 | 2024-05-07 | 上海贝斯昂科生物科技有限公司 | 基因编辑修复阿尔兹海默症相关psen1位点突变 |
Citations (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2010132092A2 (en) * | 2009-05-12 | 2010-11-18 | The Scripps Research Institute | Cytidine deaminase fusions and related methods |
US20110104787A1 (en) * | 2009-11-05 | 2011-05-05 | President And Fellows Of Harvard College | Fusion Peptides That Bind to and Modify Target Nucleic Acid Sequences |
WO2013176772A1 (en) * | 2012-05-25 | 2013-11-28 | The Regents Of The University Of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
WO2014144288A1 (en) * | 2013-03-15 | 2014-09-18 | The General Hospital Corporation | Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing |
Family Cites Families (1585)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
US4182449A (en) | 1978-04-18 | 1980-01-08 | Kozlow William J | Adhesive bandage and package |
US4501728A (en) | 1983-01-06 | 1985-02-26 | Technology Unlimited, Inc. | Masking of liposomes from RES recognition |
US4880635B1 (en) | 1984-08-08 | 1996-07-02 | Liposome Company | Dehydrated liposomes |
US4921757A (en) | 1985-04-26 | 1990-05-01 | Massachusetts Institute Of Technology | System for delayed and pulsed release of biologically active substances |
US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
US4737323A (en) | 1986-02-13 | 1988-04-12 | Liposome Technology, Inc. | Liposome extrusion method |
US4920016A (en) | 1986-12-24 | 1990-04-24 | Linear Technology, Inc. | Liposomes with enhanced circulation time |
US4837028A (en) | 1986-12-24 | 1989-06-06 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
JPH0825869B2 (ja) | 1987-02-09 | 1996-03-13 | 株式会社ビタミン研究所 | 抗腫瘍剤包埋リポソ−ム製剤 |
US4917951A (en) | 1987-07-28 | 1990-04-17 | Micro-Pak, Inc. | Lipid vesicles formed of surfactants and steroids |
US4911928A (en) | 1987-03-13 | 1990-03-27 | Micro-Pak, Inc. | Paucilamellar lipid vesicles |
RO108522B1 (ro) | 1987-04-23 | 1994-06-30 | Fmc Corp | Derivati de di(aril)-cilopropil substituiti si intermediari ai acestora |
US5580737A (en) | 1990-06-11 | 1996-12-03 | Nexstar Pharmaceuticals, Inc. | High-affinity nucleic acid ligands that discriminate between theophylline and caffeine |
US6872816B1 (en) | 1996-01-24 | 2005-03-29 | Third Wave Technologies, Inc. | Nucleic acid detection kits |
JPH05274181A (ja) | 1992-03-25 | 1993-10-22 | Nec Corp | ブレークポイント設定・解除方式 |
US5651981A (en) | 1994-03-29 | 1997-07-29 | Northwestern University | Cationic phospholipids for transfection |
US5449639A (en) | 1994-10-24 | 1995-09-12 | Taiwan Semiconductor Manufacturing Company Ltd. | Disposable metal anti-reflection coating process used together with metal dry/wet etch |
US5767099A (en) | 1994-12-09 | 1998-06-16 | Genzyme Corporation | Cationic amphiphiles containing amino acid or dervatized amino acid groups for intracellular delivery of therapeutic molecules |
US6057153A (en) | 1995-01-13 | 2000-05-02 | Yale University | Stabilized external guide sequences |
US5795587A (en) | 1995-01-23 | 1998-08-18 | University Of Pittsburgh | Stable lipid-comprising drug delivery complexes and methods for their production |
US5830430A (en) | 1995-02-21 | 1998-11-03 | Imarx Pharmaceutical Corp. | Cationic lipids and the use thereof |
US5851548A (en) | 1995-06-07 | 1998-12-22 | Gen-Probe Incorporated | Liposomes containing cationic lipids and vitamin D |
US5962313A (en) | 1996-01-18 | 1999-10-05 | Avigen, Inc. | Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme |
US5981182A (en) | 1997-03-13 | 1999-11-09 | Albert Einstein College Of Medicine Of Yeshiva University | Vector constructs for the selection and identification of open reading frames |
US8097648B2 (en) | 1998-06-17 | 2012-01-17 | Eisai R&D Management Co., Ltd. | Methods and compositions for use in treating cancer |
EP2298728A1 (en) | 1998-11-12 | 2011-03-23 | Life Technologies Corporation | Transfection reagents |
US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
US7013219B2 (en) | 1999-01-12 | 2006-03-14 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
US20090130718A1 (en) | 1999-02-04 | 2009-05-21 | Diversa Corporation | Gene site saturation mutagenesis |
CA2365601A1 (en) | 1999-03-29 | 2000-10-05 | Kansai Technology Licensing Organization Co., Ltd. | Novel cytidine deaminase |
DE60043158D1 (de) | 1999-11-18 | 2009-11-26 | Pharmexa Inc | Heteroklitische analoga von klasse-i epitopen |
AU785007B2 (en) | 1999-11-24 | 2006-08-24 | Mcs Micro Carrier Systems Gmbh | Polypeptides comprising multimers of nuclear localization signals or of protein transduction domains and their use for transferring molecules into cells |
IL150069A0 (en) | 1999-12-06 | 2002-12-01 | Sangamo Biosciences Inc | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
US6689558B2 (en) | 2000-02-08 | 2004-02-10 | Sangamo Biosciences, Inc. | Cells for drug discovery |
MXPA03003690A (es) | 2000-10-27 | 2004-05-05 | Chiron Spa | Acidos nucleicos y proteinas de los grupos a y b de estreptococos. |
CN101317825A (zh) | 2000-10-30 | 2008-12-10 | 欧罗赛铁克股份有限公司 | 控释氢可酮制剂 |
US20040003420A1 (en) | 2000-11-10 | 2004-01-01 | Ralf Kuhn | Modified recombinase |
AU2002227882B2 (en) | 2001-01-25 | 2007-06-28 | Evolva Ltd | Concatemers of differentially expressed multiple genes |
US20050222030A1 (en) * | 2001-02-21 | 2005-10-06 | Anthony Allison | Modified annexin proteins and methods for preventing thrombosis |
ATE398180T1 (de) * | 2001-02-27 | 2008-07-15 | Univ Rochester | Verfahren und zusammensetzungen zur modifikation der bearbeitung von apolipoprotein b-mrna |
CA2444098C (en) | 2001-04-19 | 2016-06-21 | The Scripps Research Institute | Methods and composition for the production of orthogonal trna-aminoacyltrna synthetase pairs |
AU2002330714A1 (en) | 2001-05-30 | 2003-01-02 | Biomedical Center | In silico screening for phenotype-associated expressed sequences |
EP1578932A4 (en) | 2002-07-12 | 2006-08-30 | Affymetrix Inc | SYNTHETIC MARKER GENES |
JP2006500030A (ja) | 2002-09-20 | 2006-01-05 | イェール ユニバーシティ | リボスイッチ、その使用方法、ならびにリボスイッチとともに用いるための組成物 |
AU2004230592B2 (en) | 2003-04-14 | 2007-10-04 | Caliper Life Sciences, Inc. | Reduction of migration shift assay interference |
DE602004011789T2 (de) | 2003-07-07 | 2009-02-12 | The Scripps Research Institute, La Jolla | Zusammensetzungen der orthogonalen Lysyl-tRNA und Aminoacyl-tRNA Synthetase Paaren und ihre Verwendungen |
ES2808687T3 (es) | 2003-08-08 | 2021-03-01 | Sangamo Therapeutics Inc | Métodos y composiciones para escisión dirigida y recombinación |
WO2005098043A2 (en) | 2004-03-30 | 2005-10-20 | The President And Fellows Of Harvard College | Ligand-dependent protein splicing |
US7919277B2 (en) | 2004-04-28 | 2011-04-05 | Danisco A/S | Detection and typing of bacterial strains |
CA2632216C (en) | 2004-07-06 | 2013-11-12 | Societe De Commercialisation Des Produits De La Recherche Appliquee Socpra Sciences Sante Et Humaines S.E.C. | A target-dependent nucleic acid adapter |
US8728526B2 (en) | 2004-08-19 | 2014-05-20 | The United States of America, Represented by Secretary of Department of Health and Human Services, NIH | Coacervate microparticles useful for the sustained release administration of therapeutic agents |
US9315862B2 (en) | 2004-10-05 | 2016-04-19 | California Institute Of Technology | Aptamer regulated nucleic acids and uses thereof |
JP2006248978A (ja) | 2005-03-10 | 2006-09-21 | Mebiopharm Co Ltd | 新規なリポソーム製剤 |
AU2015252023B2 (en) | 2005-08-26 | 2017-06-29 | Dupont Nutrition Biosciences Aps | Use |
AU2012244264B2 (en) | 2005-08-26 | 2015-08-06 | Dupont Nutrition Biosciences Aps | Use |
DK3284833T3 (da) | 2005-08-26 | 2022-02-07 | Dupont Nutrition Biosci Aps | Anvendelse af crispr-associerede gener (cas) |
US20080051317A1 (en) | 2005-12-15 | 2008-02-28 | George Church | Polypeptides comprising unnatural amino acids, methods for their production and uses therefor |
CA2651389C (en) | 2006-05-05 | 2017-04-25 | Molecular Transfer, Inc. | Novel reagents for transfection of eukaryotic cells |
ES2373586T3 (es) | 2006-05-19 | 2012-02-06 | Danisco A/S | Microorganismos marcados y métodos para marcar. |
CA2656298A1 (en) | 2006-06-02 | 2007-12-13 | President And Fellows Of Harvard College | Protein surface remodeling |
BRPI0808704B1 (pt) | 2007-03-02 | 2022-01-18 | Dupont Nutrition Biosciences Aps | Método para gerar uma cultura inicial compreendendo pelo menos duas cepas variantes resistentes a bacteriófagos, cultura iniciadora e método de fermentação |
WO2009033027A2 (en) | 2007-09-05 | 2009-03-12 | Medtronic, Inc. | Suppression of scn9a gene expression and/or function for the treatment of pain |
EP2188384B1 (en) | 2007-09-27 | 2015-07-15 | Sangamo BioSciences, Inc. | Rapid in vivo identification of biologically active nucleases |
US20110014616A1 (en) | 2009-06-30 | 2011-01-20 | Sangamo Biosciences, Inc. | Rapid screening of biologically active nucleases and isolation of nuclease-modified cells |
US9029524B2 (en) | 2007-12-10 | 2015-05-12 | California Institute Of Technology | Signal activated RNA interference |
WO2009100373A1 (en) | 2008-02-08 | 2009-08-13 | Sangamo Biosciences, Inc. | Treatment of chronic pain with zinc finger proteins |
WO2009146179A1 (en) | 2008-04-15 | 2009-12-03 | University Of Iowa Research Foundation | Zinc finger nuclease for the cftr gene and methods of use thereof |
CN102066405B (zh) | 2008-04-28 | 2015-09-30 | 哈佛大学校长及研究员协会 | 用于细胞穿透的超荷电蛋白 |
US8394604B2 (en) | 2008-04-30 | 2013-03-12 | Paul Xiang-Qin Liu | Protein splicing using short terminal split inteins |
US9400597B2 (en) | 2008-07-23 | 2016-07-26 | Microsoft Technology Licensing, Llc | Presenting dynamic grids |
US8546553B2 (en) | 2008-07-25 | 2013-10-01 | University Of Georgia Research Foundation, Inc. | Prokaryotic RNAi-like system and methods of use |
EP2159286A1 (en) | 2008-09-01 | 2010-03-03 | Consiglio Nazionale Delle Ricerche | Method for obtaining oligonucleotide aptamers and uses thereof |
US8790664B2 (en) | 2008-09-05 | 2014-07-29 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Multimodular assembly useful for intracellular delivery |
US8636884B2 (en) | 2008-09-15 | 2014-01-28 | Abbott Diabetes Care Inc. | Cationic polymer based wired enzyme formulations for use in analyte sensors |
US20100076057A1 (en) | 2008-09-23 | 2010-03-25 | Northwestern University | TARGET DNA INTERFERENCE WITH crRNA |
US9404098B2 (en) | 2008-11-06 | 2016-08-02 | University Of Georgia Research Foundation, Inc. | Method for cleaving a target RNA using a Cas6 polypeptide |
US10662227B2 (en) | 2008-11-07 | 2020-05-26 | Dupont Nutrition Biosciences Aps | Bifidobacteria CRISPR sequences |
US9175338B2 (en) | 2008-12-11 | 2015-11-03 | Pacific Biosciences Of California, Inc. | Methods for identifying nucleic acid modifications |
AU2009325069B2 (en) | 2008-12-11 | 2015-03-19 | Pacific Biosciences Of California, Inc. | Classification of nucleic acid templates |
WO2010075424A2 (en) | 2008-12-22 | 2010-07-01 | The Regents Of University Of California | Compositions and methods for downregulating prokaryotic genes |
WO2010091294A2 (en) | 2009-02-05 | 2010-08-12 | The Regents Of The University Of California | New targeted antimicrobial moieties |
CN102421897B (zh) | 2009-03-06 | 2015-12-16 | 合成基因组股份有限公司 | 用于克隆和操作基因组的方法 |
EP2425023B1 (en) | 2009-04-27 | 2015-12-23 | Pacific Biosciences of California, Inc. | Real-time sequencing methods and systems |
WO2010129023A2 (en) | 2009-04-28 | 2010-11-11 | President And Fellows Of Harvard College | Supercharged proteins for cell penetration |
US9063156B2 (en) | 2009-06-12 | 2015-06-23 | Pacific Biosciences Of California, Inc. | Real-time analytical methods and systems |
WO2011000106A1 (en) | 2009-07-01 | 2011-01-06 | Protiva Biotherapeutics, Inc. | Improved cationic lipids and methods for the delivery of therapeutic agents |
NZ598457A (en) | 2009-08-03 | 2014-06-27 | Recombinetics Inc | Methods and compositions for targeted gene modification |
WO2011017293A2 (en) | 2009-08-03 | 2011-02-10 | The General Hospital Corporation | Engineering of zinc finger arrays by context-dependent assembly |
GB0913681D0 (en) | 2009-08-05 | 2009-09-16 | Glaxosmithkline Biolog Sa | Immunogenic composition |
DK2494060T3 (en) | 2009-10-30 | 2016-08-01 | Synthetic Genomics Inc | Coding of text for nucleic acid sequences |
SG10201912571XA (en) | 2009-11-02 | 2020-02-27 | Univ Washington | Therapeutic nuclease compositions and methods |
US20110142886A1 (en) | 2009-12-01 | 2011-06-16 | Intezyne Technologies, Incorporated | Pegylated polyplexes for polynucleotide delivery |
JP2013514779A (ja) * | 2009-12-18 | 2013-05-02 | ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティ | 脱メチル化および細胞の再プログラミングを促進するためのシチジンデアミナーゼ関連薬剤の使用 |
NZ600546A (en) | 2010-01-22 | 2014-08-29 | Dow Agrosciences Llc | Excision of transgenes in genetically modified organisms |
JP2013520989A (ja) | 2010-03-05 | 2013-06-10 | シンセティック ジェノミクス インコーポレーテッド | ゲノムのクローニングおよび操作のための方法 |
WO2011123830A2 (en) | 2010-04-02 | 2011-10-06 | Amunix Operating Inc. | Alpha 1-antitrypsin compositions and methods of making and using same |
BR112012028805A2 (pt) | 2010-05-10 | 2019-09-24 | The Regents Of The Univ Of California E Nereus Pharmaceuticals Inc | composições de endorribonuclease e métodos de uso das mesmas. |
CN103025344B (zh) | 2010-05-17 | 2016-06-29 | 桑格摩生物科学股份有限公司 | 新型dna-结合蛋白及其用途 |
GB201008267D0 (en) | 2010-05-18 | 2010-06-30 | Univ Edinburgh | Cationic lipids |
US8748667B2 (en) | 2010-06-04 | 2014-06-10 | Sirna Therapeutics, Inc. | Low molecular weight cationic lipids for oligonucleotide delivery |
US20110201118A1 (en) | 2010-06-14 | 2011-08-18 | Iowa State University Research Foundation, Inc. | Nuclease activity of tal effector and foki fusion protein |
AU2011305572B2 (en) | 2010-09-20 | 2016-08-04 | Diane Goll | Microencapsulation process and product |
DK2630156T3 (en) | 2010-10-20 | 2018-12-17 | Dupont Nutrition Biosci Aps | CRISPR-CAS SEQUENCES OF LACTOCOCCUS |
EP2642985A4 (en) | 2010-11-26 | 2014-05-07 | Univ Witwatersrand Jhb | POLYMER MATRIX OF POLYMER-LIPID NANOPARTICLES AS DOSED PHARMACEUTICAL FORM |
KR101255338B1 (ko) | 2010-12-15 | 2013-04-16 | 포항공과대학교 산학협력단 | 표적 세포에 대한 폴리뉴클레오티드 전달체 |
ES2753198T5 (es) | 2010-12-16 | 2023-05-31 | Amgen Europe Gmbh | Formas farmacéuticas orales de liberación controlada de fármacos poco solubles y sus usos |
US9499592B2 (en) | 2011-01-26 | 2016-11-22 | President And Fellows Of Harvard College | Transcription activator-like effectors |
US9528124B2 (en) | 2013-08-27 | 2016-12-27 | Recombinetics, Inc. | Efficient non-meiotic allele introgression |
WO2012125445A2 (en) | 2011-03-11 | 2012-09-20 | President And Fellows Of Harvard College | Small molecule-dependent inteins and uses thereof |
US9164079B2 (en) | 2011-03-17 | 2015-10-20 | Greyledge Technologies Llc | Systems for autologous biological therapeutics |
US20120244601A1 (en) | 2011-03-22 | 2012-09-27 | Bertozzi Carolyn R | Riboswitch based inducible gene expression platform |
US8709466B2 (en) | 2011-03-31 | 2014-04-29 | International Business Machines Corporation | Cationic polymers for antimicrobial applications and delivery of bioactive materials |
WO2012138927A2 (en) | 2011-04-05 | 2012-10-11 | Philippe Duchateau | Method for the generation of compact tale-nucleases and uses thereof |
WO2012148953A1 (en) | 2011-04-25 | 2012-11-01 | Stc.Unm | Solid compositions for pharmaceutical use |
CN103492578B (zh) | 2011-04-27 | 2016-10-19 | 阿迈瑞斯公司 | 用于基因组修饰的方法 |
WO2012158985A2 (en) | 2011-05-17 | 2012-11-22 | Transposagen Biopharmaceuticals, Inc. | Methods for site-specific genetic modification in spermatogonial stem cells using zinc finger nuclease (zfn) for the creation of model organisms |
WO2012158986A2 (en) | 2011-05-17 | 2012-11-22 | Transposagen Biopharmaceuticals, Inc. | Methods for site-specific genetic modification in stem cells using xanthomonas tal nucleases (xtn) for the creation of model organisms |
US8691750B2 (en) | 2011-05-17 | 2014-04-08 | Axolabs Gmbh | Lipids and compositions for intracellular delivery of biologically active compounds |
US20140113376A1 (en) | 2011-06-01 | 2014-04-24 | Rotem Sorek | Compositions and methods for downregulating prokaryotic genes |
EP2732038B1 (en) | 2011-07-15 | 2018-09-05 | The General Hospital Corporation | Methods of transcription activator like effector assembly |
EP2734622B1 (en) | 2011-07-19 | 2018-09-05 | Vivoscript, Inc. | Compositions and methods for re-programming cells without genetic modification for repairing cartilage damage |
US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
DK2762569T3 (en) | 2011-09-28 | 2018-08-27 | Ribomic Inc | NGF aptamer and its use. |
GB2496687A (en) | 2011-11-21 | 2013-05-22 | Gw Pharma Ltd | Tetrahydrocannabivarin (THCV) in the protection of pancreatic islet cells |
IN2014MN00974A (ja) | 2011-12-16 | 2015-04-24 | Targetgene Biotechnologies Ltd | |
MX2014007233A (es) | 2011-12-16 | 2015-02-04 | Moderna Therapeutics Inc | Composiciones de nucleosidos, nucleotidos y acidos nucleicos modificados. |
GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
WO2013119602A1 (en) | 2012-02-06 | 2013-08-15 | President And Fellows Of Harvard College | Arrdc1-mediated microvesicles (armms) and uses thereof |
RU2650811C2 (ru) | 2012-02-24 | 2018-04-17 | Фред Хатчинсон Кэнсер Рисерч Сентер | Композиции и способы лечения гемоглобинопатии |
EP2820159B1 (en) | 2012-02-29 | 2019-10-23 | Sangamo Therapeutics, Inc. | Methods and compositions for treating huntington's disease |
JP2015512255A (ja) | 2012-03-17 | 2015-04-27 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | ざ瘡の高速診断および個人化された治療 |
WO2013141680A1 (en) | 2012-03-20 | 2013-09-26 | Vilnius University | RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX |
US9637739B2 (en) | 2012-03-20 | 2017-05-02 | Vilnius University | RNA-directed DNA cleavage by the Cas9-crRNA complex |
WO2013152359A1 (en) | 2012-04-06 | 2013-10-10 | The Regents Of The University Of California | Novel tetrazines and method of synthesizing the same |
BR112014026203A2 (pt) | 2012-04-23 | 2017-07-18 | Bayer Cropscience Nv | engenharia do genoma direcionado nas plantas |
KR102091298B1 (ko) | 2012-05-02 | 2020-03-19 | 다우 아그로사이언시즈 엘엘씨 | 말산 탈수소효소의 표적화된 변형 |
JP6559063B2 (ja) | 2012-05-07 | 2019-08-14 | サンガモ セラピューティクス, インコーポレイテッド | 導入遺伝子のヌクレアーゼ媒介標的化組み込みのための方法および組成物 |
WO2013169398A2 (en) | 2012-05-09 | 2013-11-14 | Georgia Tech Research Corporation | Systems and methods for improving nuclease specificity and activity |
PL2855667T3 (pl) | 2012-05-25 | 2024-03-25 | Cellectis | Sposoby uzyskiwania metodami inżynierii allogenicznych i opornych na immunosupresję limfocytów t do immunoterapii |
US20150017136A1 (en) | 2013-07-15 | 2015-01-15 | Cellectis | Methods for engineering allogeneic and highly active t cell for immunotherapy |
US20140056868A1 (en) | 2012-05-30 | 2014-02-27 | University of Washington Center for Commercialization | Supercoiled MiniVectors as a Tool for DNA Repair, Alteration and Replacement |
US9102936B2 (en) | 2012-06-11 | 2015-08-11 | Agilent Technologies, Inc. | Method of adaptor-dimer subtraction using a CRISPR CAS6 protein |
CN104540382A (zh) | 2012-06-12 | 2015-04-22 | 弗·哈夫曼-拉罗切有限公司 | 用于产生条件性敲除等位基因的方法和组合物 |
EP2674501A1 (en) | 2012-06-14 | 2013-12-18 | Agence nationale de sécurité sanitaire de l'alimentation,de l'environnement et du travail | Method for detecting and identifying enterohemorrhagic Escherichia coli |
US9688971B2 (en) | 2012-06-15 | 2017-06-27 | The Regents Of The University Of California | Endoribonuclease and methods of use thereof |
EP2861737B1 (en) | 2012-06-19 | 2019-04-17 | Regents Of The University Of Minnesota | Gene targeting in plants using dna viruses |
CN104508130B (zh) | 2012-06-29 | 2016-09-28 | 麻省理工学院 | 大规模并行组合遗传学 |
US9125508B2 (en) | 2012-06-30 | 2015-09-08 | Seasons 4, Inc. | Collapsible tree system |
EP2872154B1 (en) | 2012-07-11 | 2017-05-31 | Sangamo BioSciences, Inc. | Methods and compositions for delivery of biologics |
EP3444342B1 (en) | 2012-07-11 | 2020-05-27 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
CN116622704A (zh) | 2012-07-25 | 2023-08-22 | 布罗德研究所有限公司 | 可诱导的dna结合蛋白和基因组干扰工具及其应用 |
EP2879678B1 (en) | 2012-07-31 | 2023-03-01 | Yeda Research and Development Co. Ltd. | Enoxacin for treating amyotrophic lateral sclerosis |
US10058078B2 (en) | 2012-07-31 | 2018-08-28 | Recombinetics, Inc. | Production of FMDV-resistant livestock by allele substitution |
EP2880171B1 (en) | 2012-08-03 | 2018-10-03 | The Regents of The University of California | Methods and compositions for controlling gene expression by rna processing |
KR102474010B1 (ko) | 2012-08-29 | 2022-12-02 | 상가모 테라퓨틱스, 인코포레이티드 | 유전적 병태를 치료하기 위한 방법 및 조성물 |
WO2014039513A2 (en) | 2012-09-04 | 2014-03-13 | The Trustees Of The University Of Pennsylvania | Inhibition of diacylglycerol kinase to augment adoptive t cell transfer |
EP4148134A1 (en) | 2012-09-04 | 2023-03-15 | The Scripps Research Institute | Chimeric polypeptides having targeted binding specificity |
ES2714523T3 (es) | 2012-09-04 | 2019-05-28 | Cellectis | Receptor quimérico de antígenos multicatenario y usos del mismo |
US9914930B2 (en) | 2012-09-07 | 2018-03-13 | Dow Agrosciences Llc | FAD3 performance loci and corresponding target site specific binding proteins capable of inducing targeted breaks |
UA119135C2 (uk) | 2012-09-07 | 2019-05-10 | ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі | Спосіб отримання трансгенної рослини |
UA118090C2 (uk) | 2012-09-07 | 2018-11-26 | ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі | Спосіб інтегрування послідовності нуклеїнової кислоти, що представляє інтерес, у ген fad2 у клітині сої та специфічний для локусу fad2 білок, що зв'язується, здатний індукувати спрямований розрив |
US20140075593A1 (en) | 2012-09-07 | 2014-03-13 | Dow Agrosciences Llc | Fluorescence activated cell sorting (facs) enrichment to generate plants |
WO2014043143A1 (en) | 2012-09-11 | 2014-03-20 | Life Technologies Corporation | Nucleic acid amplification |
GB201216564D0 (en) | 2012-09-17 | 2012-10-31 | Univ Edinburgh | Genetically edited animal |
WO2014047103A2 (en) | 2012-09-18 | 2014-03-27 | The Translational Genomics Research Institute | Isolated genes and transgenic organisms for producing biofuels |
JP6401704B2 (ja) | 2012-10-10 | 2018-10-10 | サンガモ セラピューティクス, インコーポレイテッド | T細胞を修飾する化合物およびその使用 |
EP2906602B1 (en) | 2012-10-12 | 2019-01-16 | The General Hospital Corporation | Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins |
ES2926021T3 (es) | 2012-10-23 | 2022-10-21 | Toolgen Inc | Composición para escindir un ADN objetivo que comprende un ARN guía específico para el ADN objetivo y ácido nucleico codificador de proteína Cas o proteína Cas, y uso de la misma |
US20140115728A1 (en) | 2012-10-24 | 2014-04-24 | A. Joseph Tector | Double knockout (gt/cmah-ko) pigs, organs and tissues |
JP2015533284A (ja) | 2012-10-30 | 2015-11-24 | リコンビネティクス・インコーポレイテッドRecombinetics,Inc. | 動物における性成熟の制御 |
WO2014068346A2 (en) | 2012-10-31 | 2014-05-08 | Mezögazdásagi Biotechnológiai Kutatóközpont | Identification of a xanthomonas euvesicatoria resistance gene from pepper (capsicum annuum) and method for generating plants with resistance |
BR112015009812A2 (pt) | 2012-10-31 | 2017-08-22 | Cellectis | Método para a inserção genética específica em um genoma de planta, célula de planta transformada e seu uso, planta resistente a herbicidas, kit, vetor, e célula hospedeira |
WO2014071235A1 (en) | 2012-11-01 | 2014-05-08 | Massachusetts Institute Of Technology | Genetic device for the controlled destruction of dna |
RU2019143431A (ru) | 2012-11-01 | 2020-04-28 | Фэктор Байосайенс Инк. | Способы и продукты для экспрессии белков в клетках |
US20140127752A1 (en) | 2012-11-07 | 2014-05-08 | Zhaohui Zhou | Method, composition, and reagent kit for targeted genomic enrichment |
US9879227B2 (en) | 2012-11-09 | 2018-01-30 | Marco Archetti | Diffusible factors and cancer cells |
WO2014081730A1 (en) | 2012-11-20 | 2014-05-30 | Cold Spring Harbor Laboratory | Mutations in solanaceae plants that modulate shoot architecture and enhance yield-related phenotypes |
KR20150085846A (ko) | 2012-11-20 | 2015-07-24 | 제이.알.심프롯캄패니 | Tal-매개 전이 DNA 삽입방법 |
WO2014081855A1 (en) | 2012-11-20 | 2014-05-30 | Universite De Montreal | Methods and compositions for muscular dystrophies |
CN109554350B (zh) | 2012-11-27 | 2022-09-23 | 儿童医疗中心有限公司 | 用于胎儿血红蛋白再诱导的靶向bcl11a远端调控元件 |
WO2014085261A1 (en) | 2012-11-29 | 2014-06-05 | North Carolina State University | Synthetic pathway for biological carbon dioxide sequestration |
WO2014085830A2 (en) | 2012-11-30 | 2014-06-05 | The Parkinson's Institute | Screening assays for therapeutics for parkinson's disease |
DK2925866T3 (en) | 2012-11-30 | 2018-10-29 | Univ Aarhus | CIRCULAR RNA FOR INHIBITING MICRO-RNA |
AU2013355327A1 (en) | 2012-12-05 | 2015-06-11 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of metabolic disorders |
EP3617309A3 (en) | 2012-12-06 | 2020-05-06 | Sigma Aldrich Co. LLC | Crispr-based genome modification and regulation |
ES2740648T3 (es) | 2012-12-06 | 2020-02-06 | Synthetic Genomics Inc | Mutantes de algas que tienen un fenotipo aclimatado a alta luz bloqueado |
US9447422B2 (en) | 2012-12-06 | 2016-09-20 | Synthetic Genomics, Inc. | Autonomous replication sequences and episomal DNA molecules |
US10272163B2 (en) | 2012-12-07 | 2019-04-30 | The Regents Of The University Of California | Factor VIII mutation repair and tolerance induction |
US9914931B2 (en) | 2012-12-07 | 2018-03-13 | Synthetic Genomics, Inc. | Nannochloropsis spliced leader sequences and uses therefor |
WO2014093479A1 (en) | 2012-12-11 | 2014-06-19 | Montana State University | Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation |
SG10201912328UA (en) | 2012-12-12 | 2020-02-27 | Broad Inst Inc | Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications |
US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
EP3144390B1 (en) | 2012-12-12 | 2020-03-18 | The Broad Institute, Inc. | Engineering of systems, methods and optimized guide compositions for sequence manipulation |
CN114634950A (zh) | 2012-12-12 | 2022-06-17 | 布罗德研究所有限公司 | 用于序列操纵的crispr-cas组分系统、方法以及组合物 |
EP2931899A1 (en) | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
WO2014093709A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
IL239344B1 (en) | 2012-12-12 | 2024-02-01 | Broad Inst Inc | Systems engineering, methods and optimal guiding components for sequence manipulation |
ES2576128T3 (es) | 2012-12-12 | 2016-07-05 | The Broad Institute, Inc. | Modificación por tecnología genética y optimización de sistemas, métodos y composiciones para la manipulación de secuencias con dominios funcionales |
US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
CN113355357A (zh) | 2012-12-12 | 2021-09-07 | 布罗德研究所有限公司 | 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化 |
DK2825654T3 (en) | 2012-12-12 | 2017-08-21 | Broad Inst Inc | SYSTEMS, PROCEDURES, AND COMPOSITIONS WITH CRISPR-CAS COMPONENTS FOR SEQUENCE MANIPULATION. |
RU2015128052A (ru) | 2012-12-13 | 2017-01-19 | Дау Агросайенсиз Ллс | Точный таргетинг генов в отношении конкретного локуса кукурузы |
AU2013359146B2 (en) | 2012-12-13 | 2017-12-07 | Corteva Agriscience Llc | DNA detection methods for site specific nuclease activity |
CN105144204B (zh) | 2012-12-13 | 2018-02-27 | 麻省理工学院 | 基于重组酶的逻辑与存储系统 |
IL308158A (en) | 2012-12-17 | 2023-12-01 | Harvard College | RNA-guided human genome engineering |
CN105120656A (zh) | 2012-12-21 | 2015-12-02 | 塞尔克蒂斯股份有限公司 | 冷诱导甜味降低的马铃薯 |
DK3491915T3 (da) | 2012-12-27 | 2023-08-28 | Keygene Nv | Fremgangsmåde til at inducere en målrettet translokation i en plante |
WO2014110006A1 (en) | 2013-01-10 | 2014-07-17 | Ge Healthcare Dharmacon, Inc. | Templates, libraries, kits and methods for generating molecules |
CN105142396A (zh) | 2013-01-14 | 2015-12-09 | 重组股份有限公司 | 缺角家畜 |
CN104995302B (zh) | 2013-01-16 | 2021-08-31 | 爱默蕾大学 | Cas9-核酸复合物及其相关用途 |
CN103233028B (zh) | 2013-01-25 | 2015-05-13 | 南京徇齐生物技术有限公司 | 一种无物种限制无生物安全性问题的真核生物基因打靶方法及螺旋结构dna序列 |
AU2014215025B2 (en) | 2013-02-05 | 2018-08-30 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Cell lines for virus production and methods of use |
WO2014124226A1 (en) | 2013-02-07 | 2014-08-14 | The Rockefeller University | Sequence specific antimicrobials |
WO2014127287A1 (en) | 2013-02-14 | 2014-08-21 | Massachusetts Institute Of Technology | Method for in vivo tergated mutagenesis |
ES2768291T3 (es) | 2013-02-14 | 2020-06-22 | Univ Osaka | Método para aislar una región genómica específica utilizando una molécula que se une específicamente a una secuencia de ADN endógena |
MX2015010841A (es) | 2013-02-20 | 2016-05-09 | Regeneron Pharma | Modificacion genetica de ratas. |
US20150353885A1 (en) | 2013-02-21 | 2015-12-10 | Cellectis | Method to counter-select cells or organisms by linking loci to nuclease components |
ES2522765B2 (es) | 2013-02-22 | 2015-03-18 | Universidad De Alicante | Método para dectectar inserciones de espaciadores en estructuras CRISPR |
JP6491113B2 (ja) | 2013-02-25 | 2019-03-27 | サンガモ セラピューティクス, インコーポレイテッド | ヌクレアーゼ媒介性遺伝子破壊を増強するための方法および組成物 |
EP2922393B2 (en) | 2013-02-27 | 2022-12-28 | Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) | Gene editing in the oocyte by cas9 nucleases |
WO2014138379A1 (en) | 2013-03-06 | 2014-09-12 | The Johns Hopkins University | The telomerator-a tool for chromosome engineering |
WO2014143381A1 (en) | 2013-03-09 | 2014-09-18 | Agilent Technologies, Inc. | Methods of in vivo engineering of large sequences using multiple crispr/cas selections of recombineering events |
WO2014165177A1 (en) | 2013-03-12 | 2014-10-09 | Sangamo Biosciences, Inc. | Methods and compositions for modification of hla |
US10329574B2 (en) | 2013-03-12 | 2019-06-25 | E I Du Pont De Nemours And Company | Methods for the identification of variant recognition sites for rare-cutting engineered double-strand-break-inducing agents and compositions and uses thereof |
AU2014235794A1 (en) | 2013-03-14 | 2015-10-22 | Caribou Biosciences, Inc. | Compositions and methods of nucleic acid-targeting nucleic acids |
WO2014153118A1 (en) | 2013-03-14 | 2014-09-25 | The Board Of Trustees Of The Leland Stanford Junior University | Treatment of diseases and conditions associated with dysregulation of mammalian target of rapamycin complex 1 (mtorc1) |
IL290273B2 (en) | 2013-03-15 | 2024-03-01 | Cibus Us Llc | Methods and compositions for increasing the efficiency of targeted gene modification through the use of gene repair with oligonucleotides - mediators |
US9234213B2 (en) | 2013-03-15 | 2016-01-12 | System Biosciences, Llc | Compositions and methods directed to CRISPR/Cas genomic engineering systems |
US20160046959A1 (en) | 2013-03-15 | 2016-02-18 | Carlisle P. Landel | Reproducible method for testis-mediated genetic modification (tgm) and sperm-mediated genetic modification (sgm) |
US10760064B2 (en) | 2013-03-15 | 2020-09-01 | The General Hospital Corporation | RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci |
WO2015099850A1 (en) | 2013-12-26 | 2015-07-02 | The General Hospital Corporation | Multiplex guide rnas |
US11332719B2 (en) | 2013-03-15 | 2022-05-17 | The Broad Institute, Inc. | Recombinant virus and preparations thereof |
US20140273235A1 (en) | 2013-03-15 | 2014-09-18 | Regents Of The University Of Minnesota | ENGINEERING PLANT GENOMES USING CRISPR/Cas SYSTEMS |
WO2014144094A1 (en) | 2013-03-15 | 2014-09-18 | J.R. Simplot Company | Tal-mediated transfer dna insertion |
US20140349400A1 (en) | 2013-03-15 | 2014-11-27 | Massachusetts Institute Of Technology | Programmable Modification of DNA |
US20140273230A1 (en) | 2013-03-15 | 2014-09-18 | Sigma-Aldrich Co., Llc | Crispr-based genome modification and regulation |
WO2014204578A1 (en) | 2013-06-21 | 2014-12-24 | The General Hospital Corporation | Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing |
CA2906970C (en) | 2013-03-21 | 2021-05-18 | Ospedale San Raffaele Srl | Targeted disruption of t cell receptor genes using engineered zinc finger protein nucleases |
WO2014161821A1 (en) | 2013-04-02 | 2014-10-09 | Bayer Cropscience Nv | Targeted genome engineering in eukaryotes |
EP3789487A1 (en) | 2013-04-03 | 2021-03-10 | Memorial Sloan Kettering Cancer Center | Effective generation of tumor-targeted t-cells derived from pluripotent stem cells |
CA2908253C (en) | 2013-04-04 | 2024-01-09 | Trustees Of Dartmouth College | Compositions and methods for in vivo excision of hiv-1 proviral dna |
EP2981617B1 (en) | 2013-04-04 | 2023-07-05 | President and Fellows of Harvard College | Therapeutic uses of genome editing with crispr/cas systems |
EP2981166B1 (en) | 2013-04-05 | 2020-09-09 | Dow AgroSciences LLC | Methods and compositions for integration of an exogenous sequence within the genome of plants |
US20150056629A1 (en) | 2013-04-14 | 2015-02-26 | Katriona Guthrie-Honea | Compositions, systems, and methods for detecting a DNA sequence |
WO2014172470A2 (en) | 2013-04-16 | 2014-10-23 | Whitehead Institute For Biomedical Research | Methods of mutating, modifying or modulating nucleic acid in a cell or nonhuman mammal |
EP2986709A4 (en) | 2013-04-16 | 2017-03-15 | University Of Washington Through Its Center For Commercialization | Activating an alternative pathway for homology-directed repair to stimulate targeted gene correction and genome engineering |
DK2986729T3 (en) | 2013-04-16 | 2018-10-29 | Regeneron Pharma | TARGETED MODIFICATION OF ROOT THROUGH |
EP2796558A1 (en) | 2013-04-23 | 2014-10-29 | Rheinische Friedrich-Wilhelms-Universität Bonn | Improved gene targeting and nucleic acid carrier molecule, in particular for use in plants |
CN103224947B (zh) | 2013-04-28 | 2015-06-10 | 陕西师范大学 | 一种基因打靶系统 |
CA2910427C (en) | 2013-05-10 | 2024-02-20 | Sangamo Biosciences, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
JP6599848B2 (ja) | 2013-05-10 | 2019-10-30 | ホワイトヘッド・インスティテュート・フォー・バイオメディカル・リサーチ | ソルタギング可能なタンパク質を有する赤血球のinvitro生成 |
CA2912375C (en) | 2013-05-13 | 2023-03-14 | Cellectis | Methods for engineering highly active t cell for immunotherapy |
FI2997141T3 (fi) | 2013-05-13 | 2022-12-15 | CD19-spesifinen kimeerinen antigeenireseptori ja sen käyttöjä | |
EP3730615A3 (en) | 2013-05-15 | 2020-12-09 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
WO2014186686A2 (en) | 2013-05-17 | 2014-11-20 | Two Blades Foundation | Targeted mutagenesis and genome engineering in plants using rna-guided cas nucleases |
US20140349405A1 (en) | 2013-05-22 | 2014-11-27 | Wisconsin Alumni Research Foundation | Rna-directed dna cleavage and gene editing by cas9 enzyme from neisseria meningitidis |
CA2913830C (en) | 2013-05-29 | 2021-06-29 | Cellectis | Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system |
JP7065564B2 (ja) | 2013-05-29 | 2022-05-12 | セレクティス | Cas9ニッカーゼ活性を用いて正確なdna切断をもたらすための方法 |
US11414695B2 (en) | 2013-05-29 | 2022-08-16 | Agilent Technologies, Inc. | Nucleic acid enrichment using Cas9 |
US11685935B2 (en) | 2013-05-29 | 2023-06-27 | Cellectis | Compact scaffold of Cas9 in the type II CRISPR system |
WO2014194190A1 (en) | 2013-05-30 | 2014-12-04 | The Penn State Research Foundation | Gene targeting and genetic modification of plants via rna-guided genome editing |
AU2014273089B2 (en) | 2013-05-31 | 2018-02-22 | Cellectis | A LAGLIDADG homing endonuclease cleaving the C-C Chemokine Receptor Type-5 (CCR5) gene and uses thereof |
US20140359796A1 (en) | 2013-05-31 | 2014-12-04 | Recombinetics, Inc. | Genetically sterile animals |
US10000746B2 (en) | 2013-05-31 | 2018-06-19 | Cellectis | LAGLIDADG homing endonuclease cleaving the T cell receptor alpha gene and uses thereof |
EP3603679B1 (en) | 2013-06-04 | 2022-08-10 | President and Fellows of Harvard College | Rna-guided transcriptional regulation |
US9267135B2 (en) | 2013-06-04 | 2016-02-23 | President And Fellows Of Harvard College | RNA-guided transcriptional regulation |
CA2914519A1 (en) | 2013-06-05 | 2014-12-11 | Duke University | Rna-guided gene editing and gene regulation |
US20150315252A1 (en) | 2013-06-11 | 2015-11-05 | Clontech Laboratories, Inc. | Protein enriched microvesicles and methods of making and using the same |
WO2014200659A1 (en) | 2013-06-11 | 2014-12-18 | Clontech Laboratories, Inc. | Protein enriched microvesicles and methods of making and using the same |
WO2014201015A2 (en) | 2013-06-11 | 2014-12-18 | The Regents Of The University Of California | Methods and compositions for target dna modification |
WO2014199358A1 (en) | 2013-06-14 | 2014-12-18 | Cellectis | Methods for non-transgenic genome editing in plants |
AU2014281028B2 (en) | 2013-06-17 | 2020-09-10 | Massachusetts Institute Of Technology | Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy |
EP3011035B1 (en) | 2013-06-17 | 2020-05-13 | The Broad Institute, Inc. | Assay for quantitative evaluation of target site cleavage by one or more crispr-cas guide sequences |
EP3725885A1 (en) | 2013-06-17 | 2020-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
WO2014204724A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
EP3011034B1 (en) | 2013-06-17 | 2019-08-07 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components |
WO2014204725A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
CA2915845A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells |
SG11201510297QA (en) | 2013-06-19 | 2016-01-28 | Sigma Aldrich Co Llc | Targeted integration |
CA2915779A1 (en) | 2013-06-25 | 2014-12-31 | Cellectis | Modified diatoms for biofuel production |
US20160369268A1 (en) | 2013-07-01 | 2016-12-22 | The Board Of Regents Of The University Of Texas System | Transcription activator-like effector (tale) libraries and methods of synthesis and use |
RU2764637C2 (ru) | 2013-07-09 | 2022-01-19 | Президент Энд Фэллоуз Оф Харвард Коллидж | Мультиплексная геномная инженерия, направляемая рнк |
JP2016528890A (ja) | 2013-07-09 | 2016-09-23 | プレジデント アンド フェローズ オブ ハーバード カレッジ | CRISPR/Cas系を用いるゲノム編集の治療用の使用 |
RU2016104064A (ru) | 2013-07-10 | 2017-08-15 | Новартис Аг | Клетки мицелиальных грибов с множественной недостаточностью протеаз и способы их использования |
AU2014287250A1 (en) | 2013-07-10 | 2016-02-18 | EffStock, LLC | Mrap2 knockouts |
SG10201913015XA (en) | 2013-07-10 | 2020-02-27 | Harvard College | Orthogonal cas9 proteins for rna-guided gene regulation and editing |
JP7019233B2 (ja) | 2013-07-11 | 2022-02-15 | モデルナティエックス インコーポレイテッド | CRISPR関連タンパク質をコードする合成ポリヌクレオチドおよび合成sgRNAを含む組成物ならびに使用方法 |
CN106222197A (zh) | 2013-07-16 | 2016-12-14 | 中国科学院上海生命科学研究院 | 植物基因组定点修饰方法 |
US9663782B2 (en) | 2013-07-19 | 2017-05-30 | Larix Bioscience Llc | Methods and compositions for producing double allele knock outs |
GB201313235D0 (en) | 2013-07-24 | 2013-09-04 | Univ Edinburgh | Antiviral Compositions Methods and Animals |
CN103388006B (zh) | 2013-07-26 | 2015-10-28 | 华东师范大学 | 一种基因定点突变的构建方法 |
US10563225B2 (en) | 2013-07-26 | 2020-02-18 | President And Fellows Of Harvard College | Genome engineering |
US10421957B2 (en) | 2013-07-29 | 2019-09-24 | Agilent Technologies, Inc. | DNA assembly using an RNA-programmable nickase |
WO2015017866A1 (en) | 2013-08-02 | 2015-02-05 | Enevolv, Inc. | Processes and host cells for genome, pathway, and biomolecular engineering |
ITTO20130669A1 (it) | 2013-08-05 | 2015-02-06 | Consiglio Nazionale Ricerche | Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari |
US20150044772A1 (en) | 2013-08-09 | 2015-02-12 | Sage Labs, Inc. | Crispr/cas system-based novel fusion protein and its applications in genome editing |
US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
WO2015021990A1 (en) | 2013-08-16 | 2015-02-19 | University Of Copenhagen | Rna probing method and reagents |
WO2015024017A2 (en) | 2013-08-16 | 2015-02-19 | President And Fellows Of Harvard College | Rna polymerase, methods of purification and methods of use |
CN105705640B (zh) | 2013-08-20 | 2019-02-15 | 非营利性组织佛兰芒综合大学生物技术研究所 | 抑制lncRNA用于治疗黑素瘤 |
AU2014308899B2 (en) | 2013-08-22 | 2020-11-19 | E. I. Du Pont De Nemours And Company | Methods for producing genetic modifications in a plant genome without incorporating a selectable transgene marker, and compositions thereof |
US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
GB201315321D0 (en) | 2013-08-28 | 2013-10-09 | Koninklijke Nederlandse Akademie Van Wetenschappen | Transduction Buffer |
JP6588438B2 (ja) | 2013-08-28 | 2019-10-09 | サンガモ セラピューティクス, インコーポレイテッド | Dna結合ドメインと切断ドメインとを連結するための組成物 |
SG10201801658XA (en) | 2013-08-29 | 2018-03-28 | Univ Temple | Methods and compositions for rna-guided treatment of hiv infection |
EP3041345B1 (de) | 2013-09-04 | 2024-05-01 | KWS SAAT SE & Co. KGaA | Helminthosporium turcicum-resistente pflanze |
CA2922823C (en) | 2013-09-04 | 2023-01-17 | Dow Agrosciences Llc | Rapid targeting analysis in crops for determining donor insertion |
SG10201801782PA (en) | 2013-09-04 | 2018-04-27 | Csir | Site-specific nuclease single-cell assay targeting gene regulatory elements to silence gene expression |
US10760065B2 (en) | 2013-09-05 | 2020-09-01 | Massachusetts Institute Of Technology | Tuning microbial populations with programmable nucleases |
US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
WO2015040075A1 (en) | 2013-09-18 | 2015-03-26 | Genome Research Limited | Genomic screening methods using rna-guided endonucleases |
DE202014010413U1 (de) | 2013-09-18 | 2015-12-08 | Kymab Limited | Zellen und Organismen |
PT3049116T (pt) | 2013-09-23 | 2019-04-03 | Rensselaer Polytech Inst | Distribuição genética mediada por nanopartículas, edição genómica e modificação direcionada a ligantes em várias populações celulares |
WO2015048690A1 (en) | 2013-09-27 | 2015-04-02 | The Regents Of The University Of California | Optimized small guide rnas and methods of use |
US20160237455A1 (en) | 2013-09-27 | 2016-08-18 | Editas Medicine, Inc. | Crispr-related methods and compositions |
US20160237451A1 (en) | 2013-09-30 | 2016-08-18 | Regents Of The University Of Minnesota | Conferring resistance to geminiviruses in plants using crispr/cas systems |
CN105593375A (zh) | 2013-09-30 | 2016-05-18 | 加利福尼亚大学董事会 | 新趋化因子受体cxcr8的鉴别 |
US20160208214A1 (en) | 2013-10-02 | 2016-07-21 | Northeastern University | Methods and compositions for generation of developmentally-incompetent eggs in recipients of nuclear genetic transfer |
JP5774657B2 (ja) | 2013-10-04 | 2015-09-09 | 国立大学法人京都大学 | エレクトロポレーションを利用した哺乳類の遺伝子改変方法 |
CA2932581A1 (en) | 2013-10-07 | 2015-04-16 | Northeastern University | Methods and compositions for ex vivo generation of developmentally competent eggs from germ line cells using autologous cell systems |
DE102013111099B4 (de) | 2013-10-08 | 2023-11-30 | Eberhard Karls Universität Tübingen Medizinische Fakultät | Permanente Genkorrektur mittels nukleotidmodifizierter messenger RNA |
WO2015052231A2 (en) | 2013-10-08 | 2015-04-16 | Technical University Of Denmark | Multiplex editing system |
US20150098954A1 (en) | 2013-10-08 | 2015-04-09 | Elwha Llc | Compositions and Methods Related to CRISPR Targeting |
EP3055423B1 (en) | 2013-10-11 | 2019-12-25 | Cellectis | Method for detecting nucleic acid sequences of interest using talen protein |
WO2015057671A1 (en) | 2013-10-14 | 2015-04-23 | The Broad Institute, Inc. | Artificial transcription factors comprising a sliding domain and uses thereof |
JP6734774B2 (ja) | 2013-10-15 | 2020-08-05 | ザ スクリプス リサーチ インスティテュート | ペプチドキメラ抗原受容体t細胞スイッチおよびその使用 |
US20150238631A1 (en) | 2013-10-15 | 2015-08-27 | The California Institute For Biomedical Research | Chimeric antigen receptor t cell switches and uses thereof |
CN105899665B (zh) | 2013-10-17 | 2019-10-22 | 桑格摩生物科学股份有限公司 | 用于核酸酶介导的基因组工程改造的递送方法和组合物 |
CA2926078C (en) | 2013-10-17 | 2021-11-16 | Sangamo Biosciences, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells |
CA2927965A1 (en) | 2013-10-25 | 2015-04-30 | Cellectis | Design of rare-cutting endonucleases for efficient and specific targeting dna sequences comprising highly repetitive motives |
WO2015065964A1 (en) | 2013-10-28 | 2015-05-07 | The Broad Institute Inc. | Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof |
US10584358B2 (en) | 2013-10-30 | 2020-03-10 | North Carolina State University | Compositions and methods related to a type-II CRISPR-Cas system in Lactobacillus buchneri |
TWI721478B (zh) | 2013-11-04 | 2021-03-11 | 美商陶氏農業科學公司 | 最適玉米基因座(二) |
EP3065540B1 (en) | 2013-11-04 | 2021-12-15 | Corteva Agriscience LLC | Optimal maize loci |
EP3066202B1 (en) | 2013-11-04 | 2021-03-03 | Dow AgroSciences LLC | Optimal soybean loci |
CA2926534A1 (en) | 2013-11-04 | 2015-05-07 | Dow Agrosciences Llc | A universal donor system for gene targeting |
TWI659103B (zh) | 2013-11-04 | 2019-05-11 | 陶氏農業科學公司 | 最適大豆基因座(二) |
WO2015069682A2 (en) | 2013-11-05 | 2015-05-14 | President And Fellows Of Harvard College | Precise microbiota engineering at the cellular level |
WO2015070083A1 (en) | 2013-11-07 | 2015-05-14 | Editas Medicine,Inc. | CRISPR-RELATED METHODS AND COMPOSITIONS WITH GOVERNING gRNAS |
WO2015077058A2 (en) | 2013-11-08 | 2015-05-28 | The Broad Institute, Inc. | Compositions and methods for selecting a treatment for b-cell neoplasias |
US10369201B2 (en) | 2013-11-11 | 2019-08-06 | Sangamo Therapeutics, Inc. | Methods and compositions for treating Huntington's disease |
WO2015070193A1 (en) | 2013-11-11 | 2015-05-14 | Liu Oliver | Compositions and methods for targeted gene disruption in prokaryotes |
SI3068881T1 (sl) | 2013-11-13 | 2019-05-31 | Children's Medical Center Corporation | Z nukleazo posredovano uravnavanje izražanja genov |
CA2930590C (en) | 2013-11-15 | 2021-02-16 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Engineering neural stem cells using homologous recombination |
CN106103703A (zh) | 2013-11-18 | 2016-11-09 | 耶鲁大学 | 使用转座子的组合物和方法 |
JP2016537028A (ja) | 2013-11-18 | 2016-12-01 | クリスパー セラピューティクス アーゲー | Crispr−casシステムの材料及び方法 |
US9074199B1 (en) | 2013-11-19 | 2015-07-07 | President And Fellows Of Harvard College | Mutant Cas9 proteins |
WO2015075056A1 (en) | 2013-11-19 | 2015-05-28 | Thermo Fisher Scientific Baltics Uab | Programmable enzymes for isolation of specific dna fragments |
US10787684B2 (en) | 2013-11-19 | 2020-09-29 | President And Fellows Of Harvard College | Large gene excision and insertion |
WO2015075154A2 (en) | 2013-11-20 | 2015-05-28 | Fondazione Telethon | Artificial dna-binding proteins and uses thereof |
CN105765061A (zh) | 2013-11-22 | 2016-07-13 | 塞勒克提斯公司 | 工程化耐受化疗药物的t-细胞用于免疫治疗的方法 |
EP3071686B1 (en) | 2013-11-22 | 2020-07-22 | Cellectis SA | Method for generating batches of allogeneic t-cells with averaged potency |
CA2930973A1 (en) | 2013-11-22 | 2015-05-28 | Pal SAERTROM | C/ebp alpha short activating rna compositions and methods of use |
CN103642836A (zh) | 2013-11-26 | 2014-03-19 | 苏州同善生物科技有限公司 | 一种基于crispr基因敲除技术建立脆性x综合症灵长类动物模型的方法 |
CN103614415A (zh) | 2013-11-27 | 2014-03-05 | 苏州同善生物科技有限公司 | 一种基于crispr基因敲除技术建立肥胖症大鼠动物模型的方法 |
WO2015079056A1 (en) | 2013-11-28 | 2015-06-04 | Haplogen Genomics Gmbh | Somatic human cell line mutations |
JP6535684B2 (ja) | 2013-12-09 | 2019-06-26 | サンガモ セラピューティクス, インコーポレイテッド | ゲノム操作のための方法および組成物 |
RU2685914C1 (ru) | 2013-12-11 | 2019-04-23 | Регенерон Фармасьютикалс, Инк. | Способы и композиции для направленной модификации генома |
EP3835419A1 (en) | 2013-12-12 | 2021-06-16 | The Regents of The University of California | Methods and compositions for modifying a single stranded target nucleic acid |
US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
WO2015089427A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes |
CN111206032A (zh) | 2013-12-12 | 2020-05-29 | 布罗德研究所有限公司 | 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用 |
EP3080271B1 (en) | 2013-12-12 | 2020-02-12 | The Broad Institute, Inc. | Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems |
WO2015089473A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation |
CA2932472A1 (en) | 2013-12-12 | 2015-06-18 | Massachusetts Institute Of Technology | Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders |
CA2932475A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
WO2015089465A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders |
US20160312233A1 (en) | 2013-12-13 | 2016-10-27 | Cellectis | New method of selection of algal-transformed cells using nuclease |
CA2933134A1 (en) | 2013-12-13 | 2015-06-18 | Cellectis | Cas9 nuclease platform for microalgae genome engineering |
US20150191744A1 (en) | 2013-12-17 | 2015-07-09 | University Of Massachusetts | Cas9 effector-mediated regulation of transcription, differentiation and gene editing/labeling |
AU2014368982B2 (en) | 2013-12-19 | 2021-03-25 | Amyris, Inc. | Methods for genomic integration |
ES2818625T3 (es) | 2013-12-30 | 2021-04-13 | Univ Pittsburgh Commonwealth Sys Higher Education | Genes de fusión asociados con el cáncer de próstata progresivo |
CN103668472B (zh) | 2013-12-31 | 2014-12-24 | 北京大学 | 利用CRISPR/Cas9系统构建真核基因敲除文库的方法 |
US9963689B2 (en) | 2013-12-31 | 2018-05-08 | The Regents Of The University Of California | Cas9 crystals and methods of use thereof |
SG10201805815YA (en) | 2014-01-08 | 2018-08-30 | Harvard College | Rna-guided gene drives |
CN106459996A (zh) | 2014-01-14 | 2017-02-22 | Lam疗法公司 | 诱变方法 |
US10774338B2 (en) | 2014-01-16 | 2020-09-15 | The Regents Of The University Of California | Generation of heritable chimeric plant traits |
GB201400962D0 (en) | 2014-01-21 | 2014-03-05 | Kloehn Peter C | Screening for target-specific affinity binders using RNA interference |
WO2015109752A1 (en) | 2014-01-21 | 2015-07-30 | The Institute Of Genetics And Developmental Biology Chinese Academy Of Sciences | Modified plants |
CA2936976A1 (en) | 2014-01-24 | 2015-07-30 | Children's Medical Center Corporation | High-throughput mouse model for optimizing antibody affinities |
EP3097212A4 (en) | 2014-01-24 | 2017-10-04 | North Carolina State University | Methods and compositions for sequences guiding cas9 targeting |
WO2015113063A1 (en) | 2014-01-27 | 2015-07-30 | Georgia Tech Research Corporation | Methods and systems for identifying crispr/cas off-target sites |
CN104805078A (zh) | 2014-01-28 | 2015-07-29 | 北京大学 | 用于高效基因组编辑的rna分子的设计、合成及其应用 |
US9850525B2 (en) | 2014-01-29 | 2017-12-26 | Agilent Technologies, Inc. | CAS9-based isothermal method of detection of specific DNA sequence |
US20150291969A1 (en) | 2014-01-30 | 2015-10-15 | Chromatin, Inc. | Compositions for reduced lignin content in sorghum and improving cell wall digestibility, and methods of making the same |
US10233456B2 (en) | 2014-01-30 | 2019-03-19 | The Board Of Trustees Of The University Of Arkansas | Method, vectors, cells, seeds and kits for stacking genes into a single genomic site |
MX2016009771A (es) | 2014-01-31 | 2016-11-14 | Factor Bioscience Inc | Metodos y productos para la produccion y administracion de acido nucleico. |
GB201401707D0 (en) | 2014-01-31 | 2014-03-19 | Sec Dep For Health The | Adeno-associated viral vectors |
WO2015115903A1 (en) | 2014-02-03 | 2015-08-06 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Site-specific dna break-induced genome editing using engineered nucleases |
PL3102673T3 (pl) | 2014-02-03 | 2020-11-02 | Sangamo Therapeutics, Inc. | Sposoby i kompozycje do leczenia talasemii beta |
DK3102722T3 (da) | 2014-02-04 | 2020-11-16 | Jumpcode Genomics Inc | Genom fraktionering |
USRE48856E1 (en) | 2014-02-07 | 2021-12-21 | Vib Vzw | Inhibition of NEAT1 for treatment of solid tumors |
EP3690044B1 (en) | 2014-02-11 | 2024-01-10 | The Regents of the University of Colorado, a body corporate | Crispr enabled multiplexed genome engineering |
CA2939621C (en) | 2014-02-13 | 2019-10-15 | Takara Bio Usa, Inc. | Methods of depleting a target molecule from an initial collection of nucleic acids, and compositions and kits for practicing the same |
EP3105317B1 (en) | 2014-02-14 | 2018-09-19 | Cellectis | Cells for immunotherapy engineered for targeting antigen present both on immune cells and pathological cells |
SG11201606819QA (en) | 2014-02-18 | 2016-09-29 | Univ Duke | Compositions for the inactivation of virus replication and methods of making and using the same |
AU2015220765A1 (en) | 2014-02-20 | 2016-09-01 | Dsm Ip Assets B.V. | Phage insensitive Streptococcus thermophilus |
JP6665102B2 (ja) | 2014-02-21 | 2020-03-13 | セレクティスCellectis | 制御性t細胞をインサイチューで阻害するための方法 |
CA2939942A1 (en) | 2014-02-24 | 2015-08-27 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease-mediated targeted integration |
WO2015127428A1 (en) | 2014-02-24 | 2015-08-27 | Massachusetts Institute Of Technology | Methods for in vivo genome editing |
JP6521669B2 (ja) | 2014-02-25 | 2019-05-29 | 国立研究開発法人農業・食品産業技術総合研究機構 | 標的dnaに変異が導入された植物細胞、及びその製造方法 |
ES2898460T3 (es) | 2014-02-27 | 2022-03-07 | Monsanto Technology Llc | Composiciones y procedimientos para la modificación genómica dirigida al sitio |
CN103820454B (zh) | 2014-03-04 | 2016-03-30 | 上海金卫生物技术有限公司 | CRISPR-Cas9特异性敲除人PD1基因的方法以及用于特异性靶向PD1基因的sgRNA |
CN103820441B (zh) | 2014-03-04 | 2017-05-17 | 黄行许 | CRISPR‑Cas9特异性敲除人CTLA4基因的方法以及用于特异性靶向CTLA4基因的sgRNA |
CN111471675A (zh) | 2014-03-05 | 2020-07-31 | 国立大学法人神户大学 | 特异性转变靶向dna序列的核酸碱基的基因组序列的修饰方法、及其使用的分子复合体 |
EP3114227B1 (en) | 2014-03-05 | 2021-07-21 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
WO2015138510A1 (en) | 2014-03-10 | 2015-09-17 | Editas Medicine., Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
WO2015136001A1 (en) | 2014-03-11 | 2015-09-17 | Cellectis | Method for generating t-cells compatible for allogenic transplantation |
EP3116533B1 (en) | 2014-03-12 | 2020-08-12 | Precision Biosciences, Inc. | Dystrophin gene exon deletion using engineered nucleases |
WO2015138870A2 (en) | 2014-03-13 | 2015-09-17 | The Trustees Of The University Of Pennsylvania | Compositions and methods for targeted epigenetic modification |
WO2015138855A1 (en) | 2014-03-14 | 2015-09-17 | The Regents Of The University Of California | Vectors and methods for fungal genome engineering by crispr-cas9 |
AU2015229095B2 (en) | 2014-03-14 | 2022-01-27 | Cibus Europe B.V. | Methods and compositions for increasing efficiency of targeted gene modification using oligonucleotide-mediated gene repair |
WO2015143046A2 (en) | 2014-03-18 | 2015-09-24 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of zinc finger protein expression |
AU2015234204A1 (en) | 2014-03-20 | 2016-10-06 | Universite Laval | CRISPR-based methods and products for increasing frataxin levels and uses thereof |
CN106460009A (zh) | 2014-03-21 | 2017-02-22 | 小利兰·斯坦福大学托管委员会 | 无核酸酶的基因组编辑 |
CA2942347C (en) | 2014-03-24 | 2023-03-14 | IMMCO Diagnostics, Inc. | Improved anti-nuclear antibody detection and diagnostics for systemic and non-systemic autoimmune disorders |
WO2015148680A1 (en) | 2014-03-25 | 2015-10-01 | Ginkgo Bioworks, Inc. | Methods and genetic systems for cell engineering |
EP3129484A1 (en) | 2014-03-25 | 2017-02-15 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating hiv infection and aids |
WO2015148860A1 (en) | 2014-03-26 | 2015-10-01 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating beta-thalassemia |
CN106459951A (zh) | 2014-03-26 | 2017-02-22 | 马里兰大学派克分院 | 驯养的大型动物的受精卵中的靶向基因组编辑 |
US9609415B2 (en) | 2014-03-26 | 2017-03-28 | Bose Corporation | Headphones with cable management |
WO2015148863A2 (en) | 2014-03-26 | 2015-10-01 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
AU2015237746B2 (en) | 2014-03-28 | 2020-01-30 | Aposense Ltd. | Compounds and methods for trans-membrane delivery of molecules |
US9993563B2 (en) | 2014-03-28 | 2018-06-12 | Aposense Ltd. | Compounds and methods for trans-membrane delivery of molecules |
EP3498845B1 (en) | 2014-04-01 | 2022-06-22 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating herpes simplex virus type 1 (hsv-1) |
WO2015153760A2 (en) | 2014-04-01 | 2015-10-08 | Sangamo Biosciences, Inc. | Methods and compositions for prevention or treatment of a nervous system disorder |
WO2015153791A1 (en) | 2014-04-01 | 2015-10-08 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating herpes simplex virus type 2 (hsv-2) |
US10507232B2 (en) | 2014-04-02 | 2019-12-17 | University Of Florida Research Foundation, Incorporated | Materials and methods for the treatment of latent viral infection |
WO2015153780A1 (en) | 2014-04-02 | 2015-10-08 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
WO2015153940A1 (en) | 2014-04-03 | 2015-10-08 | Massachusetts Institute Of Technology | Methods and compositions for the production of guide rna |
CN103911376B (zh) | 2014-04-03 | 2017-02-15 | 黄行许 | CRISPR‑Cas9靶向敲除乙肝病毒cccDNA及其特异性sgRNA |
US11439712B2 (en) | 2014-04-08 | 2022-09-13 | North Carolina State University | Methods and compositions for RNA-directed repression of transcription using CRISPR-associated genes |
EP3556858A3 (en) | 2014-04-09 | 2020-01-22 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating cystic fibrosis |
US10253311B2 (en) | 2014-04-10 | 2019-04-09 | The Regents Of The University Of California | Methods and compositions for using argonaute to modify a single stranded target nucleic acid |
JP2017513472A (ja) | 2014-04-11 | 2017-06-01 | セレクティスCellectis | アルギニンおよび/またはトリプトファン枯渇微小環境に対して抵抗性を有する免疫細胞を作製するための方法 |
US20170037414A1 (en) | 2014-04-14 | 2017-02-09 | Nemesis Bioscience Ltd | Therapeutic |
CN103923911B (zh) | 2014-04-14 | 2016-06-08 | 上海金卫生物技术有限公司 | CRISPR-Cas9特异性敲除人CCR5基因的方法以及用于特异性靶向CCR5基因的sgRNA |
ES2962509T3 (es) | 2014-04-14 | 2024-03-19 | Maxcyte Inc | Métodos y composiciones para modificar ADN genómico |
GB201406970D0 (en) | 2014-04-17 | 2014-06-04 | Green Biologics Ltd | Targeted mutations |
GB201406968D0 (en) | 2014-04-17 | 2014-06-04 | Green Biologics Ltd | Deletion mutants |
EP3800248A3 (en) | 2014-04-18 | 2021-08-04 | Editas Medicine, Inc. | Crispr-cas-related methods, compositions and components for cancer immunotherapy |
CN105039399A (zh) | 2014-04-23 | 2015-11-11 | 复旦大学 | 多能干细胞-遗传性心肌病心肌细胞及其制备方法 |
WO2015164748A1 (en) | 2014-04-24 | 2015-10-29 | Sangamo Biosciences, Inc. | Engineered transcription activator like effector (tale) proteins |
KR20160145186A (ko) | 2014-04-24 | 2016-12-19 | 보드 오브 리전츠, 더 유니버시티 오브 텍사스 시스템 | 입양 세포 요법 생성물을 생성하기 위한 유도 만능 줄기 세포의 응용 |
KR101823661B1 (ko) | 2014-04-24 | 2018-01-30 | 기초과학연구원 | 마이크로 상동 기반의 유전자 녹아웃을 위한 뉴클레아제 표적 서열을 확인하는 방법 |
CN111647627A (zh) | 2014-04-28 | 2020-09-11 | 重组股份有限公司 | 多重基因编辑 |
WO2015168158A1 (en) | 2014-04-28 | 2015-11-05 | Fredy Altpeter | Targeted genome editing to modify lignin biosynthesis and cell wall composition |
US20150307889A1 (en) | 2014-04-28 | 2015-10-29 | Dow Agrosciences Llc | Haploid maize transformation |
WO2015167766A1 (en) | 2014-04-29 | 2015-11-05 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Ccr5 disruption of cells expressing anti-hiv chimeric antigen receptor (car) derived from broadly neutralizing antibodies |
WO2015165276A1 (zh) | 2014-04-30 | 2015-11-05 | 清华大学 | 利用tale转录抑制子在哺乳动物细胞中模块化构建合成基因线路的试剂盒 |
WO2015168404A1 (en) | 2014-04-30 | 2015-11-05 | Massachusetts Institute Of Technology | Toehold-gated guide rna for programmable cas9 circuitry with rna input |
US10563207B2 (en) | 2014-04-30 | 2020-02-18 | Tsinghua University | Modular construction of synthetic gene circuits in mammalian cells using TALE transcriptional repressors |
CN104178506B (zh) | 2014-04-30 | 2017-03-01 | 清华大学 | Taler蛋白通过空间位阻发挥转录抑制作用及其应用 |
WO2015168547A2 (en) | 2014-05-01 | 2015-11-05 | Andre Lieber | In vivo gene engineering with adenoviral vectors |
GB201407852D0 (en) | 2014-05-02 | 2014-06-18 | Iontas Ltd | Preparation of libraries od protein variants expressed in eukaryotic cells and use for selecting binding molecules |
WO2015171603A1 (en) | 2014-05-06 | 2015-11-12 | Two Blades Foundation | Methods for producing plants with enhanced resistance to oomycete pathogens |
WO2015171932A1 (en) | 2014-05-08 | 2015-11-12 | Sangamo Biosciences, Inc. | Methods and compositions for treating huntington's disease |
US10487336B2 (en) | 2014-05-09 | 2019-11-26 | The Regents Of The University Of California | Methods for selecting plants after genome editing |
EP3140403A4 (en) | 2014-05-09 | 2017-12-20 | Université Laval | Prevention and treatment of alzheimer's disease by genome editing using the crispr/cas system |
US20170266197A1 (en) | 2014-05-09 | 2017-09-21 | Indiana University Research And Technology Corporation | Methods and compositions for treating hepatitis b virus infections |
WO2015175642A2 (en) | 2014-05-13 | 2015-11-19 | Sangamo Biosciences, Inc. | Methods and compositions for prevention or treatment of a disease |
WO2015173436A1 (en) | 2014-05-16 | 2015-11-19 | Vrije Universiteit Brussel | Genetic correction of myotonic dystrophy type 1 |
CN103981212B (zh) | 2014-05-16 | 2016-06-01 | 安徽省农业科学院水稻研究所 | 将黄色颖壳的水稻品种的颖壳颜色改为褐色的育种方法 |
CN104017821B (zh) | 2014-05-16 | 2016-07-06 | 安徽省农业科学院水稻研究所 | 定向编辑颖壳颜色决定基因OsCHI创制褐壳水稻材料的方法 |
CN103981211B (zh) | 2014-05-16 | 2016-07-06 | 安徽省农业科学院水稻研究所 | 一种创制闭颖授粉水稻材料的育种方法 |
CN104004782B (zh) | 2014-05-16 | 2016-06-08 | 安徽省农业科学院水稻研究所 | 一种延长水稻生育期的育种方法 |
WO2015179540A1 (en) | 2014-05-20 | 2015-11-26 | Regents Of The University Of Minnesota | Method for editing a genetic sequence |
CA2852593A1 (en) | 2014-05-23 | 2015-11-23 | Universite Laval | Methods for producing dopaminergic neurons and uses thereof |
US10653123B2 (en) | 2014-05-27 | 2020-05-19 | Dana-Farber Cancer Institute, Inc. | Methods and compositions for perturbing gene expression in hematopoietic stem cell lineages in vivo |
WO2015183025A1 (ko) | 2014-05-28 | 2015-12-03 | 주식회사 툴젠 | 표적 특이적 뉴클레아제를 이용한 표적 dna의 민감한 검출 방법 |
AU2015266770A1 (en) | 2014-05-30 | 2016-12-08 | The Board Of Trustees Of The Leland Stanford Junior University | Compositions and methods of delivering treatments for latent viral infections |
US9970001B2 (en) | 2014-06-05 | 2018-05-15 | Sangamo Therapeutics, Inc. | Methods and compositions for nuclease design |
WO2015188191A1 (en) | 2014-06-06 | 2015-12-10 | Wong Wilson W | Dna recombinase circuits for logical control of gene expression |
CN104004778B (zh) | 2014-06-06 | 2016-03-02 | 重庆高圣生物医药有限责任公司 | 含有CRISPR/Cas9系统的靶向敲除载体及其腺病毒和应用 |
WO2015188135A1 (en) | 2014-06-06 | 2015-12-10 | The California Institute For Biomedical Research | Constant region antibody fusion proteins and compositions thereof |
CA2950178A1 (en) | 2014-06-06 | 2015-12-10 | The California Institute For Biomedical Research | Methods of constructing amino terminal immunoglobulin fusion proteins and compositions thereof |
KR102374379B1 (ko) | 2014-06-06 | 2022-03-17 | 리제너론 파마슈티칼스 인코포레이티드 | 표적화된 좌를 변형시키는 방법 및 조성물 |
WO2015188094A1 (en) | 2014-06-06 | 2015-12-10 | President And Fellows Of Harvard College | Methods for targeted modification of genomic dna |
US11274302B2 (en) | 2016-08-17 | 2022-03-15 | Diacarta Ltd | Specific synthetic chimeric Xenonucleic acid guide RNA; s(XNA-gRNA) for enhancing CRISPR mediated genome editing efficiency |
WO2015191693A2 (en) | 2014-06-10 | 2015-12-17 | Massachusetts Institute Of Technology | Method for gene editing |
ES2937642T3 (es) | 2014-06-11 | 2023-03-30 | Univ Duke | Composiciones y métodos para el control de flujo rápido y dinámico usando válvulas metabólicas sintéticas |
CA2951882A1 (en) | 2014-06-11 | 2015-12-17 | Tom E. HOWARD | Factor viii mutation repair and tolerance induction and related cdnas, compositions, methods and systems |
US11584936B2 (en) | 2014-06-12 | 2023-02-21 | King Abdullah University Of Science And Technology | Targeted viral-mediated plant genome editing using CRISPR /Cas9 |
WO2015191911A2 (en) | 2014-06-12 | 2015-12-17 | Clontech Laboratories, Inc. | Protein enriched microvesicles and methods of making and using the same |
JP6930834B2 (ja) | 2014-06-16 | 2021-09-01 | ザ・ジョンズ・ホプキンス・ユニバーシティー | H1プロモーターを用いるcrisprガイドrnaの発現のための組成物および方法 |
WO2015195547A1 (en) | 2014-06-16 | 2015-12-23 | University Of Washington | Methods for controlling stem cell potential and for gene editing in stem cells |
AU2015277180A1 (en) | 2014-06-17 | 2017-01-12 | Poseida Therapeutics, Inc. | A method for directing proteins to specific loci in the genome and uses thereof |
CA2952906A1 (en) | 2014-06-20 | 2015-12-23 | Cellectis | Potatoes with reduced granule-bound starch synthase |
KR102598819B1 (ko) | 2014-06-23 | 2023-11-03 | 더 제너럴 하스피탈 코포레이션 | 서열결정에 의해 평가된 DSB의 게놈 전체에 걸친 비편향된 확인 (GUIDE-Seq) |
EP3155099B1 (en) | 2014-06-23 | 2018-03-21 | Regeneron Pharmaceuticals, Inc. | Nuclease-mediated dna assembly |
WO2015200555A2 (en) | 2014-06-25 | 2015-12-30 | Caribou Biosciences, Inc. | Rna modification to engineer cas9 activity |
GB201411344D0 (en) | 2014-06-26 | 2014-08-13 | Univ Leicester | Cloning |
ES2901074T3 (es) | 2014-06-26 | 2022-03-21 | Regeneron Pharma | Métodos y composiciones para modificaciones genéticas objetivo y métodos de uso |
EP3162328B1 (en) | 2014-06-30 | 2022-01-19 | Kao Corporation | Adhesive sheet for cooling |
CN106662033B (zh) | 2014-06-30 | 2019-01-18 | 日产自动车株式会社 | 内燃机 |
US20180187172A1 (en) | 2014-07-01 | 2018-07-05 | Board Of Regents, The University Of Texas System | Regulated gene expression from viral vectors |
WO2016007604A1 (en) | 2014-07-09 | 2016-01-14 | Gen9, Inc. | Compositions and methods for site-directed dna nicking and cleaving |
EP2966170A1 (en) | 2014-07-10 | 2016-01-13 | Heinrich-Pette-Institut Leibniz-Institut für experimentelle Virologie-Stiftung bürgerlichen Rechts - | HBV inactivation |
MX2017000422A (es) | 2014-07-11 | 2017-05-01 | Du Pont | Composiciones y metodos para producir plantas resistentes al herbicida glifosato. |
BR112017000621B1 (pt) | 2014-07-11 | 2024-03-12 | Pioneer Hi-Bred International, Inc | Método para melhorar um traço agronômico de uma planta de milho ou de soja |
CN104109687A (zh) | 2014-07-14 | 2014-10-22 | 四川大学 | 运动发酵单胞菌CRISPR-Cas9系统的构建与应用 |
WO2016011080A2 (en) | 2014-07-14 | 2016-01-21 | The Regents Of The University Of California | Crispr/cas transcriptional modulation |
US10738278B2 (en) | 2014-07-15 | 2020-08-11 | Juno Therapeutics, Inc. | Engineered cells for adoptive cell therapy |
US9944933B2 (en) | 2014-07-17 | 2018-04-17 | Georgia Tech Research Corporation | Aptamer-guided gene targeting |
EP3193944B1 (en) | 2014-07-17 | 2021-04-07 | University of Pittsburgh - Of the Commonwealth System of Higher Education | Methods of treating cells containing fusion genes |
US20160053272A1 (en) | 2014-07-18 | 2016-02-25 | Whitehead Institute For Biomedical Research | Methods Of Modifying A Sequence Using CRISPR |
US20160053304A1 (en) | 2014-07-18 | 2016-02-25 | Whitehead Institute For Biomedical Research | Methods Of Depleting Target Sequences Using CRISPR |
US10975406B2 (en) | 2014-07-18 | 2021-04-13 | Massachusetts Institute Of Technology | Directed endonucleases for repeatable nucleic acid cleavage |
EP3172237A2 (en) | 2014-07-21 | 2017-05-31 | Novartis AG | Treatment of cancer using humanized anti-bcma chimeric antigen receptor |
CA3176503A1 (en) | 2014-07-21 | 2016-01-28 | Illumina, Inc | Polynucleotide enrichment using crispr-cas systems |
JP6653420B2 (ja) | 2014-07-22 | 2020-02-26 | パナソニックIpマネジメント株式会社 | 複合磁性材料とこれを用いたコイル部品ならびに複合磁性材料の製造方法 |
DK3172315T3 (da) | 2014-07-24 | 2020-12-21 | Dsm Ip Assets Bv | Fagresistente mælkesyrebakterier |
WO2016014794A1 (en) | 2014-07-25 | 2016-01-28 | Sangamo Biosciences, Inc. | Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells |
US9757420B2 (en) | 2014-07-25 | 2017-09-12 | Sangamo Therapeutics, Inc. | Gene editing for HIV gene therapy |
US20170211048A1 (en) | 2014-07-25 | 2017-07-27 | Boehringer Ingelheim International Gmbh | Enhanced reprogramming to ips cells |
EP3194600B1 (en) | 2014-07-26 | 2019-08-28 | Consiglio Nazionale Delle Ricerche | Compositions and methods for treatment of muscular dystrophy |
EP3177718B1 (en) | 2014-07-30 | 2022-03-16 | President and Fellows of Harvard College | Cas9 proteins including ligand-dependent inteins |
FR3024464A1 (fr) | 2014-07-30 | 2016-02-05 | Centre Nat Rech Scient | Ciblage de vecteurs integratifs non-viraux dans les sequences d'adn nucleolaires chez les eucaryotes |
WO2016019144A2 (en) | 2014-07-30 | 2016-02-04 | Sangamo Biosciences, Inc. | Gene correction of scid-related genes in hematopoietic stem and progenitor cells |
US9850521B2 (en) | 2014-08-01 | 2017-12-26 | Agilent Technologies, Inc. | In vitro assay buffer for Cas9 |
EP2982758A1 (en) | 2014-08-04 | 2016-02-10 | Centre Hospitalier Universitaire Vaudois (CHUV) | Genome editing for the treatment of huntington's disease |
US20160076093A1 (en) | 2014-08-04 | 2016-03-17 | University Of Washington | Multiplex homology-directed repair |
CN113789317B (zh) | 2014-08-06 | 2024-02-23 | 基因工具股份有限公司 | 使用空肠弯曲杆菌crispr/cas系统衍生的rna引导的工程化核酸酶的基因编辑 |
JP6598860B2 (ja) | 2014-08-06 | 2019-10-30 | カレッジ オブ メディシン ポチョン チャ ユニバーシティ インダストリー−アカデミック コーオペレイション ファウンデーション | Hlaをコードする遺伝子のヌクレアーゼ媒介編集により作製される免疫適合性細胞 |
WO2016022931A1 (en) | 2014-08-07 | 2016-02-11 | The Rockefeller University | Compositions and methods for transcription-based crispr-cas dna editing |
WO2016022866A1 (en) | 2014-08-07 | 2016-02-11 | Agilent Technologies, Inc. | Cis-blocked guide rna |
CA2959130A1 (en) | 2014-08-11 | 2016-02-18 | The Board Of Regents Of The University Of Texas System | Prevention of muscular dystrophy by crispr/cas9-mediated gene editing |
US10513711B2 (en) | 2014-08-13 | 2019-12-24 | Dupont Us Holding, Llc | Genetic targeting in non-conventional yeast using an RNA-guided endonuclease |
US11071289B2 (en) | 2014-08-14 | 2021-07-27 | Biocytogen Boston Corp | DNA knock-in system |
CN104178461B (zh) | 2014-08-14 | 2017-02-01 | 北京蛋白质组研究中心 | 携带cas9的重组腺病毒及其应用 |
US9879270B2 (en) | 2014-08-15 | 2018-01-30 | Wisconsin Alumni Research Foundation | Constructs and methods for genome editing and genetic engineering of fungi and protists |
WO2016028682A1 (en) | 2014-08-17 | 2016-02-25 | The Broad Institute Inc. | Genome editing using cas9 nickases |
AU2015305570C1 (en) | 2014-08-19 | 2020-07-23 | President And Fellows Of Harvard College | RNA-guided systems for probing and mapping of nucleic acids |
WO2016028887A1 (en) | 2014-08-19 | 2016-02-25 | Pacific Biosciences Of California, Inc. | Compositions and methods for enrichment of nucleic acids |
WO2016026444A1 (en) | 2014-08-20 | 2016-02-25 | Shanghai Institutes For Biological Sciences, Chinese Academy Of Sciences | Biomarker and therapeutic target for triple negative breast cancer |
CA2957938A1 (en) | 2014-08-25 | 2016-03-03 | GeneWeave Biosciences, Inc. | Non-replicative transduction particles and transduction particle-based reporter systems |
WO2016033230A1 (en) | 2014-08-26 | 2016-03-03 | The Regents Of The University Of California | Hypersensitive aba receptors |
ES2730378T3 (es) | 2014-08-27 | 2019-11-11 | Caribou Biosciences Inc | Procedimientos para incrementar la eficiencia de la modificación mediada por Cas9 |
US10450584B2 (en) | 2014-08-28 | 2019-10-22 | North Carolina State University | Cas9 proteins and guiding features for DNA targeting and genome editing |
US10570418B2 (en) | 2014-09-02 | 2020-02-25 | The Regents Of The University Of California | Methods and compositions for RNA-directed target DNA modification |
WO2016035044A1 (en) | 2014-09-05 | 2016-03-10 | Vilnius University | Programmable rna shredding by the type iii-a crispr-cas system of streptococcus thermophilus |
WO2016037157A2 (en) | 2014-09-05 | 2016-03-10 | The Johns Hopkins University | Targeting capn9/capns2 activity as a therapeutic strategy for the treatment of myofibroblast differentiation and associated pathologies |
WO2016040594A1 (en) | 2014-09-10 | 2016-03-17 | The Regents Of The University Of California | Reconstruction of ancestral cells by enzymatic recording |
CN108064129A (zh) | 2014-09-12 | 2018-05-22 | 纳幕尔杜邦公司 | 玉米和大豆中复合性状基因座的位点特异性整合位点的产生和使用方法 |
EP3878948A1 (en) | 2014-09-16 | 2021-09-15 | Sangamo Therapeutics, Inc. | Methods and compositions for nuclease-mediated genome engineering and correction in hematopoietic stem cells |
CN106715431A (zh) | 2014-09-16 | 2017-05-24 | 吉利德科学公司 | Toll样受体调节剂的固体形式 |
WO2016049251A1 (en) | 2014-09-24 | 2016-03-31 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes |
KR20230067694A (ko) | 2014-09-24 | 2023-05-16 | 시티 오브 호프 | 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법 |
WO2016049163A2 (en) | 2014-09-24 | 2016-03-31 | The Broad Institute Inc. | Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder |
WO2016049024A2 (en) | 2014-09-24 | 2016-03-31 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo |
WO2016049258A2 (en) | 2014-09-25 | 2016-03-31 | The Broad Institute Inc. | Functional screening with optimized functional crispr-cas systems |
WO2016046635A1 (en) | 2014-09-25 | 2016-03-31 | Institut Pasteur | Methods for characterizing human papillomavirus associated cervical lesions |
WO2016054225A1 (en) | 2014-09-30 | 2016-04-07 | Stc.Unm | Plasmid delivery in the treatment of cancer and other disease states |
EP3845655A1 (en) | 2014-10-01 | 2021-07-07 | The General Hospital Corporation | Methods for increasing efficiency of nuclease-induced homology-directed repair |
CA2963840A1 (en) | 2014-10-09 | 2016-04-14 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Long poly(a) plasmids and methods for introduction of long poly(a) sequences into the plasmid |
US9879283B2 (en) | 2014-10-09 | 2018-01-30 | Life Technologies Corporation | CRISPR oligonucleotides and gene editing |
US10583201B2 (en) | 2014-10-10 | 2020-03-10 | Massachusetts Eye And Ear Infirmary | Efficient delivery of therapeutic molecules in vitro and in vivo |
EP3204496A1 (en) | 2014-10-10 | 2017-08-16 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
WO2016061073A1 (en) | 2014-10-14 | 2016-04-21 | Memorial Sloan-Kettering Cancer Center | Composition and method for in vivo engineering of chromosomal rearrangements |
MX2017004890A (es) | 2014-10-15 | 2018-02-12 | Regeneron Pharma | Metodos y composiciones para la generación o el mantenimiento de células pluripotentes. |
CN107208086A (zh) | 2014-10-17 | 2017-09-26 | 霍华德休斯医学研究所 | 基因组探针 |
US10308947B2 (en) | 2014-10-17 | 2019-06-04 | The Penn State Research Foundation | Methods and compositions for multiplex RNA guided genome editing and other RNA technologies |
CN104342457A (zh) | 2014-10-17 | 2015-02-11 | 杭州师范大学 | 一种将外源基因定点整合到靶标基因的方法 |
BR112017008082A2 (pt) | 2014-10-20 | 2017-12-26 | Envirologix Inc | composições e métodos para detectar um vírus de rna |
WO2016069591A2 (en) | 2014-10-27 | 2016-05-06 | The Broad Institute Inc. | Compositions, methods and use of synthetic lethal screening |
WO2016069912A1 (en) | 2014-10-29 | 2016-05-06 | Massachusetts Eye And Ear Infirmary | Efficient delivery of therapeutic molecules in vitro and in vivo |
MA40880A (fr) | 2014-10-30 | 2017-09-05 | Temple Univ Of The Commonwealth | Éradication guidée par l'arn du virus jc humain et d'autres polyomavirus |
EP3212165B1 (en) | 2014-10-30 | 2024-02-28 | President and Fellows of Harvard College | Delivery of negatively charged proteins using cationic lipids |
TWI716367B (zh) | 2014-10-31 | 2021-01-21 | 麻省理工學院 | 用於常間回文重複序列叢集(crispr)之大量平行組合性基因學 |
EP3215168B1 (en) | 2014-10-31 | 2023-08-02 | The Trustees of the University of Pennsylvania | Altering gene expression in modified t cells and uses thereof |
US9816080B2 (en) | 2014-10-31 | 2017-11-14 | President And Fellows Of Harvard College | Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs) |
CN104404036B (zh) | 2014-11-03 | 2017-12-01 | 赛业(苏州)生物科技有限公司 | 基于CRISPR/Cas9技术的条件性基因敲除方法 |
CN104504304B (zh) | 2014-11-03 | 2017-08-25 | 深圳先进技术研究院 | 一种成簇的规律间隔的短回文重复序列识别方法及装置 |
US10435697B2 (en) | 2014-11-03 | 2019-10-08 | Nanyang Technological University | Recombinant expression system that senses pathogenic microorganisms |
EP3739047A1 (en) | 2014-11-04 | 2020-11-18 | National University Corporation Kobe University | Method for modifying genome sequence to introduce specific mutation to targeted dna sequence by base-removal reaction, and molecular complex used therein |
WO2016073559A1 (en) | 2014-11-05 | 2016-05-12 | The Regents Of The University Of California | Methods for autocatalytic genome editing and neutralizing autocatalytic genome editing |
CN107406838A (zh) | 2014-11-06 | 2017-11-28 | 纳幕尔杜邦公司 | Rna引导的内切核酸酶向细胞中的肽介导的递送 |
WO2016073990A2 (en) | 2014-11-07 | 2016-05-12 | Editas Medicine, Inc. | Methods for improving crispr/cas-mediated genome-editing |
CN114438174A (zh) | 2014-11-11 | 2022-05-06 | 伊鲁米那股份有限公司 | 使用crispr-cas系统的多核苷酸扩增 |
WO2016077273A1 (en) | 2014-11-11 | 2016-05-19 | Q Therapeutics, Inc. | Engineering mesenchymal stem cells using homologous recombination |
EP3219810B1 (en) | 2014-11-14 | 2022-01-05 | Institute for Basic Science | Method for detecting off-target site of genetic scissors in genome |
WO2016075662A2 (en) | 2014-11-15 | 2016-05-19 | Zumutor Biologics, Inc. | Dna-binding domain, non-fucosylated and partially fucosylated proteins, and methods thereof |
US11470826B2 (en) | 2014-11-17 | 2022-10-18 | National University Corporation Tokyo Medical And Dental University | Method of conveniently producing genetically modified non-human mammal with high efficiency |
CN107109422B (zh) | 2014-11-19 | 2021-08-13 | 基础科学研究院 | 使用由两个载体表达的拆分的Cas9的基因组编辑 |
US11319555B2 (en) | 2014-11-20 | 2022-05-03 | Duke University | Compositions, systems and methods for cell therapy |
US10227661B2 (en) | 2014-11-21 | 2019-03-12 | GeneWeave Biosciences, Inc. | Sequence-specific detection and phenotype determination |
NZ731962A (en) | 2014-11-21 | 2022-07-01 | Regeneron Pharma | Methods and compositions for targeted genetic modification using paired guide rnas |
WO2016086177A2 (en) | 2014-11-25 | 2016-06-02 | Drexel University | Compositions and methods for hiv quasi-species excision from hiv-1-infected patients |
WO2016084088A1 (en) | 2014-11-26 | 2016-06-02 | Ramot At Tel-Aviv University Ltd. | Targeted elimination of bacterial genes |
CA2968808A1 (en) | 2014-11-27 | 2016-06-02 | Danziger Innovations Ltd. | Nucleic acid constructs for genome editing |
GB201421096D0 (en) | 2014-11-27 | 2015-01-14 | Imp Innovations Ltd | Genome editing methods |
CN105695485B (zh) | 2014-11-27 | 2020-02-21 | 中国科学院上海生命科学研究院 | 一种用于丝状真菌Crispr-Cas系统的Cas9编码基因及其应用 |
WO2016082135A1 (zh) | 2014-11-27 | 2016-06-02 | 中国农业科学院北京畜牧兽医研究所 | 一种利用定点切割系统对猪h11位点定点插入的方法 |
US10479997B2 (en) | 2014-12-01 | 2019-11-19 | Novartis Ag | Compositions and methods for diagnosis and treatment of prostate cancer |
WO2016089866A1 (en) | 2014-12-01 | 2016-06-09 | President And Fellows Of Harvard College | Rna-guided systems for in vivo gene editing |
CN107250148B (zh) | 2014-12-03 | 2021-04-16 | 安捷伦科技有限公司 | 具有化学修饰的指导rna |
CN104450774A (zh) | 2014-12-04 | 2015-03-25 | 中国农业科学院作物科学研究所 | 一种大豆CRISPR/Cas9体系的构建及其在大豆基因修饰中的应用 |
WO2016090385A1 (en) | 2014-12-05 | 2016-06-09 | Applied Stemcell, Inc. | Site-directed crispr/recombinase compositions and methods of integrating transgenes |
CN104531704B (zh) | 2014-12-09 | 2019-05-21 | 中国农业大学 | 利用CRISPR-Cas9系统敲除动物FGF5基因的方法 |
CN104531705A (zh) | 2014-12-09 | 2015-04-22 | 中国农业大学 | 利用CRISPR-Cas9系统敲除动物myostatin基因的方法 |
WO2016094679A1 (en) | 2014-12-10 | 2016-06-16 | Regents Of The University Of Minnesota | Genetically modified cells, tissues, and organs for treating disease |
WO2016094872A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Dead guides for crispr transcription factors |
EP3889260A1 (en) | 2014-12-12 | 2021-10-06 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
CA2969464A1 (en) | 2014-12-12 | 2016-06-16 | Tod M. Woolf | Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides |
CN104480144B (zh) | 2014-12-12 | 2017-04-12 | 武汉大学 | 用于艾滋病基因治疗的CRISPR/Cas9重组慢病毒载体及其慢病毒 |
WO2016094880A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs) |
WO2016094874A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Escorted and functionalized guides for crispr-cas systems |
US20170362580A1 (en) | 2014-12-12 | 2017-12-21 | James Zhu | Methods and compositions for selectively eliminating cells of interest |
CN107667171A (zh) | 2014-12-16 | 2018-02-06 | 丹尼斯科美国公司 | 真菌基因组修饰系统及使用方法 |
AU2015362618B2 (en) | 2014-12-16 | 2022-01-27 | C3J Therapeutics, Inc. | Compositions of and methods for in vitro viral genome engineering |
CA2969384A1 (en) | 2014-12-17 | 2016-06-23 | Cellectis | Inhibitory chimeric antigen receptor (icar or n-car) expressing non-t cell transduction domain |
PL3234134T3 (pl) | 2014-12-17 | 2020-12-28 | Proqr Therapeutics Ii B.V. | Ukierunkowana edycja rna |
AU2015363113B2 (en) | 2014-12-17 | 2021-03-11 | E. I. Du Pont De Nemours And Company | Compositions and methods for efficient gene editing in E. coli using guide RNA/Cas endonuclease systems in combination with circular polynucleotide modification templates |
WO2016097751A1 (en) | 2014-12-18 | 2016-06-23 | The University Of Bath | Method of cas9 mediated genome engineering |
US9840702B2 (en) | 2014-12-18 | 2017-12-12 | Integrated Dna Technologies, Inc. | CRISPR-based compositions and methods of use |
WO2016100974A1 (en) | 2014-12-19 | 2016-06-23 | The Broad Institute Inc. | Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing |
CN104745626B (zh) | 2014-12-19 | 2018-05-01 | 中国航天员科研训练中心 | 一种条件性基因敲除动物模型的快速构建方法及应用 |
CA2971444A1 (en) | 2014-12-20 | 2016-06-23 | Arc Bio, Llc | Compositions and methods for targeted depletion, enrichment, and partitioning of nucleic acids using crispr/cas system proteins |
CN104560864B (zh) | 2014-12-22 | 2017-08-11 | 中国科学院微生物研究所 | 利用CRISPR‑Cas9系统构建的敲除IFN‑β基因的293T细胞系 |
US10190106B2 (en) | 2014-12-22 | 2019-01-29 | Univesity Of Massachusetts | Cas9-DNA targeting unit chimeras |
WO2016106239A1 (en) | 2014-12-23 | 2016-06-30 | The Regents Of The University Of California | Methods and compositions for nucleic acid integration |
WO2016106236A1 (en) | 2014-12-23 | 2016-06-30 | The Broad Institute Inc. | Rna-targeting system |
EP3237615B2 (en) | 2014-12-24 | 2023-07-26 | The Broad Institute, Inc. | Crispr having or associated with destabilization domains |
AU2015101792A4 (en) | 2014-12-24 | 2016-01-28 | Massachusetts Institute Of Technology | Engineering of systems, methods and optimized enzyme and guide scaffolds for sequence manipulation |
CA2968939A1 (en) | 2014-12-24 | 2016-06-30 | The Johns Hopkins University | Systems and methods for genome modification and regulation |
CN104651398A (zh) | 2014-12-24 | 2015-05-27 | 杭州师范大学 | 利用CRISPR-Cas9特异敲出microRNA基因家族的方法 |
JPWO2016104716A1 (ja) | 2014-12-26 | 2017-10-05 | 国立研究開発法人理化学研究所 | 遺伝子のノックアウト方法 |
WO2016109255A1 (en) | 2014-12-30 | 2016-07-07 | University Of South Florida | Methods and compositions for cloning into large vectors |
CN104498493B (zh) | 2014-12-30 | 2017-12-26 | 武汉大学 | CRISPR/Cas9特异性敲除乙型肝炎病毒的方法以及用于特异性靶向HBV DNA的gRNA |
WO2016108926A1 (en) | 2014-12-30 | 2016-07-07 | The Broad Institute Inc. | Crispr mediated in vivo modeling and genetic screening of tumor growth and metastasis |
WO2016109840A2 (en) | 2014-12-31 | 2016-07-07 | Synthetic Genomics, Inc. | Compositions and methods for high efficiency in vivo genome editing |
CN104651399B (zh) | 2014-12-31 | 2018-11-16 | 广西大学 | 一种利用CRISPR/Cas系统在猪胚胎细胞中实现基因敲除的方法 |
US11396665B2 (en) | 2015-01-06 | 2022-07-26 | Dsm Ip Assets B.V. | CRISPR-CAS system for a filamentous fungal host cell |
CN108064287A (zh) | 2015-01-06 | 2018-05-22 | 帝斯曼知识产权资产管理有限公司 | 用于解脂酵母宿主细胞的crispr-cas系统 |
EP3242949B1 (en) | 2015-01-06 | 2021-11-03 | DSM IP Assets B.V. | A crispr-cas system for a yeast host cell |
ES2842151T3 (es) | 2015-01-06 | 2021-07-13 | Univ Yonsei Iacf | Endonucleasa dirigida al gen del factor VIII de coagulación sanguínea y composición para el tratamiento de la hemofilia que comprende el mismo |
CN104651392B (zh) | 2015-01-06 | 2018-07-31 | 华南农业大学 | 一种利用CRISPR/Cas9系统定点突变P/TMS12-1获得温敏不育系的方法 |
WO2016112242A1 (en) | 2015-01-08 | 2016-07-14 | President And Fellows Of Harvard College | Split cas9 proteins |
CN104593422A (zh) | 2015-01-08 | 2015-05-06 | 中国农业大学 | 一种抗蓝耳病克隆猪的制备方法 |
EP3242938B1 (en) | 2015-01-09 | 2020-01-08 | Bio-Rad Laboratories, Inc. | Detection of genome editing |
WO2016114972A1 (en) | 2015-01-12 | 2016-07-21 | The Regents Of The University Of California | Heterodimeric cas9 and methods of use thereof |
JP7278027B2 (ja) | 2015-01-12 | 2023-05-19 | マサチューセッツ インスティテュート オブ テクノロジー | マイクロ流体送達による遺伝子編集 |
WO2016112963A1 (en) | 2015-01-13 | 2016-07-21 | Riboxx Gmbh | Delivery of biomolecules into cells |
MA41349A (fr) | 2015-01-14 | 2017-11-21 | Univ Temple | Éradication de l'herpès simplex de type i et d'autres virus de l'herpès associés guidée par arn |
CN107429263A (zh) | 2015-01-15 | 2017-12-01 | 斯坦福大学托管董事会 | 调控基因组编辑的方法 |
CN104611370A (zh) | 2015-01-16 | 2015-05-13 | 深圳市科晖瑞生物医药有限公司 | 一种剔除β2-微球蛋白基因片段的方法 |
AR103446A1 (es) | 2015-01-19 | 2017-05-10 | Inst Genetics & Dev Biolog Cas | Método para modificar con precisión una planta a través de la expresión transitoria de genes |
CN104725626B (zh) | 2015-01-22 | 2016-06-29 | 漳州亚邦化学有限公司 | 一种适用于人造石英石的不饱和树脂的制备方法 |
CN105821072A (zh) | 2015-01-23 | 2016-08-03 | 深圳华大基因研究院 | 用于DNA组装的CRISPR-Cas9系统及DNA组装方法 |
WO2016123071A1 (en) | 2015-01-26 | 2016-08-04 | Cold Spring Harbor Laboratory | Methods of identifying essential protein domains |
CN104561095B (zh) | 2015-01-27 | 2017-08-22 | 深圳市国创纳米抗体技术有限公司 | 一种能够生产人神经生长因子的转基因小鼠的制备方法 |
US10059940B2 (en) | 2015-01-27 | 2018-08-28 | Minghong Zhong | Chemically ligated RNAs for CRISPR/Cas9-lgRNA complexes as antiviral therapeutic agents |
NZ733702A (en) | 2015-01-28 | 2022-04-29 | Caribou Biosciences Inc | Crispr hybrid dna/rna polynucleotides and methods of use |
EP3250689B1 (en) | 2015-01-28 | 2020-11-04 | The Regents of The University of California | Methods and compositions for labeling a single-stranded target nucleic acid |
AU2016211118B2 (en) | 2015-01-29 | 2022-03-31 | Centre National De La Recherche Scientifique | Method for inducing targeted meiotic recombinations |
EP3250693B2 (en) | 2015-01-30 | 2023-12-20 | The Regents of The University of California | Protein delivery in primary hematopoietic cells |
BR112017016639A2 (pt) | 2015-02-02 | 2018-06-19 | Meiragtx Uk Ii Ltd | regulação da expressão genética pela modulação mediada de aptâmero de espalhamento alternativo |
CN104593418A (zh) | 2015-02-06 | 2015-05-06 | 中国医学科学院医学实验动物研究所 | 一种人源化大鼠药物评价动物模型建立的方法 |
WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
KR101584933B1 (ko) | 2015-02-10 | 2016-01-13 | 성균관대학교산학협력단 | 항생제 내성 억제용 재조합 벡터 및 이의 용도 |
WO2016130697A1 (en) | 2015-02-11 | 2016-08-18 | Memorial Sloan Kettering Cancer Center | Methods and kits for generating vectors that co-express multiple target molecules |
CN104726494B (zh) | 2015-02-12 | 2018-10-23 | 中国人民解放军第二军医大学 | CRISPR-Cas9技术构建染色体易位干细胞及动物模型的方法 |
CN104928321B (zh) | 2015-02-12 | 2018-06-01 | 中国科学院西北高原生物研究所 | 一种由Crispr/Cas9诱导的鳞片缺失斑马鱼模式及建立方法 |
EP3256170B1 (en) | 2015-02-13 | 2020-09-23 | University of Massachusetts | Compositions and methods for transient delivery of nucleases |
US20160244784A1 (en) | 2015-02-15 | 2016-08-25 | Massachusetts Institute Of Technology | Population-Hastened Assembly Genetic Engineering |
WO2016132122A1 (en) | 2015-02-17 | 2016-08-25 | University Of Edinburgh | Assay construct |
JP6354100B2 (ja) | 2015-02-19 | 2018-07-11 | 国立大学法人徳島大学 | Cas9 mRNAを哺乳動物の受精卵にエレクトロポレーションにより導入する方法 |
US20180245073A1 (en) | 2015-02-23 | 2018-08-30 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
US20180200387A1 (en) | 2015-02-23 | 2018-07-19 | Crispr Therapeutics Ag | Materials and methods for treatment of human genetic diseases including hemoglobinopathies |
AU2016225179C1 (en) | 2015-02-23 | 2022-11-03 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
CN107406858A (zh) | 2015-02-25 | 2017-11-28 | 先锋国际良种公司 | 用于指导rna/cas内切核酸酶复合物的调节型表达的组合物和方法 |
US20160244829A1 (en) | 2015-02-25 | 2016-08-25 | University-Industry Foundation, Yonsei University | Method for target dna enrichment using crispr system |
WO2016135507A1 (en) | 2015-02-27 | 2016-09-01 | University Of Edinburgh | Nucleic acid editing systems |
CN104805099B (zh) | 2015-03-02 | 2018-04-13 | 中国人民解放军第二军医大学 | 一种安全编码Cas9蛋白的核酸分子及其表达载体 |
EP3858990A1 (en) | 2015-03-03 | 2021-08-04 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
CN104651401B (zh) | 2015-03-05 | 2019-03-08 | 东华大学 | 一种mir-505双等位基因敲除的方法 |
CN104673816A (zh) | 2015-03-05 | 2015-06-03 | 广东医学院 | 一种pCr-NHEJ载体及其构建方法及其用于细菌基因定点敲除的应用 |
US20180271891A1 (en) | 2015-03-11 | 2018-09-27 | The Broad Institute Inc. | Selective treatment of prmt5 dependent cancer |
CN105969792A (zh) | 2015-03-12 | 2016-09-28 | 中国科学院遗传与发育生物学研究所 | 一种提高植物对入侵的dna 病毒的抵御能力的方法 |
GB201504223D0 (en) | 2015-03-12 | 2015-04-29 | Genome Res Ltd | Biallelic genetic modification |
EP3858992A1 (en) | 2015-03-13 | 2021-08-04 | The Jackson Laboratory | A three-component crispr/cas complex system and uses thereof |
AR103926A1 (es) | 2015-03-16 | 2017-06-14 | Inst Genetics & Dev Biolog Cas | Método para realizar modificaciones sitio dirigidas en el genoma de una planta usando materiales no heredables |
CN106032540B (zh) | 2015-03-16 | 2019-10-25 | 中国科学院上海生命科学研究院 | CRISPR/Cas9核酸内切酶体系的腺相关病毒载体构建及其用途 |
EP3271849B1 (en) | 2015-03-17 | 2021-09-08 | Bio-Rad Laboratories, Inc. | Detection of genome editing |
WO2016149484A2 (en) | 2015-03-17 | 2016-09-22 | Temple University Of The Commonwealth System Of Higher Education | Compositions and methods for specific reactivation of hiv latent reservoir |
EP3271461A1 (en) | 2015-03-20 | 2018-01-24 | Danmarks Tekniske Universitet | Crispr/cas9 based engineering of actinomycetal genomes |
MA41382A (fr) | 2015-03-20 | 2017-11-28 | Univ Temple | Édition génique basée sur le système crispr/endonucléase à induction par tat |
CN104726449A (zh) | 2015-03-23 | 2015-06-24 | 国家纳米科学中心 | 一种用于预防和/或治疗HIV的CRISPR-Cas9系统及其制备方法和用途 |
CN106148416B (zh) | 2015-03-24 | 2019-12-17 | 华东师范大学 | Cyp基因敲除大鼠的培育方法及其肝微粒体的制备方法 |
EP4019635A1 (en) | 2015-03-25 | 2022-06-29 | Editas Medicine, Inc. | Crispr/cas-related methods, compositions and components |
EP3274453B1 (en) | 2015-03-26 | 2021-01-27 | Editas Medicine, Inc. | Crispr/cas-mediated gene conversion |
CA2981509A1 (en) | 2015-03-30 | 2016-10-06 | The Board Of Regents Of The Nevada System Of Higher Educ. On Behalf Of The University Of Nevada, La | Compositions comprising talens and methods of treating hiv |
US20180080051A1 (en) | 2015-03-31 | 2018-03-22 | Exeligen Scientific, Inc. | Cas 9 retroviral integrase and cas 9 recombinase systems for targeted incorporation of a dna sequence into a genome of a cell or organism |
WO2016161380A1 (en) | 2015-04-01 | 2016-10-06 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy |
EP3300507A4 (en) | 2015-04-02 | 2019-03-13 | Agenovir Corporation | GENERIC ADMINISTRATION AND COMPOSITIONS |
CN106434737A (zh) | 2015-04-03 | 2017-02-22 | 内蒙古中科正标生物科技有限责任公司 | 基于CRISPR/Cas9技术的单子叶植物基因敲除载体及其应用 |
US20170166928A1 (en) | 2015-04-03 | 2017-06-15 | Whitehead Institute For Biomedical Research | Compositions And Methods For Genetically Modifying Yeast |
WO2016161446A1 (en) | 2015-04-03 | 2016-10-06 | Dana-Farber Cancer Institute, Inc. | Composition and methods of genome editing of b-cells |
KR102648489B1 (ko) | 2015-04-06 | 2024-03-15 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna |
US11214779B2 (en) | 2015-04-08 | 2022-01-04 | University of Pittsburgh—of the Commonwealth System of Higher Education | Activatable CRISPR/CAS9 for spatial and temporal control of genome editing |
US11390860B2 (en) | 2015-04-13 | 2022-07-19 | The University Of Tokyo | Set of polypeptides exhibiting nuclease activity or nickase activity with dependence on light or in presence of drug or suppressing or activating expression of target gene |
US10155938B2 (en) | 2015-04-14 | 2018-12-18 | City Of Hope | Coexpression of CAS9 and TREX2 for targeted mutagenesis |
GB201506509D0 (en) | 2015-04-16 | 2015-06-03 | Univ Wageningen | Nuclease-mediated genome editing |
US10738290B2 (en) | 2015-04-21 | 2020-08-11 | Novartis Ag | RNA-guided gene editing system and uses thereof |
CN104805118A (zh) | 2015-04-22 | 2015-07-29 | 扬州大学 | 一种苏禽黄鸡胚胎干细胞特定基因进行靶向敲除方法 |
CN104762321A (zh) | 2015-04-22 | 2015-07-08 | 东北林业大学 | 基于CRISPR/Cas9系统靶向敲除KHV基因的敲除载体构建方法及其crRNA原件 |
SG11201708653RA (en) | 2015-04-24 | 2017-11-29 | Editas Medicine Inc | Evaluation of cas9 molecule/guide rna molecule complexes |
CN107614012A (zh) | 2015-04-24 | 2018-01-19 | 加利福尼亚大学董事会 | 使用工程化的细胞检测、监测或治疗疾病或病况的系统及制备和使用它们的方法 |
US11268158B2 (en) | 2015-04-24 | 2022-03-08 | St. Jude Children's Research Hospital, Inc. | Assay for safety assessment of therapeutic genetic manipulations, gene therapy vectors and compounds |
WO2016174056A1 (en) | 2015-04-27 | 2016-11-03 | Genethon | Compositions and methods for the treatment of nucleotide repeat expansion disorders |
WO2016176191A1 (en) | 2015-04-27 | 2016-11-03 | The Trustees Of The University Of Pennsylvania | Dual aav vector system for crispr/cas9 mediated correction of human disease |
EP3087974A1 (en) | 2015-04-29 | 2016-11-02 | Rodos BioTarget GmbH | Targeted nanocarriers for targeted drug delivery of gene therapeutics |
EP3289080B1 (en) | 2015-04-30 | 2021-08-25 | The Trustees of Columbia University in the City of New York | Gene therapy for autosomal dominant diseases |
US20190002920A1 (en) | 2015-04-30 | 2019-01-03 | The Brigham And Women's Hospital, Inc. | Methods and kits for cloning-free genome editing |
EP3289076B1 (en) | 2015-05-01 | 2021-11-17 | Precision Biosciences, Inc. | Precise deletion of chromoscomal sequences in vivo |
US20160346359A1 (en) | 2015-05-01 | 2016-12-01 | Spark Therapeutics, Inc. | Adeno-associated Virus-Mediated CRISPR-Cas9 Treatment of Ocular Disease |
CN104894068A (zh) | 2015-05-04 | 2015-09-09 | 南京凯地生物科技有限公司 | 一种利用CRISPR/Cas9制备CAR-T细胞的方法 |
US11845928B2 (en) | 2015-05-04 | 2023-12-19 | Tsinghua University | Methods and kits for fragmenting DNA |
WO2016177682A1 (en) | 2015-05-06 | 2016-11-10 | Snipr Technologies Limited | Altering microbial populations & modifying microbiota |
GB2531454A (en) | 2016-01-10 | 2016-04-20 | Snipr Technologies Ltd | Recombinogenic nucleic acid strands in situ |
WO2016182893A1 (en) | 2015-05-08 | 2016-11-17 | Teh Broad Institute Inc. | Functional genomics using crispr-cas systems for saturating mutagenesis of non-coding elements, compositions, methods, libraries and applications thereof |
WO2016182917A1 (en) | 2015-05-08 | 2016-11-17 | Children's Medical Center Corporation | Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction |
JP7030522B2 (ja) | 2015-05-11 | 2022-03-07 | エディタス・メディシン、インコーポレイテッド | 幹細胞における遺伝子編集のための最適化crispr/cas9システムおよび方法 |
WO2016183236A1 (en) | 2015-05-11 | 2016-11-17 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating hiv infection and aids |
SG10202112057QA (en) | 2015-05-12 | 2021-12-30 | Sangamo Therapeutics Inc | Nuclease-mediated regulation of gene expression |
KR101785847B1 (ko) | 2015-05-12 | 2017-10-17 | 연세대학교 산학협력단 | 선형 이중가닥 DNA를 활용한 CRISPR/Cas9 시스템을 이용한 표적 유전체 교정 |
CN105886498A (zh) | 2015-05-13 | 2016-08-24 | 沈志荣 | CRISPR-Cas9特异性敲除人PCSK9基因的方法以及用于特异性靶向PCSK9基因的sgRNA |
US10920221B2 (en) | 2015-05-13 | 2021-02-16 | President And Fellows Of Harvard College | Methods of making and using guide RNA for use with Cas9 systems |
EP3294774A1 (en) | 2015-05-13 | 2018-03-21 | Zumutor Biologics, Inc. | Afucosylated protein, cell expressing said protein and associated methods |
CN108136047B (zh) | 2015-05-13 | 2022-12-13 | 西雅图儿童医院(Dba西雅图儿童研究所) | 提高原代细胞中基于核酸内切酶的基因编辑 |
WO2016183438A1 (en) | 2015-05-14 | 2016-11-17 | Massachusetts Institute Of Technology | Self-targeting genome editing system |
CN107614680A (zh) | 2015-05-14 | 2018-01-19 | 南加利福尼亚大学 | 利用重组核酸内切酶系统的最佳化基因编辑 |
CA2985506A1 (en) | 2015-05-15 | 2016-11-24 | Pioneer Hi-Bred International, Inc. | Guide rna/cas endonuclease systems |
US20180142236A1 (en) | 2015-05-15 | 2018-05-24 | Ge Healthcare Dharmacon, Inc. | Synthetic single guide rna for cas9-mediated gene editing |
BR112017024514A2 (pt) | 2015-05-16 | 2018-07-24 | Genzyme Corporation | edição genética de mutações intrônicas profundas |
WO2016185411A1 (en) | 2015-05-18 | 2016-11-24 | King Abdullah University Of Science And Technology | Method of inhibiting plant virus pathogen infections by crispr/cas9-mediated interference |
CN104846010B (zh) | 2015-05-18 | 2018-07-06 | 安徽省农业科学院水稻研究所 | 一种删除转基因水稻筛选标记基因的方法 |
EP3095870A1 (en) | 2015-05-19 | 2016-11-23 | Kws Saat Se | Methods for the in planta transformation of plants and manufacturing processes and products based and obtainable therefrom |
CN106011104B (zh) | 2015-05-21 | 2019-09-27 | 清华大学 | 利用拆分Cas系统进行基因编辑和表达调控方法 |
CN105518135B (zh) | 2015-05-22 | 2020-11-24 | 深圳市第二人民医院 | CRISPR-Cas9特异性敲除猪CMAH基因的方法及用于特异性靶向CMAH基因的sgRNA |
WO2016187904A1 (zh) | 2015-05-22 | 2016-12-01 | 深圳市第二人民医院 | CRISPR-Cas9特异性敲除猪CMAH基因的方法及用于特异性靶向CMAH基因的sgRNA |
WO2016187717A1 (en) | 2015-05-26 | 2016-12-01 | Exerkine Corporation | Exosomes useful for genome editing |
US20180148711A1 (en) | 2015-05-28 | 2018-05-31 | Coda Biotherapeutics, Inc. | Genome editing vectors |
CN104894075B (zh) | 2015-05-28 | 2019-08-06 | 华中农业大学 | CRISPR/Cas9和Cre/lox系统编辑伪狂犬病毒基因组制备疫苗方法和应用 |
CN105624146B (zh) | 2015-05-28 | 2019-02-15 | 中国科学院微生物研究所 | 基于CRISPR/Cas9和酿酒酵母细胞内源的同源重组的分子克隆方法 |
KR102451796B1 (ko) | 2015-05-29 | 2022-10-06 | 노쓰 캐롤라이나 스테이트 유니버시티 | 크리스퍼 핵산을 사용하여 박테리아, 고세균, 조류 및 효모를 스크리닝하는 방법 |
JP2018516983A (ja) | 2015-05-29 | 2018-06-28 | アジェノビア コーポレーション | ウイルス感染を処置するための組成物および方法 |
JP2018516596A (ja) | 2015-05-29 | 2018-06-28 | アジェノビア コーポレーション | 抗ウイルスの方法および組成物 |
US20160346362A1 (en) | 2015-05-29 | 2016-12-01 | Agenovir Corporation | Methods and compositions for treating cytomegalovirus infections |
EP3302556A4 (en) | 2015-05-29 | 2018-12-05 | Clark Atlanta University | Human cell lines mutant for zic2 |
US10117911B2 (en) | 2015-05-29 | 2018-11-06 | Agenovir Corporation | Compositions and methods to treat herpes simplex virus infections |
US20160346360A1 (en) | 2015-05-29 | 2016-12-01 | Agenovir Corporation | Compositions and methods for cell targeted hpv treatment |
CA3000182A1 (en) | 2015-05-29 | 2016-12-08 | Agenovir Corporation | Methods and compositions for treating cells for transplant |
EA037359B1 (ru) | 2015-06-01 | 2021-03-17 | Тэмпл Юниверсити - Оф Зе Коммонвэлс Систем Оф Хайе Эдьюкейшн | Способы и композиции для рнк-направляемого лечения инфекции, вызываемой вич |
CA2987684A1 (en) | 2015-06-01 | 2016-12-08 | The Hospital For Sick Children | Delivery of structurally diverse polypeptide cargo into mammalian cells by a bacterial toxin |
CN105112445B (zh) | 2015-06-02 | 2018-08-10 | 广州辉园苑医药科技有限公司 | 一种基于CRISPR-Cas9基因敲除技术的miR-205基因敲除试剂盒 |
WO2016196887A1 (en) | 2015-06-03 | 2016-12-08 | Board Of Regents Of The University Of Nebraska | Dna editing using single-stranded dna |
WO2016196655A1 (en) | 2015-06-03 | 2016-12-08 | The Regents Of The University Of California | Cas9 variants and methods of use thereof |
US20180245074A1 (en) | 2015-06-04 | 2018-08-30 | Protiva Biotherapeutics, Inc. | Treating hepatitis b virus infection using crispr |
US10626393B2 (en) | 2015-06-04 | 2020-04-21 | Arbutus Biopharma Corporation | Delivering CRISPR therapeutics with lipid nanoparticles |
US11279926B2 (en) | 2015-06-05 | 2022-03-22 | The Regents Of The University Of California | Methods and compositions for generating CRISPR/Cas guide RNAs |
CN105039339B (zh) | 2015-06-05 | 2017-12-19 | 新疆畜牧科学院生物技术研究所 | 一种以RNA介导的特异性敲除绵羊FecB基因的方法及其专用sgRNA |
CN116334142A (zh) | 2015-06-09 | 2023-06-27 | 爱迪塔斯医药公司 | 用于改善移植的crispr/cas相关方法和组合物 |
WO2016198500A1 (en) | 2015-06-10 | 2016-12-15 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and compositions for rna-guided treatment of human cytomegalovirus (hcmv) infection |
ES2807440T3 (es) | 2015-06-10 | 2021-02-23 | Firmenich & Cie | Procedimiento de identificación de compuestos de almizcle |
US20160362667A1 (en) | 2015-06-10 | 2016-12-15 | Caribou Biosciences, Inc. | CRISPR-Cas Compositions and Methods |
ES2961258T3 (es) | 2015-06-10 | 2024-03-11 | Firmenich & Cie | Líneas celulares para el cribado de receptores de aroma y olor |
CN106414740A (zh) | 2015-06-11 | 2017-02-15 | 深圳市第二人民医院 | CRISPR‑Cas9特异性敲除猪SLA‑3基因的方法及用于特异性靶向SLA‑3基因的sgRNA |
CN105492608B (zh) | 2015-06-11 | 2021-07-23 | 深圳市第二人民医院 | CRISPR-Cas9特异性敲除猪PDX1基因的方法及用于特异性靶向PDX1基因的sgRNA |
WO2016197360A1 (zh) | 2015-06-11 | 2016-12-15 | 深圳市第二人民医院 | CRISPR-Cas9特异性敲除猪GFRA1基因的方法及用于特异性靶向GFRA1基因的sgRNA |
WO2016197355A1 (zh) | 2015-06-11 | 2016-12-15 | 深圳市第二人民医院 | CRISPR-Cas9特异性敲除猪SALL1基因的方法及用于特异性靶向SALL1基因的sgRNA |
WO2016197361A1 (zh) | 2015-06-11 | 2016-12-15 | 深圳市第二人民医院 | CRISPR-Cas9特异性敲除猪GGTA1基因的方法及用于特异性靶向GGTA1基因的sgRNA |
CN105518134A (zh) | 2015-06-11 | 2016-04-20 | 深圳市第二人民医院 | CRISPR-Cas9特异性敲除猪SLA-2基因的方法及用于特异性靶向SLA-2基因的sgRNA |
CN105593367A (zh) | 2015-06-11 | 2016-05-18 | 深圳市第二人民医院 | CRISPR-Cas9特异性敲除猪SLA-1基因的方法及用于特异性靶向SLA-1基因的sgRNA |
WO2016197362A1 (zh) | 2015-06-11 | 2016-12-15 | 深圳市第二人民医院 | CRISPR-Cas9特异性敲除猪vWF基因的方法及用于特异性靶向vWF基因的sgRNA |
CN105518139B (zh) | 2015-06-11 | 2021-02-02 | 深圳市第二人民医院 | CRISPR-Cas9特异性敲除猪FGL2基因的方法及用于特异性靶向FGL2基因的sgRNA |
US20180187190A1 (en) | 2015-06-12 | 2018-07-05 | Erasmus University Medical Center Rotterdam | New crispr assays |
WO2016201138A1 (en) | 2015-06-12 | 2016-12-15 | The Regents Of The University Of California | Reporter cas9 variants and methods of use thereof |
GB201510296D0 (en) | 2015-06-12 | 2015-07-29 | Univ Wageningen | Thermostable CAS9 nucleases |
CA2983874C (en) | 2015-06-15 | 2022-06-21 | North Carolina State University | Methods and compositions for efficient delivery of nucleic acids and rna-based antimicrobials |
WO2016205728A1 (en) | 2015-06-17 | 2016-12-22 | Massachusetts Institute Of Technology | Crispr mediated recording of cellular events |
EP3310932B1 (en) | 2015-06-17 | 2023-08-30 | The UAB Research Foundation | Crispr/cas9 complex for genomic editing |
WO2016205623A1 (en) | 2015-06-17 | 2016-12-22 | North Carolina State University | Methods and compositions for genome editing in bacteria using crispr-cas9 systems |
AU2016278959A1 (en) | 2015-06-17 | 2018-01-18 | The Uab Research Foundation | CRISPR/Cas9 complex for introducing a functional polypeptide into cells of blood cell lineage |
WO2016205745A2 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Cell sorting |
WO2016205749A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Novel crispr enzymes and systems |
WO2016205759A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation |
EP3430134B1 (en) | 2015-06-18 | 2022-09-21 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
EP3800255A3 (en) | 2015-06-18 | 2021-06-23 | Robert D. Bowles | Rna-guided transcriptional regulation and methods of using the same for the treatment of back pain |
CN109536474A (zh) | 2015-06-18 | 2019-03-29 | 布罗德研究所有限公司 | 降低脱靶效应的crispr酶突变 |
US9957501B2 (en) | 2015-06-18 | 2018-05-01 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
US9790490B2 (en) | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
US11279928B2 (en) | 2015-06-29 | 2022-03-22 | Massachusetts Institute Of Technology | Compositions comprising nucleic acids and methods of using the same |
GB201511376D0 (en) | 2015-06-29 | 2015-08-12 | Ecolab Usa Inc | Process for the treatment of produced water from chemical enhanced oil recovery |
WO2017004261A1 (en) | 2015-06-29 | 2017-01-05 | Ionis Pharmaceuticals, Inc. | Modified crispr rna and modified single crispr rna and uses thereof |
CA2986314C (en) | 2015-06-30 | 2024-04-23 | Cellectis | Methods for improving functionality in nk cell by gene inactivation using specific endonuclease |
BR112017028201A2 (pt) | 2015-07-02 | 2018-08-28 | Univ Johns Hopkins | tratamentos com base em crisp/cas9 |
DK3320091T3 (da) | 2015-07-06 | 2021-02-01 | Dsm Ip Assets Bv | Guide-rna-samlingsvektor |
US20170009242A1 (en) | 2015-07-06 | 2017-01-12 | Whitehead Institute For Biomedical Research | CRISPR-Mediated Genome Engineering for Protein Depletion |
CN105132451B (zh) | 2015-07-08 | 2019-07-23 | 电子科技大学 | 一种CRISPR/Cas9单一转录单元定向修饰骨架载体及其应用 |
US20170014449A1 (en) | 2015-07-13 | 2017-01-19 | Elwha LLC, a limited liability company of the State of Delaware | Site-specific epigenetic editing |
CA2991301A1 (en) | 2015-07-13 | 2017-01-19 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
US20180200342A1 (en) | 2015-07-13 | 2018-07-19 | Institut Pasteur | Improving sequence-specific antimicrobials by blocking dna repair |
EP3323890A4 (en) | 2015-07-14 | 2019-01-30 | Fukuoka University | METHOD FOR INDUCING SITE SPECIFIC RNA MUTATIONS, TARGET EDITION GUIDING RNA-GUIDE USED IN THE METHOD, AND TARGET EDITING GUID-RNA TARGET RNA COMPLEX |
MA42895A (fr) | 2015-07-15 | 2018-05-23 | Juno Therapeutics Inc | Cellules modifiées pour thérapie cellulaire adoptive |
CN108291218B (zh) | 2015-07-15 | 2022-08-19 | 新泽西鲁特格斯州立大学 | 核酸酶非依赖性靶向基因编辑平台及其用途 |
US20170020922A1 (en) | 2015-07-16 | 2017-01-26 | Batu Biologics Inc. | Gene editing for immunological destruction of neoplasia |
WO2017015101A1 (en) | 2015-07-17 | 2017-01-26 | University Of Washington | Methods for maximizing the efficiency of targeted gene correction |
WO2017015015A1 (en) | 2015-07-17 | 2017-01-26 | Emory University | Crispr-associated protein from francisella and uses related thereto |
US10676735B2 (en) | 2015-07-22 | 2020-06-09 | Duke University | High-throughput screening of regulatory element function with epigenome editing technologies |
WO2017015545A1 (en) | 2015-07-22 | 2017-01-26 | President And Fellows Of Harvard College | Evolution of site-specific recombinases |
AU2016297167A1 (en) | 2015-07-23 | 2018-02-22 | Mayo Foundation For Medical Education And Research | Editing mitochondrial DNA |
CA2993474A1 (en) | 2015-07-25 | 2017-02-02 | Habib FROST | A system, device and a method for providing a therapy or a cure for cancer and other pathological states |
CN106399360A (zh) | 2015-07-27 | 2017-02-15 | 上海药明生物技术有限公司 | 基于crispr技术敲除fut8基因的方法 |
CN105063061B (zh) | 2015-07-28 | 2018-10-30 | 华南农业大学 | 一种水稻千粒重基因tgw6突变体及其制备方法与应用 |
DK3329001T3 (da) | 2015-07-28 | 2021-12-20 | Danisco Us Inc | Genomredigeringssystemer og anvendelsesfremgangsmåder |
CN106701808A (zh) | 2015-07-29 | 2017-05-24 | 深圳华大基因研究院 | Dna聚合酶i缺陷型菌株及其构建方法 |
US10612011B2 (en) | 2015-07-30 | 2020-04-07 | President And Fellows Of Harvard College | Evolution of TALENs |
US10166255B2 (en) | 2015-07-31 | 2019-01-01 | Regents Of The University Of Minnesota | Intracellular genomic transplant and methods of therapy |
US20200123533A1 (en) | 2015-07-31 | 2020-04-23 | The Trustees Of Columbia University In The City Of New York | High-throughput strategy for dissecting mammalian genetic interactions |
WO2017023974A1 (en) | 2015-08-03 | 2017-02-09 | President And Fellows Of Harvard College | Cas9 genome editing and transcriptional regulation |
WO2017024047A1 (en) | 2015-08-03 | 2017-02-09 | Emendobio Inc. | Compositions and methods for increasing nuclease induced recombination rate in cells |
WO2017024318A1 (en) | 2015-08-06 | 2017-02-09 | Dana-Farber Cancer Institute, Inc. | Targeted protein degradation to attenuate adoptive t-cell therapy associated adverse inflammatory responses |
CN104962523B (zh) | 2015-08-07 | 2018-05-25 | 苏州大学 | 一种测定非同源末端连接修复活性的方法 |
US9580727B1 (en) | 2015-08-07 | 2017-02-28 | Caribou Biosciences, Inc. | Compositions and methods of engineered CRISPR-Cas9 systems using split-nexus Cas9-associated polynucleotides |
BR112018002558A2 (pt) | 2015-08-07 | 2018-09-25 | Commw Scient Ind Res Org | método para produção de um animal compreendendo uma modificação genética na linhagem germinativa |
BR112018002600A2 (pt) | 2015-08-11 | 2018-10-23 | Cellectis | células para imunoterapia modificadas para direcionamento ao antígeno cd38 e para inativação do gene cd38 |
EP3334832A4 (en) | 2015-08-14 | 2019-06-26 | Institute Of Genetics And Developmental Biology, Chinese Academy Of Sciences | METHOD FOR OBTAINING GLYPHOSATE-RESISTANT RICE BY DIRECTED NUCLEOTIDE SUBSTITUTION |
CN105255937A (zh) | 2015-08-14 | 2016-01-20 | 西北农林科技大学 | 一种真核细胞III型启动子表达CRISPR sgRNA的方法及其应用 |
JP6986509B2 (ja) | 2015-08-19 | 2021-12-22 | アーク バイオ, エルエルシー | 核酸ガイド化ヌクレアーゼをベースとした系を使用する核酸の捕捉 |
CN105112519A (zh) | 2015-08-20 | 2015-12-02 | 郑州大学 | 一种基于crispr的大肠杆菌o157:h7菌株检测试剂盒及检测方法 |
CN107922953B (zh) | 2015-08-20 | 2022-03-04 | 应用干细胞有限公司 | 提高基因编辑效率的核酸酶 |
CN105177126B (zh) | 2015-08-21 | 2018-12-04 | 东华大学 | 一种利用荧光pcr技术对小鼠的分型鉴定方法 |
ES2929110T3 (es) | 2015-08-25 | 2022-11-24 | Univ Duke | Composiciones y métodos para mejorar la especificidad en ingeniería genética usando endonucleasas guiadas por ARN |
CN106480083B (zh) | 2015-08-26 | 2021-12-14 | 中国科学院分子植物科学卓越创新中心 | CRISPR/Cas9介导的大片段DNA拼接方法 |
US9512446B1 (en) | 2015-08-28 | 2016-12-06 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
WO2017040348A1 (en) | 2015-08-28 | 2017-03-09 | The General Hospital Corporation | Engineered crispr-cas9 nucleases |
US9926546B2 (en) | 2015-08-28 | 2018-03-27 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
WO2017040709A1 (en) | 2015-08-31 | 2017-03-09 | Caribou Biosciences, Inc. | Directed nucleic acid repair |
WO2017040511A1 (en) | 2015-08-31 | 2017-03-09 | Agilent Technologies, Inc. | Compounds and methods for crispr/cas-based genome editing by homologous recombination |
CN105087620B (zh) | 2015-08-31 | 2017-12-29 | 中国农业大学 | 一种过表达猪共刺激受体4‑1bb载体及其应用 |
AU2016316027B2 (en) | 2015-09-01 | 2022-04-07 | Dana-Farber Cancer Institute Inc. | Systems and methods for selection of gRNA targeting strands for Cas9 localization |
EP3344756A4 (en) | 2015-09-02 | 2019-09-04 | University of Massachusetts | DETECTION OF LOCI OF GENES COMPRISING MATRIX REPETITIONS OF CRISPR AND / OR POLYCHROMATIC MONO-GUIDE RIBONUCLEIC ACIDS |
WO2017040786A1 (en) | 2015-09-04 | 2017-03-09 | Massachusetts Institute Of Technology | Multilayer genetic safety kill circuits based on single cas9 protein and multiple engineered grna in mammalian cells |
CN105400810B (zh) | 2015-09-06 | 2019-05-07 | 吉林大学 | 采用敲除技术建立低磷性佝偻病模型的方法 |
WO2017044419A1 (en) | 2015-09-08 | 2017-03-16 | University Of Massachusetts | Dnase h activity of neisseria meningitidis cas9 |
CA2998087A1 (en) | 2015-09-09 | 2017-03-16 | National University Corporation Kobe University | Method for modifying genome sequence that specifically converts nucleobase of targeted dna sequence, and molecular complex used in said method |
ES2938623T3 (es) | 2015-09-09 | 2023-04-13 | Univ Kobe Nat Univ Corp | Método para convertir una secuencia del genoma de una bacteria gram-positiva mediante una conversión específica de una base de ácido nucleico de una secuencia de ADN seleccionada como diana y el complejo molecular utilizado en el mismo |
CN108291224A (zh) | 2015-09-10 | 2018-07-17 | 优美佳生物技术有限公司 | 用于治疗青光眼的方法和组合物 |
WO2017044776A1 (en) | 2015-09-10 | 2017-03-16 | Texas Tech University System | Single-guide rna (sgrna) with improved knockout efficiency |
CN105274144A (zh) | 2015-09-14 | 2016-01-27 | 徐又佳 | 通过CRISPR/Cas9技术得到敲除铁调素基因斑马鱼的制备方法 |
CN105210981B (zh) | 2015-09-15 | 2018-09-28 | 中国科学院生物物理研究所 | 建立可应用于人类疾病研究的雪貂模型的方法及其应用 |
US10301613B2 (en) | 2015-09-15 | 2019-05-28 | Arizona Board Of Regents On Behalf Of Arizona State University | Targeted remodeling of prokaryotic genomes using CRISPR-nickases |
CN105112422B (zh) | 2015-09-16 | 2019-11-08 | 中山大学 | 基因miR408和UCL在培育高产水稻中的应用 |
WO2017049129A2 (en) | 2015-09-18 | 2017-03-23 | President And Fellows Of Harvard College | Methods of making guide rna |
US10369232B2 (en) | 2015-09-21 | 2019-08-06 | Arcturus Therapeutics, Inc. | Allele selective gene editing and uses thereof |
CN105132427B (zh) | 2015-09-21 | 2019-01-08 | 新疆畜牧科学院生物技术研究所 | 一种以RNA介导的特异性敲除双基因获得基因编辑绵羊的方法及其专用sgRNA |
WO2017053312A1 (en) | 2015-09-21 | 2017-03-30 | The Regents Of The University Of California | Compositions and methods for target nucleic acid modification |
EP3352776A4 (en) | 2015-09-23 | 2019-03-13 | Sangamo Therapeutics, Inc. | HTT REPRESSORS AND USES THEREOF |
US11667911B2 (en) | 2015-09-24 | 2023-06-06 | Editas Medicine, Inc. | Use of exonucleases to improve CRISPR/CAS-mediated genome editing |
EP3337909B1 (en) | 2015-09-24 | 2020-03-18 | Sigma Aldrich Co. LLC | Methods and reagents for molecular proximity detection using rna-guided nucleic acid binding proteins |
WO2017064546A1 (en) | 2015-09-24 | 2017-04-20 | Crispr Therapeutics Ag | Novel family of rna-programmable endonucleases and their uses in genome editing and other applications |
KR101745863B1 (ko) | 2015-09-25 | 2017-06-12 | 전남대학교산학협력단 | Crispr/cas9 시스템을 이용한 프로히비틴2 유전자 제거용 시발체 |
WO2017053729A1 (en) | 2015-09-25 | 2017-03-30 | The Board Of Trustees Of The Leland Stanford Junior University | Nuclease-mediated genome editing of primary cells and enrichment thereof |
EP3353309A4 (en) | 2015-09-25 | 2019-04-10 | Tarveda Therapeutics, Inc. | COMPOSITIONS AND METHOD FOR GENERIC EDITING |
KR101795999B1 (ko) | 2015-09-25 | 2017-11-09 | 전남대학교산학협력단 | Crispr/cas9 시스템을 이용한 베타2-마이크로글로불린 유전자 제거용 시발체 |
EP3147363B1 (en) | 2015-09-26 | 2019-10-16 | B.R.A.I.N. Ag | Activation of taste receptor genes in mammalian cells using crispr-cas-9 |
EP3356521A4 (en) | 2015-09-28 | 2019-03-13 | Temple University - Of The Commonwealth System of Higher Education | METHOD AND COMPOSITIONS FOR RNA-TREATED TREATMENT OF HIV INFECTIONS |
CN105177038B (zh) | 2015-09-29 | 2018-08-24 | 中国科学院遗传与发育生物学研究所 | 一种高效定点编辑植物基因组的CRISPR/Cas9系统 |
WO2017058791A1 (en) | 2015-09-29 | 2017-04-06 | Agenovir Corporation | Compositions and methods for treatment of latent viral infections |
US20170087225A1 (en) | 2015-09-29 | 2017-03-30 | Agenovir Corporation | Compositions and methods for latent viral transcription regulation |
US20170088587A1 (en) | 2015-09-29 | 2017-03-30 | Agenovir Corporation | Antiviral fusion proteins and genes |
CN108601883A (zh) | 2015-09-29 | 2018-09-28 | 埃吉诺维亚公司 | 递送方法和组合物 |
CN105331627B (zh) | 2015-09-30 | 2019-04-02 | 华中农业大学 | 一种利用内源CRISPR-Cas系统进行原核生物基因组编辑的方法 |
EP3356520B1 (en) | 2015-10-02 | 2022-03-23 | The U.S.A. as represented by the Secretary, Department of Health and Human Services | Lentiviral protein delivery system for rna-guided genome editing |
CA3004710A1 (en) | 2015-10-06 | 2017-04-13 | The Children's Hospital Of Philadelphia | Compositions and methods for treating fragile x syndrome and related syndromes |
WO2017062754A1 (en) | 2015-10-07 | 2017-04-13 | New York University | Compositions and methods for enhancing crispr activity by polq inhibition |
WO2017062723A1 (en) | 2015-10-08 | 2017-04-13 | President And Fellows Of Harvard College | Multiplexed genome editing |
WO2017062886A1 (en) | 2015-10-08 | 2017-04-13 | Cellink Corporation | Battery interconnects |
AU2016335572B2 (en) | 2015-10-09 | 2022-12-08 | The Children's Hospital Of Philadelphia | Compositions and methods for treating Huntington's disease and related disorders |
WO2017062855A1 (en) | 2015-10-09 | 2017-04-13 | Monsanto Technology Llc | Novel rna-guided nucleases and uses thereof |
WO2017066175A1 (en) | 2015-10-12 | 2017-04-20 | E. I. Du Pont De Nemours And Company | Protected dna templates for gene modification and increased homologous recombination in cells and methods of use |
EP4089175A1 (en) | 2015-10-13 | 2022-11-16 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
WO2017066707A1 (en) | 2015-10-14 | 2017-04-20 | Life Technologies Corporation | Ribonucleoprotein transfection agents |
CN105400779A (zh) | 2015-10-15 | 2016-03-16 | 芜湖医诺生物技术有限公司 | 嗜热链球菌CRISPR-Cas9系统识别的人CCR5基因的靶序列和sgRNA及其应用 |
AU2016337408B2 (en) | 2015-10-16 | 2022-11-03 | Astrazeneca Ab | Inducible modification of a cell genome |
FR3042506B1 (fr) | 2015-10-16 | 2018-11-30 | IFP Energies Nouvelles | Outil genetique de transformation de bacteries clostridium |
CN108778343A (zh) | 2015-10-16 | 2018-11-09 | 天普大学-联邦高等教育系统 | 利用cpf1进行rna向导的基因编辑的方法和组合物 |
EP3365269A4 (en) | 2015-10-19 | 2019-06-19 | The Methodist Hospital | DISTRIBUTION, BY MEMBRANE DEFORMATION, FROM CRISPR-CAS9 TO DIFFICULT CELLS TO BE TRANSFERRED |
CN105331607A (zh) | 2015-10-19 | 2016-02-17 | 芜湖医诺生物技术有限公司 | 嗜热链球菌CRISPR-Cas9系统识别的人CCR5基因的靶序列和sgRNA及其应用 |
CN105316337A (zh) | 2015-10-20 | 2016-02-10 | 芜湖医诺生物技术有限公司 | 嗜热链球菌CRISPR-Cas9系统识别的人CXCR4基因的靶序列和sgRNA及其应用 |
CA2996326A1 (en) | 2015-10-20 | 2017-04-27 | Pioneer Hi-Bred International, Inc. | Methods and compositions for marker-free genome modification |
WO2017068077A1 (en) | 2015-10-20 | 2017-04-27 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods and products for genetic engineering |
CN105331608A (zh) | 2015-10-20 | 2016-02-17 | 芜湖医诺生物技术有限公司 | 脑膜炎双球菌CRISPR-Cas9系统识别的人CXCR4基因的靶序列和sgRNA及其应用 |
CN105316324A (zh) | 2015-10-20 | 2016-02-10 | 芜湖医诺生物技术有限公司 | 嗜热链球菌CRISPR-Cas9系统识别的人CXCR4基因的靶序列和sgRNA及其应用 |
CN105331609A (zh) | 2015-10-20 | 2016-02-17 | 芜湖医诺生物技术有限公司 | 脑膜炎双球菌CRISPR-Cas9系统识别的人CCR5基因的靶序列和sgRNA及其应用 |
AU2016341919A1 (en) | 2015-10-21 | 2018-04-19 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating hepatitis b virus |
CN116814590A (zh) | 2015-10-22 | 2023-09-29 | 布罗德研究所有限公司 | Vi-b型crispr酶和系统 |
CN105219799A (zh) | 2015-10-22 | 2016-01-06 | 天津吉诺沃生物科技有限公司 | 一种基于CRISPR/Cas系统的多年生黑麦草的育种方法 |
DK3350327T3 (en) | 2015-10-23 | 2019-01-21 | Caribou Biosciences Inc | CONSTRUCTED CRISPR CLASS-2-NUCLEIC ACID TARGETING-NUCLEIC ACID |
EP3365356B1 (en) | 2015-10-23 | 2023-06-28 | President and Fellows of Harvard College | Nucleobase editors and uses thereof |
EP3159407A1 (en) | 2015-10-23 | 2017-04-26 | Silence Therapeutics (London) Ltd | Guide rnas, methods and uses |
TW201715041A (zh) | 2015-10-26 | 2017-05-01 | 國立清華大學 | 細菌基因編輯方法 |
US9988637B2 (en) | 2015-10-26 | 2018-06-05 | National Tsing Hua Univeristy | Cas9 plasmid, genome editing system and method of Escherichia coli |
KR20180091821A (ko) | 2015-10-27 | 2018-08-16 | 리컴비네틱스 인코포레이티드 | 유전적 상보성에 의한 인간화 car t-세포 및 혈소판의 조작방법 |
US10280411B2 (en) | 2015-10-27 | 2019-05-07 | Pacific Biosciences of California, In.c | Methods, systems, and reagents for direct RNA sequencing |
WO2017072590A1 (en) | 2015-10-28 | 2017-05-04 | Crispr Therapeutics Ag | Materials and methods for treatment of duchenne muscular dystrophy |
US20180230489A1 (en) | 2015-10-28 | 2018-08-16 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
WO2017074526A1 (en) | 2015-10-28 | 2017-05-04 | Sangamo Biosciences, Inc. | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
WO2017074962A1 (en) | 2015-10-30 | 2017-05-04 | Brandeis University | Modified cas9 compositions and methods of use |
CN109312338B (zh) | 2015-10-30 | 2022-09-27 | 爱迪塔斯医药公司 | 治疗单纯疱疹病毒的crispr/cas相关方法及组合物 |
CN105238806B (zh) | 2015-11-02 | 2018-11-27 | 中国科学院天津工业生物技术研究所 | 一种用于微生物的CRISPR/Cas9基因编辑载体的构建及其应用 |
CN105316327B (zh) | 2015-11-03 | 2019-01-29 | 中国农业科学院作物科学研究所 | 小麦TaAGO4a基因CRISPR/Cas9载体及其应用 |
ES2953925T3 (es) | 2015-11-04 | 2023-11-17 | Fate Therapeutics Inc | Ingeniería genómica de células pluripotentes |
WO2017079428A1 (en) | 2015-11-04 | 2017-05-11 | President And Fellows Of Harvard College | Site specific germline modification |
SG11201803701YA (en) | 2015-11-04 | 2018-06-28 | Univ Pennsylvania | Methods and compositions for gene editing in hematopoietic stem cells |
EP3371300A1 (en) | 2015-11-05 | 2018-09-12 | Centro en Investigación Biomédica en Red | Process of gene-editing of cells isolated from a subject suffering from a metabolic disease affecting the erythroid lineage, cells obtained by said process and uses thereof |
GB2544270A (en) | 2015-11-05 | 2017-05-17 | Fundació Centre De Regulació Genòmica | Nucleic acids, peptides and methods |
WO2017078751A1 (en) | 2015-11-06 | 2017-05-11 | The Methodist Hospital | Micoluidic cell deomailiy assay for enabling rapid and efficient kinase screening via the crispr-cas9 system |
CA3004497A1 (en) | 2015-11-06 | 2017-05-11 | The Jackson Laboratory | Large genomic dna knock-in and uses thereof |
WO2017081097A1 (en) | 2015-11-09 | 2017-05-18 | Ifom Fondazione Istituto Firc Di Oncologia Molecolare | Crispr-cas sgrna library |
US11566052B2 (en) | 2015-11-11 | 2023-01-31 | Lonza Ltd. | CRISPR-associated (Cas) proteins with reduced immunogenicity |
WO2017083722A1 (en) | 2015-11-11 | 2017-05-18 | Greenberg Kenneth P | Crispr compositions and methods of using the same for gene therapy |
US10851367B2 (en) | 2015-11-12 | 2020-12-01 | Pfizer Inc. | Tissue-specific genome engineering using CRISPR-Cas9 |
US11306308B2 (en) | 2015-11-13 | 2022-04-19 | Massachusetts Institute Of Technology | High-throughput CRISPR-based library screening |
US20170191047A1 (en) | 2015-11-13 | 2017-07-06 | University Of Georgia Research Foundation, Inc. | Adenosine-specific rnase and methods of use |
KR101885901B1 (ko) | 2015-11-13 | 2018-08-07 | 기초과학연구원 | 5' 말단의 인산기가 제거된 rna를 포함하는 리보핵산단백질 전달용 조성물 |
JP7418957B2 (ja) | 2015-11-16 | 2024-01-22 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法 |
CN106893739A (zh) | 2015-11-17 | 2017-06-27 | 香港中文大学 | 用于靶向基因操作的新方法和系统 |
CN105602987A (zh) | 2015-11-23 | 2016-05-25 | 深圳市默赛尔生物医学科技发展有限公司 | 一种高效的dc细胞xbp1基因敲除方法 |
WO2017091630A1 (en) | 2015-11-23 | 2017-06-01 | The Regents Of The University Of California | Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9 |
US20170145438A1 (en) | 2015-11-24 | 2017-05-25 | University Of South Carolina | Viral Vectors for Gene Editing |
US10240145B2 (en) | 2015-11-25 | 2019-03-26 | The Board Of Trustees Of The Leland Stanford Junior University | CRISPR/Cas-mediated genome editing to treat EGFR-mutant lung cancer |
JP6500293B2 (ja) | 2015-11-25 | 2019-04-17 | 国立大学法人群馬大学 | Dnaメチル化編集用キットおよびdnaメチル化編集方法 |
US20180346940A1 (en) | 2015-11-27 | 2018-12-06 | The Regents Of The University Of California | Compositions and methods for the production of hydrocarbons, hydrogen and carbon monoxide using engineered azotobacter strains |
CN105505979A (zh) | 2015-11-28 | 2016-04-20 | 湖北大学 | 一种以CRISPR/Cas9基因编辑技术打靶Badh2基因获得香稻品系的方法 |
KR101906491B1 (ko) | 2015-11-30 | 2018-12-05 | 기초과학연구원 | F. novicida 유래 Cas9을 포함하는 유전체 교정용 조성물 |
CN106811479B (zh) | 2015-11-30 | 2019-10-25 | 中国农业科学院作物科学研究所 | 利用CRISPR/Cas9系统定点修饰ALS基因获得抗除草剂水稻的系统及其应用 |
CN105296518A (zh) | 2015-12-01 | 2016-02-03 | 中国农业大学 | 一种用于CRISPR/Cas9技术的同源臂载体构建方法 |
RU2634395C1 (ru) | 2015-12-01 | 2017-10-26 | Федеральное государственное автономное образовательное учреждение высшего профессионального образования "Балтийский Федеральный Университет имени Иммануила Канта" (БФУ им. И. Канта) | Генетическая конструкция на основе системы редактирования генома crispr/cas9, кодирующая нуклеазу cas9, специфически импортируемую в митохондрии клеток человека |
EP3383409A4 (en) | 2015-12-02 | 2019-10-02 | The Regents of The University of California | COMPOSITIONS AND METHODS FOR MODIFYING TARGET NUCLEIC ACID |
WO2017096237A1 (en) | 2015-12-02 | 2017-06-08 | Ceres, Inc. | Methods for genetic modification of plants |
WO2017093370A1 (en) | 2015-12-03 | 2017-06-08 | Technische Universität München | T-cell specific genome editing |
CN105779449B (zh) | 2015-12-04 | 2018-11-27 | 新疆农业大学 | 一种棉花启动子GbU6-5PS及应用 |
CN105779448B (zh) | 2015-12-04 | 2018-11-27 | 新疆农业大学 | 一种棉花启动子GbU6-7PS及应用 |
CN106845151B (zh) | 2015-12-07 | 2019-03-26 | 中国农业大学 | CRISPR-Cas9系统sgRNA作用靶点的筛选方法及装置 |
CN105462968B (zh) | 2015-12-07 | 2018-10-16 | 北京信生元生物医学科技有限公司 | 一种靶向apoCⅢ的CRISPR-Cas9系统及其应用 |
WO2017100431A2 (en) | 2015-12-09 | 2017-06-15 | Excision Biotherapeutics, Inc. | Gene editing methods and compositions for eliminating risk of jc virus activation and pml (progressive multifocal leukoencephalopathy) during immunosuppresive therapy |
EP3387134B1 (en) | 2015-12-11 | 2020-10-14 | Danisco US Inc. | Methods and compositions for enhanced nuclease-mediated genome modification and reduced off-target site effects |
CN105463003A (zh) | 2015-12-11 | 2016-04-06 | 扬州大学 | 一种消除卡那霉素耐药基因活性的重组载体及其构建方法 |
CN105296537A (zh) | 2015-12-12 | 2016-02-03 | 西南大学 | 一种基于睾丸内注射的基因定点编辑技术 |
WO2017105350A1 (en) | 2015-12-14 | 2017-06-22 | Cellresearch Corporation Pte Ltd | A method of generating a mammalian stem cell carrying a transgene, a mammalian stem cell generated by the method and pharmaceuticals uses of the mammalian stem cell |
CN105400773B (zh) | 2015-12-14 | 2018-06-26 | 同济大学 | 应用于大规模筛选癌症基因的CRISPR/Cas9富集测序方法 |
CN105463027A (zh) | 2015-12-17 | 2016-04-06 | 中国农业大学 | 一种高肌肉量及肥厚型心肌病模型克隆猪的制备方法 |
WO2017106616A1 (en) | 2015-12-17 | 2017-06-22 | The Regents Of The University Of Colorado, A Body Corporate | Varicella zoster virus encoding regulatable cas9 nuclease |
NO343153B1 (en) | 2015-12-17 | 2018-11-19 | Hydra Systems As | A method of assessing the integrity status of a barrier plug |
IL297018A (en) | 2015-12-18 | 2022-12-01 | Sangamo Therapeutics Inc | Directed cleavage of cell mhc receptor |
EP3390631B1 (en) | 2015-12-18 | 2020-04-08 | Danisco US Inc. | Methods and compositions for t-rna based guide rna expression |
WO2017106657A1 (en) | 2015-12-18 | 2017-06-22 | The Broad Institute Inc. | Novel crispr enzymes and systems |
JP2018537106A (ja) | 2015-12-18 | 2018-12-20 | ダニスコ・ユーエス・インク | ポリメラーゼII(Pol−II)ベースのガイドRNA発現のための方法および組成物 |
US11352631B2 (en) | 2015-12-18 | 2022-06-07 | Sangamo Therapeutics, Inc. | Targeted disruption of the T cell receptor |
US11118194B2 (en) | 2015-12-18 | 2021-09-14 | The Regents Of The University Of California | Modified site-directed modifying polypeptides and methods of use thereof |
WO2017106767A1 (en) | 2015-12-18 | 2017-06-22 | The Scripps Research Institute | Production of unnatural nucleotides using a crispr/cas9 system |
JP6700306B2 (ja) | 2015-12-18 | 2020-05-27 | 国立研究開発法人科学技術振興機構 | 受精前の卵細胞、受精卵、及び標的遺伝子の改変方法 |
WO2017112620A1 (en) | 2015-12-22 | 2017-06-29 | North Carolina State University | Methods and compositions for delivery of crispr based antimicrobials |
WO2017109134A1 (en) | 2015-12-22 | 2017-06-29 | Curevac Ag | Method for producing rna molecule compositions |
AU2016376191A1 (en) | 2015-12-23 | 2018-07-12 | Crispr Therapeutics Ag | Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration |
CN105543270A (zh) | 2015-12-24 | 2016-05-04 | 中国农业科学院作物科学研究所 | 双抗性CRISPR/Cas9载体及应用 |
CN105543266A (zh) | 2015-12-25 | 2016-05-04 | 安徽大学 | 一种维吉尼亚链霉菌IBL14中的CRISPR-Cas系统及应用其进行基因编辑的方法 |
CN105505976A (zh) | 2015-12-25 | 2016-04-20 | 安徽大学 | 一种维吉尼亚链霉菌ibl14产青霉素重组菌株的构建方法 |
JP2019500043A (ja) | 2015-12-28 | 2019-01-10 | ノバルティス アーゲー | 異常ヘモグロビン症の治療用組成物および方法 |
CN115216459A (zh) | 2015-12-29 | 2022-10-21 | 孟山都技术公司 | 新型crispr相关转座酶及其用途 |
CN105441451B (zh) | 2015-12-31 | 2019-03-22 | 暨南大学 | 一种特异靶向人ABCB1基因的sgRNA导向序列及应用 |
CN105567735A (zh) | 2016-01-05 | 2016-05-11 | 华东师范大学 | 一种凝血因子基因突变的定点修复载体系统及方法 |
EP3400296A1 (en) | 2016-01-08 | 2018-11-14 | Novozymes A/S | Genome editing in bacillus host cells |
CN105647922A (zh) | 2016-01-11 | 2016-06-08 | 中国人民解放军疾病预防控制所 | 基于一种新gRNA序列的CRISPR-Cas9系统在制备乙肝治疗药物中的应用 |
US11441146B2 (en) | 2016-01-11 | 2022-09-13 | Christiana Care Health Services, Inc. | Compositions and methods for improving homogeneity of DNA generated using a CRISPR/Cas9 cleavage system |
US11427837B2 (en) | 2016-01-12 | 2022-08-30 | The Regents Of The University Of California | Compositions and methods for enhanced genome editing |
CN108779471A (zh) | 2016-01-14 | 2018-11-09 | 孟菲斯肉类公司 | 用于延长体细胞在离体培养过程中的复制能力的方法 |
WO2017123910A1 (en) | 2016-01-14 | 2017-07-20 | The Brigham And Women's Hospital, Inc. | Genome editing for treating glioblastoma |
EP3402327A1 (en) | 2016-01-15 | 2018-11-21 | The Jackson Laboratory | Genetically modified non-human mammals by multi-cycle electroporation of cas9 protein |
CN105567734A (zh) | 2016-01-18 | 2016-05-11 | 丹弥优生物技术(湖北)有限公司 | 一种基因组dna序列精准编辑方法 |
WO2017126987A1 (ru) | 2016-01-18 | 2017-07-27 | Анатолий Викторович ЗАЗУЛЯ | Эритроциты для направленного транспорта лекарственного средства |
CN105567738A (zh) | 2016-01-18 | 2016-05-11 | 南开大学 | 使用基因组编辑技术CRISPR-Cas9诱导CCR5Δ32缺失的方法 |
US20190264186A1 (en) | 2016-01-22 | 2019-08-29 | The Broad Institute Inc. | Crystal structure of crispr cpf1 |
CA3011874A1 (en) | 2016-01-25 | 2017-08-03 | Excision Biotherapeutics, Inc. | Methods and compositions for rna-guided treatment of hiv infection |
CN105543228A (zh) | 2016-01-25 | 2016-05-04 | 宁夏农林科学院 | 一种快速将水稻转化为香稻的方法 |
WO2018106268A1 (en) | 2016-01-25 | 2018-06-14 | Excision Biotherapeutics | Rna guided eradication of human jc virus and other polyomaviruses |
CN105567689B (zh) | 2016-01-25 | 2019-04-09 | 重庆威斯腾生物医药科技有限责任公司 | CRISPR/Cas9靶向敲除人TCAB1基因及其特异性gRNA |
EP3199632A1 (en) | 2016-01-26 | 2017-08-02 | ACIB GmbH | Temperature-inducible crispr/cas system |
CN105567688A (zh) | 2016-01-27 | 2016-05-11 | 武汉大学 | 一种可用于艾滋病基因治疗的CRISPR/SaCas9系统 |
ES2949163T3 (es) | 2016-01-29 | 2023-09-26 | Univ Princeton | Inteínas divididas con actividad de corte y empalme excepcional |
JP6800171B2 (ja) | 2016-01-30 | 2020-12-16 | 株式会社ボナック | 人工単一ガイドrna及びその用途 |
CN107022562B (zh) | 2016-02-02 | 2020-07-17 | 中国种子集团有限公司 | 利用CRISPR/Cas9系统对玉米基因定点突变的方法 |
CN105647968B (zh) | 2016-02-02 | 2019-07-23 | 浙江大学 | 一种CRISPR/Cas9工作效率快速测试系统及其应用 |
CN105671083B (zh) | 2016-02-03 | 2017-09-29 | 安徽柯顿生物科技有限公司 | PD‑1基因重组病毒质粒及构建、重组逆转录病毒Lenti‑PD‑1‑Puro及包装与应用 |
WO2017136794A1 (en) | 2016-02-03 | 2017-08-10 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
WO2017136520A1 (en) | 2016-02-04 | 2017-08-10 | President And Fellows Of Harvard College | Mitochondrial genome editing and regulation |
US20190038780A1 (en) | 2016-02-05 | 2019-02-07 | Regents Of The University Of Minnesota | Vectors and system for modulating gene expression |
WO2017139264A1 (en) | 2016-02-09 | 2017-08-17 | President And Fellows Of Harvard College | Dna-guided gene editing and regulation |
EP3413908A4 (en) | 2016-02-11 | 2019-09-25 | The Regents of The University of California | METHOD AND COMPOSITIONS FOR MODIFYING A MUTANT DYSTROPHINE IN A CELL GENOM |
RU2016104674A (ru) | 2016-02-11 | 2017-08-16 | Анатолий Викторович Зазуля | Устройство модификации эритроцита с механизмом направленного транспорта лекарственного средства для функций генной терапии crispr/cas9 |
US9896696B2 (en) | 2016-02-15 | 2018-02-20 | Benson Hill Biosystems, Inc. | Compositions and methods for modifying genomes |
WO2017142835A1 (en) | 2016-02-15 | 2017-08-24 | Temple University - Of The Commonwealth System Of Higher Education | Excision of retroviral nucleic acid sequences |
CN105647962A (zh) | 2016-02-15 | 2016-06-08 | 浙江大学 | 运用CRISPR-Cas9系统敲除水稻MIRNA393b茎环序列的基因编辑方法 |
CN105647969B (zh) | 2016-02-16 | 2020-12-15 | 湖南师范大学 | 一种基因敲除选育stat1a基因缺失型斑马鱼的方法 |
CN105594664B (zh) | 2016-02-16 | 2018-10-02 | 湖南师范大学 | 一种基因敲除选育stat1a基因缺失型斑马鱼的方法 |
JP2019508037A (ja) | 2016-02-16 | 2019-03-28 | イェール ユニバーシティーYale Universit | 標的化遺伝子編集を増強するための組成物およびその使用方法 |
CN105624187A (zh) | 2016-02-17 | 2016-06-01 | 天津大学 | 酿酒酵母基因组定点突变的方法 |
US11326161B2 (en) | 2016-02-18 | 2022-05-10 | President And Fellows Of Harvard College | Methods and systems of molecular recording by CRISPR-Cas system |
CN105646719B (zh) | 2016-02-24 | 2019-12-20 | 无锡市妇幼保健院 | 一种高效定点转基因的工具及其应用 |
US20170247703A1 (en) | 2016-02-25 | 2017-08-31 | Agenovir Corporation | Antiviral nuclease methods |
EP3420077A4 (en) | 2016-02-25 | 2019-12-25 | Agenovir Corporation | VIRAL AND ONCOVIRAL NUCLEASE TREATMENT |
US20170246260A1 (en) | 2016-02-25 | 2017-08-31 | Agenovir Corporation | Modified antiviral nuclease |
WO2017147278A1 (en) | 2016-02-25 | 2017-08-31 | The Children's Medical Center Corporation | Customized class switch of immunoglobulin genes in lymphoma and hybridoma by crispr/cas9 technology |
KR20180110144A (ko) | 2016-02-26 | 2018-10-08 | 란자테크 뉴질랜드 리미티드 | C1-고정 박테리아에 대한 crispr/cas 시스템 |
US10538750B2 (en) | 2016-02-29 | 2020-01-21 | Agilent Technologies, Inc. | Methods and compositions for blocking off-target nucleic acids from cleavage by CRISPR proteins |
CN105671070B (zh) | 2016-03-03 | 2019-03-19 | 江南大学 | 一种用于枯草芽孢杆菌基因组编辑的CRISPRCas9系统及其构建方法 |
KR102587132B1 (ko) | 2016-03-04 | 2023-10-11 | 에디타스 메디신, 인코포레이티드 | 암 면역요법을 위한 crispr-cpf1-관련 방법, 조성물 및 구성성분 |
CN105821040B (zh) | 2016-03-09 | 2018-12-14 | 李旭 | 联合免疫基因抑制高危型HPV表达的sgRNA、基因敲除载体及其应用 |
CN105821039B (zh) | 2016-03-09 | 2020-02-07 | 李旭 | 联合免疫基因抑制HBV复制的特异性sgRNA、表达载体及其应用 |
CN107177591A (zh) | 2016-03-09 | 2017-09-19 | 北京大学 | 利用CRISPR技术编辑CCR5基因的sgRNA序列及其用途 |
CN105861547A (zh) | 2016-03-10 | 2016-08-17 | 黄捷 | 身份证号码永久嵌入基因组的方法 |
WO2017155717A1 (en) | 2016-03-11 | 2017-09-14 | Pioneer Hi-Bred International, Inc. | Novel cas9 systems and methods of use |
KR102532663B1 (ko) | 2016-03-14 | 2023-05-16 | 에디타스 메디신, 인코포레이티드 | 베타 이상헤모글로빈증의 치료를 위한 crispr/cas-관련 방법 및 조성물 |
WO2017160752A1 (en) | 2016-03-14 | 2017-09-21 | Intellia Therapeutics, Inc. | Methods and compositions for gene editing |
CN108885048B (zh) | 2016-03-15 | 2020-10-23 | 开利公司 | 冷藏销售柜 |
US11530394B2 (en) | 2016-03-15 | 2022-12-20 | University Of Massachusetts | Anti-CRISPR compounds and methods of use |
EP3219799A1 (en) | 2016-03-17 | 2017-09-20 | IMBA-Institut für Molekulare Biotechnologie GmbH | Conditional crispr sgrna expression |
US20200291370A1 (en) | 2016-03-18 | 2020-09-17 | President And Fellows Of Harvard College | Mutant Cas Proteins |
US11597924B2 (en) | 2016-03-25 | 2023-03-07 | Editas Medicine, Inc. | Genome editing systems comprising repair-modulating enzyme molecules and methods of their use |
WO2017165862A1 (en) | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
CN106047803A (zh) | 2016-03-28 | 2016-10-26 | 青岛市胶州中心医院 | CRISPR/Cas9靶向敲除兔BMP2基因的细胞模型及其应用 |
WO2017172644A2 (en) | 2016-03-28 | 2017-10-05 | The Charles Stark Draper Laboratory, Inc. | Bacteria identification and antibiotic susceptibility profiling device |
WO2017173004A1 (en) | 2016-03-30 | 2017-10-05 | Mikuni Takayasu | A method for in vivo precise genome editing |
KR102617874B1 (ko) | 2016-03-30 | 2023-12-22 | 인텔리아 테라퓨틱스, 인크. | Crispr/cas 성분을 위한 지질 나노입자 제제 |
GB2565461B (en) | 2016-03-31 | 2022-04-13 | Harvard College | Methods and compositions for the single tube preparation of sequencing libraries using Cas9 |
WO2017173092A1 (en) | 2016-03-31 | 2017-10-05 | The Regents Of The University Of California | Methods for genome editing in zygotes |
CN106167525B (zh) | 2016-04-01 | 2019-03-19 | 北京康明百奥新药研发有限公司 | 筛选超低岩藻糖细胞系的方法和应用 |
US10301619B2 (en) | 2016-04-01 | 2019-05-28 | New England Biolabs, Inc. | Compositions and methods relating to synthetic RNA polynucleotides created from synthetic DNA oligonucleotides |
CN109072191B (zh) | 2016-04-04 | 2024-03-22 | 苏黎世联邦理工学院 | 用于蛋白生产和文库产生的哺乳动物细胞系 |
US20190093091A1 (en) | 2016-04-06 | 2019-03-28 | Temple University - Of The Commonwealth System Of Higher Education | Compositions for eradicating flavivirus infections in subjects |
CN105802980A (zh) | 2016-04-08 | 2016-07-27 | 北京大学 | Gateway兼容性CRISPR/Cas9系统及其应用 |
CN106399306B (zh) | 2016-04-12 | 2019-11-05 | 西安交通大学第一附属医院 | 靶向人lncRNA-UCA1抑制膀胱癌的sgRNA、基因载体及其应用 |
US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
WO2017180694A1 (en) | 2016-04-13 | 2017-10-19 | Editas Medicine, Inc. | Cas9 fusion molecules gene editing systems, and methods of use thereof |
WO2017180711A1 (en) | 2016-04-13 | 2017-10-19 | Editas Medicine, Inc. | Grna fusion molecules, gene editing systems, and methods of use thereof |
EP3443085B1 (en) | 2016-04-14 | 2022-09-14 | BOCO Silicon Valley, Inc. | Genome editing of human neural stem cells using nucleases |
EP3442596A1 (en) | 2016-04-14 | 2019-02-20 | Université de Lausanne | Treatment and/or prevention of dna-triplet repeat diseases or disorders |
CN105821116A (zh) | 2016-04-15 | 2016-08-03 | 扬州大学 | 一种绵羊mstn基因定向敲除及其影响成肌分化的检测方法 |
US20200172935A1 (en) | 2016-04-16 | 2020-06-04 | Ohio State Innovation Foundation | Modified cpf1 mrna, modified guide rna, and uses thereof |
US20190134227A1 (en) | 2016-04-18 | 2019-05-09 | The Board Of Regents Of The University Of Texas System | Generation of genetically engineered animals by crispr/cas9 genome editing in spermatogonial stem cells |
US20190119678A1 (en) | 2016-04-18 | 2019-04-25 | Ruprecht-Karls-Universität Heidelberg | Means and methods for inactivating therapeutic dna in a cell |
CA3026112A1 (en) | 2016-04-19 | 2017-10-26 | The Broad Institute, Inc. | Cpf1 complexes with reduced indel activity |
CN106086062A (zh) | 2016-04-19 | 2016-11-09 | 上海市农业科学院 | 一种获得番茄基因组定点敲除突变体的方法 |
WO2017184768A1 (en) | 2016-04-19 | 2017-10-26 | The Broad Institute Inc. | Novel crispr enzymes and systems |
CA3026110A1 (en) | 2016-04-19 | 2017-11-02 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
CN105886616B (zh) | 2016-04-20 | 2020-08-07 | 广东省农业科学院农业生物基因研究中心 | 一种用于猪基因编辑的高效特异性sgRNA识别位点引导序列及其筛选方法 |
CN107304435A (zh) | 2016-04-22 | 2017-10-31 | 中国科学院青岛生物能源与过程研究所 | 一种Cas9/RNA系统及其应用 |
CN105821075B (zh) | 2016-04-22 | 2017-09-12 | 湖南农业大学 | 一种茶树咖啡因合成酶CRISPR/Cas9基因组编辑载体的构建方法 |
CN105861552B (zh) | 2016-04-25 | 2019-10-11 | 西北农林科技大学 | 一种T7 RNA聚合酶介导的CRISPR/Cas9基因编辑系统的构建方法 |
US11248216B2 (en) | 2016-04-25 | 2022-02-15 | The Regents Of The University Of California | Methods and compositions for genomic editing |
CN107326046A (zh) | 2016-04-28 | 2017-11-07 | 上海邦耀生物科技有限公司 | 一种提高外源基因同源重组效率的方法 |
KR20230038804A (ko) | 2016-04-29 | 2023-03-21 | 바스프 플랜트 사이언스 컴퍼니 게엠베하 | 표적 핵산의 변형을 위한 개선된 방법 |
CN105886534A (zh) | 2016-04-29 | 2016-08-24 | 苏州溯源精微生物科技有限公司 | 一种抑制肿瘤转移的方法 |
CN105821049B (zh) | 2016-04-29 | 2019-06-04 | 中国农业大学 | 一种Fbxo40基因敲除猪的制备方法 |
AU2017260714B2 (en) | 2016-05-01 | 2023-10-05 | Duane CHUNG | Harnessing heterologous and endogenous CRISPR-Cas machineries for efficient markerless genome editing in clostridium |
WO2017192512A2 (en) | 2016-05-02 | 2017-11-09 | Massachusetts Institute Of Technology | Amphiphilic nanoparticles for delivery of crispr based therapy |
CN105950639A (zh) | 2016-05-04 | 2016-09-21 | 广州美格生物科技有限公司 | 金黄色葡萄球菌CRISPR/Cas9系统的制备及其在构建小鼠模型中的应用 |
WO2017191210A1 (en) | 2016-05-04 | 2017-11-09 | Novozymes A/S | Genome editing by crispr-cas9 in filamentous fungal host cells |
WO2017190664A1 (zh) | 2016-05-05 | 2017-11-09 | 苏州吉玛基因股份有限公司 | 化学合成的crRNA和修饰crRNA在CRISPR/Cpf1基因编辑系统中的应用 |
CN105907785B (zh) | 2016-05-05 | 2020-02-07 | 苏州吉玛基因股份有限公司 | 化学合成的crRNA用于CRISPR/Cpf1系统在基因编辑中的应用 |
WO2017192172A1 (en) | 2016-05-05 | 2017-11-09 | Temple University - Of The Commonwealth System Of Higher Education | Rna guided eradication of varicella zoster virus |
WO2017193029A2 (en) | 2016-05-05 | 2017-11-09 | Duke University | Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy |
CN106244591A (zh) | 2016-08-23 | 2016-12-21 | 苏州吉玛基因股份有限公司 | 修饰crRNA在CRISPR/Cpf1基因编辑系统中的应用 |
CN105985985B (zh) | 2016-05-06 | 2019-12-31 | 苏州大学 | Crispr技术编辑并用igf优化的异体间充质干细胞的制备方法及在治疗心梗中应用 |
US20190300872A1 (en) | 2016-05-06 | 2019-10-03 | Tod M. Woolf | Improved Methods of Genome Editing with and without Programmable Nucleases |
US20190161743A1 (en) | 2016-05-09 | 2019-05-30 | President And Fellows Of Harvard College | Self-Targeting Guide RNAs in CRISPR System |
US20190225956A1 (en) | 2016-05-10 | 2019-07-25 | United States Government As Represented By The Department Of Veterans Affairs | Lentiviral delivery of crispr/cas constructs that cleave genes essential for hiv-1 infection and replication |
CN105861554B (zh) | 2016-05-10 | 2020-01-31 | 华南农业大学 | 一种基于对Rbmy基因进行编辑来实现动物性别控制的方法和应用 |
CN109152342A (zh) | 2016-05-12 | 2019-01-04 | 布赖恩.P.汉利 | Crispr和其他基因疗法安全递送到人类和动物中的大部分体细胞 |
CN107365786A (zh) | 2016-05-12 | 2017-11-21 | 中国科学院微生物研究所 | 一种将spacer序列克隆至CRISPR-Cas9系统中的方法及其应用 |
WO2017197238A1 (en) | 2016-05-12 | 2017-11-16 | President And Fellows Of Harvard College | Aav split cas9 genome editing and transcriptional regulation |
CN105907758B (zh) | 2016-05-18 | 2020-06-05 | 世翱(上海)生物医药科技有限公司 | CRISPR-Cas9引导序列及其引物、转基因表达载体及其构建方法 |
CN105838733A (zh) | 2016-05-18 | 2016-08-10 | 云南省农业科学院花卉研究所 | Cas9 介导的香石竹基因编辑载体和应用 |
CN106011171B (zh) | 2016-05-18 | 2019-10-11 | 西北农林科技大学 | 一种利用CRISPR/Cas9技术基于SSA修复的基因无缝编辑方法 |
MX2018014172A (es) | 2016-05-20 | 2019-08-22 | Regeneron Pharma | Métodos para romper la tolerancia inmunológica usando múltiples arn guías. |
CN106446600B (zh) | 2016-05-20 | 2019-10-18 | 同济大学 | 一种基于CRISPR/Cas9的sgRNA的设计方法 |
US20190300867A1 (en) | 2016-05-23 | 2019-10-03 | The Trustees Of Columbia University In The City Of New York | Bypassing the pam requirement of the crispr-cas system |
US20190201551A1 (en) | 2016-05-23 | 2019-07-04 | Washington University | Pulmonary targeted cas9/crispr for in vivo editing of disease genes |
CN105950560B (zh) | 2016-05-24 | 2019-07-23 | 苏州系统医学研究所 | 人源化pd-l1肿瘤细胞系及具有该细胞系的动物模型与应用 |
CN106011167B (zh) | 2016-05-27 | 2019-11-01 | 上海交通大学 | 雄性不育基因OsDPW2的应用及水稻育性恢复的方法 |
WO2017207589A1 (en) | 2016-06-01 | 2017-12-07 | Kws Saat Se | Hybrid nucleic acid sequences for genome engineering |
WO2017208247A1 (en) | 2016-06-02 | 2017-12-07 | Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. | Assay for the removal of methyl-cytosine residues from dna |
AU2017274145B2 (en) | 2016-06-02 | 2020-07-23 | Sigma-Aldrich Co Llc | Using programmable DNA binding proteins to enhance targeted genome modification |
JP2019517503A (ja) | 2016-06-03 | 2019-06-24 | テンプル ユニバーシティー オブ ザ コモンウェルス システム オブ ハイヤー エデュケーション | 遺伝子編集戦略によるhiv−1のネガティブフィードバック調節 |
US11140883B2 (en) | 2016-06-03 | 2021-10-12 | Auburn University | Gene editing of reproductive hormones to sterilize aquatic animals |
WO2017213898A2 (en) | 2016-06-07 | 2017-12-14 | Temple University - Of The Commonwealth System Of Higher Education | Rna guided compositions for preventing and treating hepatitis b virus infections |
CN106119275A (zh) | 2016-06-07 | 2016-11-16 | 湖北大学 | 基于CRISPR/Cas9技术将非糯性水稻株系改造成糯性株系的打靶载体和方法 |
US10767175B2 (en) | 2016-06-08 | 2020-09-08 | Agilent Technologies, Inc. | High specificity genome editing using chemically modified guide RNAs |
US11779657B2 (en) | 2016-06-10 | 2023-10-10 | City Of Hope | Compositions and methods for mitochondrial genome editing |
CN106086008B (zh) | 2016-06-10 | 2019-03-12 | 中国农业科学院植物保护研究所 | 烟粉虱MED隐种TRP基因的CRISPR/cas9系统及其应用 |
CN106434752A (zh) | 2016-06-14 | 2017-02-22 | 南通大学附属医院 | 敲除Wnt3a基因的过程及其验证方法 |
CN109312317A (zh) | 2016-06-14 | 2019-02-05 | 先锋国际良种公司 | Cpf1内切核酸酶用于植物基因组修饰的用途 |
CN106167821A (zh) | 2016-06-16 | 2016-11-30 | 郑州大学 | 一种金黄色葡萄球菌crispr位点检测试剂盒及检测方法 |
CN106167808A (zh) | 2016-06-16 | 2016-11-30 | 郑州大学 | 一种基于CRISPR/Cas9技术消除mecA质粒的方法 |
CN105950633B (zh) | 2016-06-16 | 2019-05-03 | 复旦大学 | 基因OsARF4在控制水稻粒长和千粒重中的应用 |
KR20190019168A (ko) | 2016-06-17 | 2019-02-26 | 더 브로드 인스티튜트, 인코퍼레이티드 | 제vi형 crispr 오솔로그 및 시스템 |
US20190330603A1 (en) | 2016-06-17 | 2019-10-31 | Genesis Technologies Limited | Crispr-cas system, materials and methods |
CN105950626B (zh) | 2016-06-17 | 2018-09-28 | 新疆畜牧科学院生物技术研究所 | 基于CRISPR/Cas9获得不同毛色绵羊的方法及靶向ASIP基因的sgRNA |
US20190323038A1 (en) | 2016-06-17 | 2019-10-24 | Montana State Univesity | Bidirectional targeting for genome editing |
CA3018430A1 (en) | 2016-06-20 | 2017-12-28 | Pioneer Hi-Bred International, Inc. | Novel cas systems and methods of use |
WO2017223107A1 (en) | 2016-06-20 | 2017-12-28 | Unity Biotechnology, Inc. | Genome modifying enzyme therapy for diseases modulated by senescent cells |
US20170362635A1 (en) | 2016-06-20 | 2017-12-21 | University Of Washington | Muscle-specific crispr/cas9 editing of genes |
KR102555713B1 (ko) | 2016-06-20 | 2023-07-14 | 키진 엔.브이. | 식물 세포에서의 표적된 dna 변경 방법 |
CN106148370A (zh) | 2016-06-21 | 2016-11-23 | 苏州瑞奇生物医药科技有限公司 | 肥胖症大鼠动物模型和构建方法 |
JP2019522481A (ja) | 2016-06-22 | 2019-08-15 | アイカーン スクール オブ メディシン アット マウント サイナイ | 自己切断リボザイムを利用したrnaのウイルス送達およびそのcrisprベースの適用 |
AU2017281497B2 (en) | 2016-06-22 | 2023-04-06 | Proqr Therapeutics Ii B.V. | Single-stranded RNA-editing oligonucleotides |
CN106047877B (zh) | 2016-06-24 | 2019-01-11 | 中山大学附属第一医院 | 一种靶向敲除FTO基因的sgRNA及CRISPR/Cas9慢病毒系统与应用 |
CN105925608A (zh) | 2016-06-24 | 2016-09-07 | 广西壮族自治区水牛研究所 | 一种利用CRISPR-Cas9靶向敲除ALK6基因的方法 |
CN106119283A (zh) | 2016-06-24 | 2016-11-16 | 广西壮族自治区水牛研究所 | 一种利用CRISPR‑Cas9靶向敲除MSTN基因的方法 |
EP3478840A1 (en) | 2016-06-29 | 2019-05-08 | Crispr Therapeutics AG | Compositions and methods for gene editing |
WO2018005873A1 (en) | 2016-06-29 | 2018-01-04 | The Broad Institute Inc. | Crispr-cas systems having destabilization domain |
CN106148286B (zh) | 2016-06-29 | 2019-10-29 | 牛刚 | 一种用于检测热原的细胞模型的构建方法和细胞模型及热原检测试剂盒 |
WO2018005691A1 (en) | 2016-06-29 | 2018-01-04 | The Regents Of The University Of California | Efficient genetic screening method |
US10927383B2 (en) | 2016-06-30 | 2021-02-23 | Ethris Gmbh | Cas9 mRNAs |
US20180004537A1 (en) | 2016-07-01 | 2018-01-04 | Microsoft Technology Licensing, Llc | Molecular State Machines |
CN109477130B (zh) | 2016-07-01 | 2022-08-30 | 微软技术许可有限责任公司 | 通过迭代dna编辑的存储 |
US10892034B2 (en) | 2016-07-01 | 2021-01-12 | Microsoft Technology Licensing, Llc | Use of homology direct repair to record timing of a molecular event |
KR20190039703A (ko) | 2016-07-05 | 2019-04-15 | 더 존스 홉킨스 유니버시티 | Crispr/cas9-기반 조성물 및 망막 변성을 치료하기 위한 방법 |
WO2018009520A1 (en) | 2016-07-06 | 2018-01-11 | Novozymes A/S | Improving a microorganism by crispr-inhibition |
CN106191057B (zh) | 2016-07-06 | 2018-12-25 | 中山大学 | 一种用于敲除人CYP2E1基因的sgRNA序列、CYP2E1基因缺失细胞株的构建方法及其应用 |
CN106051058A (zh) | 2016-07-07 | 2016-10-26 | 上海格昆机电科技有限公司 | 用于航天贮箱和粒子治疗仪的旋转机架及其传动机构 |
CN107586777A (zh) | 2016-07-08 | 2018-01-16 | 上海吉倍生物技术有限公司 | 人PDCD1基因sgRNA的用途及其相关药物 |
WO2018009822A1 (en) | 2016-07-08 | 2018-01-11 | Ohio State Innovation Foundation | Modified nucleic acids, hybrid guide rnas, and uses thereof |
CN106047930B (zh) | 2016-07-12 | 2020-05-19 | 北京百奥赛图基因生物技术有限公司 | 一种PS1基因条件性敲除flox大鼠的制备方法 |
KR102319845B1 (ko) | 2016-07-13 | 2021-11-01 | 디에스엠 아이피 어셋츠 비.브이. | 조류 숙주 세포에 대한 crispr-cas 시스템 |
WO2018013990A1 (en) | 2016-07-15 | 2018-01-18 | Zymergen Inc. | Scarless dna assembly and genome editing using crispr/cpf1 and dna ligase |
CN106191062B (zh) | 2016-07-18 | 2019-06-14 | 广东华南疫苗股份有限公司 | 一种tcr-/pd-1-双阴性t细胞及其构建方法 |
CN106190903B (zh) | 2016-07-18 | 2019-04-02 | 华中农业大学 | 鸭疫里氏杆菌Cas9基因缺失突变株及其应用 |
CN106191061B (zh) | 2016-07-18 | 2019-06-18 | 暨南大学 | 一种特异靶向人ABCG2基因的sgRNA导向序列及其应用 |
US20190151476A1 (en) | 2016-07-19 | 2019-05-23 | Duke University | Therapeutic applications of cpf1-based genome editing |
CN106434651B (zh) | 2016-07-19 | 2021-05-18 | 广西大学 | 根癌农杆菌和CRISPR-Cas9介导的基因定点插入失活方法及其应用 |
KR20230088522A (ko) | 2016-07-21 | 2023-06-19 | 맥스시티 인코포레이티드 | 게놈 dna를 변경하기 위한 방법 및 조성물 |
WO2018015444A1 (en) | 2016-07-22 | 2018-01-25 | Novozymes A/S | Crispr-cas9 genome editing with multiple guide rnas in filamentous fungi |
CN106191107B (zh) | 2016-07-22 | 2020-03-20 | 湖南农业大学 | 一种降低水稻籽粒落粒性的分子改良方法 |
CN106191064B (zh) | 2016-07-22 | 2019-06-07 | 中国农业大学 | 一种制备mc4r基因敲除猪的方法 |
EP3488001A1 (en) | 2016-07-25 | 2019-05-29 | Mayo Foundation for Medical Education and Research | Treating cancer |
CN106222193B (zh) | 2016-07-26 | 2019-09-20 | 浙江大学 | 一种重组载体及无转基因基因编辑植株的筛选方法 |
WO2018022634A1 (en) | 2016-07-26 | 2018-02-01 | The General Hospital Corporation | Variants of crispr from prevotella and francisella 1 (cpf1) |
WO2018018979A1 (zh) | 2016-07-26 | 2018-02-01 | 浙江大学 | 植物重组载体及无转基因成分的基因编辑植株的筛选方法 |
CN106086061A (zh) | 2016-07-27 | 2016-11-09 | 苏州泓迅生物科技有限公司 | 一种基于CRISPR‑Cas9系统的酿酒酵母基因组编辑载体及其应用 |
CN106191099A (zh) | 2016-07-27 | 2016-12-07 | 苏州泓迅生物科技有限公司 | 一种基于CRISPR‑Cas9系统的酿酒酵母基因组并行多重编辑载体及其应用 |
CN106434748A (zh) | 2016-07-29 | 2017-02-22 | 中国科学院重庆绿色智能技术研究院 | 一种热激诱导型 Cas9 酶转基因斑马鱼的研制及应用 |
CN106191114B (zh) | 2016-07-29 | 2020-02-11 | 中国科学院重庆绿色智能技术研究院 | 利用CRISPR-Cas9系统敲除鱼类MC4R基因的育种方法 |
CN106191124B (zh) | 2016-07-29 | 2019-10-11 | 中国科学院重庆绿色智能技术研究院 | 一种利用鱼卵保存液提高CRISPR-Cas9基因编辑和传代效率的鱼类育种方法 |
GB201613135D0 (en) | 2016-07-29 | 2016-09-14 | Medical Res Council | Genome editing |
CN106191113B (zh) | 2016-07-29 | 2020-01-14 | 中国农业大学 | 一种mc3r基因敲除猪的制备方法 |
CN106011150A (zh) | 2016-08-01 | 2016-10-12 | 云南纳博生物科技有限公司 | 一种水稻穗粒数Gn1a基因人工定点突变体及其应用 |
EP3491134B1 (en) | 2016-08-01 | 2023-10-11 | University of Pittsburgh - of The Commonwealth System of Higher Education | Human induced pluripotent stem cells for high efficiency genetic engineering |
CN106434688A (zh) | 2016-08-01 | 2017-02-22 | 云南纳博生物科技有限公司 | 一种水稻直立密穗dep1基因人工定点突变体及其应用 |
US11566263B2 (en) | 2016-08-02 | 2023-01-31 | Editas Medicine, Inc. | Compositions and methods for treating CEP290 associated disease |
WO2018025206A1 (en) | 2016-08-02 | 2018-02-08 | Kyoto University | Method for genome editing |
GB2568182A (en) | 2016-08-03 | 2019-05-08 | Harvard College | Adenosine nucleobase editors and uses thereof |
CN106282241A (zh) | 2016-08-05 | 2017-01-04 | 无锡市第二人民医院 | 通过CRISPR/Cas9得到敲除bmp2a基因的斑马鱼的方法 |
AU2017308889B2 (en) | 2016-08-09 | 2023-11-09 | President And Fellows Of Harvard College | Programmable Cas9-recombinase fusion proteins and uses thereof |
CN106222203A (zh) | 2016-08-10 | 2016-12-14 | 云南纳博生物科技有限公司 | 利用CRISPR/Cas技术获得家蚕丝素重链基因突变体及突变方法和应用 |
KR101710026B1 (ko) | 2016-08-10 | 2017-02-27 | 주식회사 무진메디 | Cas9 단백질 및 가이드 RNA의 혼성체를 함유하는 나노 리포좀 전달체 조성물 |
CN106172238B (zh) | 2016-08-12 | 2019-01-22 | 中南大学 | miR-124基因敲除小鼠动物模型的构建方法和应用 |
CN106222177B (zh) | 2016-08-13 | 2018-06-26 | 江苏集萃药康生物科技有限公司 | 一种靶向人STAT6的CRISPR-Cas9系统及其用于治疗过敏性疾病的应用 |
US11810649B2 (en) | 2016-08-17 | 2023-11-07 | The Broad Institute, Inc. | Methods for identifying novel gene editing elements |
US20210166783A1 (en) | 2016-08-17 | 2021-06-03 | The Broad Institute, Inc. | Methods for identifying class 2 crispr-cas systems |
WO2018035300A1 (en) | 2016-08-17 | 2018-02-22 | The Regents Of The University Of California | Split trans-complementing gene-drive system for suppressing aedes aegypti mosquitos |
WO2018035503A1 (en) | 2016-08-18 | 2018-02-22 | The Regents Of The University Of California | Crispr-cas genome engineering via a modular aav delivery system |
AU2017312132A1 (en) | 2016-08-19 | 2019-03-21 | Bluebird Bio, Inc. | Genome editing enhancers |
KR102594051B1 (ko) | 2016-08-20 | 2023-10-26 | 아벨리노 랩 유에스에이, 인크. | 단일 가이드 RNA, CRISPR/Cas9 시스템, 및 이의 사용방법 |
CN106191071B (zh) | 2016-08-22 | 2018-09-04 | 广州资生生物科技有限公司 | 一种CRISPR-Cas9系统及其用于治疗乳腺癌疾病的应用 |
CN106191116B (zh) | 2016-08-22 | 2019-10-08 | 西北农林科技大学 | 基于CRISPR/Cas9的外源基因敲入整合系统及其建立方法和应用 |
CN106086028B (zh) | 2016-08-23 | 2019-04-23 | 中国农业科学院作物科学研究所 | 一种通过基因组编辑提高水稻抗性淀粉含量的方法及其专用sgRNA |
CN106244555A (zh) | 2016-08-23 | 2016-12-21 | 广州医科大学附属第三医院 | 一种提高基因打靶的效率的方法及β‑球蛋白基因位点的碱基原位修复方法 |
CN106244609A (zh) | 2016-08-24 | 2016-12-21 | 浙江理工大学 | 一种调节pi3k‑akt信号通路的非编码基因的筛选系统及筛选方法 |
KR101856345B1 (ko) | 2016-08-24 | 2018-06-20 | 경상대학교산학협력단 | CRISPR/Cas9 시스템을 이용하여 APOBEC3H 및 APOBEC3CH 이중-넉아웃 고양이를 제조하는 방법 |
US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
IL264639B2 (en) | 2016-08-24 | 2024-01-01 | Sangamo Therapeutics Inc | Regulation of globulin gene expression using transgenic nucleases with zinc neurites |
CN106109417A (zh) | 2016-08-24 | 2016-11-16 | 李因传 | 一种肝细胞膜仿生脂质体药物载体、制作方法及其应用 |
BR112019003327A2 (pt) | 2016-08-24 | 2019-07-02 | Sangamo Therapeutics Inc | nucleases manipuladas de alvo específico |
CN106544357B (zh) | 2016-08-25 | 2018-08-21 | 湖南杂交水稻研究中心 | 一种培育镉低积累籼稻品种的方法 |
CN106318973B (zh) | 2016-08-26 | 2019-09-13 | 深圳市第二人民医院 | 一种基于CRISPR-Cas9的基因调控装置及基因调控方法 |
CN106350540A (zh) | 2016-08-26 | 2017-01-25 | 苏州系统医学研究所 | 一种由慢病毒介导的高效可诱导型CRISPR/Cas9基因敲除载体及其应用 |
CN107784200B (zh) | 2016-08-26 | 2020-11-06 | 深圳华大生命科学研究院 | 一种筛选新型CRISPR-Cas系统的方法和装置 |
CN106399367A (zh) | 2016-08-31 | 2017-02-15 | 深圳市卫光生物制品股份有限公司 | 提高crispr介导的同源重组效率的方法 |
CN106480097A (zh) | 2016-10-13 | 2017-03-08 | 南京凯地生物科技有限公司 | 利用CRISPR/Cas9技术敲除人PD‑1基因构建可靶向MSLN新型CAR‑T细胞的方法及其应用 |
CN106399375A (zh) | 2016-08-31 | 2017-02-15 | 南京凯地生物科技有限公司 | 利用CRISPR/Cas9敲除人PD‑1基因构建靶向CD19CAR‑T细胞的方法 |
CN107794272B (zh) | 2016-09-06 | 2021-10-12 | 中国科学院上海营养与健康研究所 | 一种高特异性的crispr基因组编辑体系 |
CN106399377A (zh) | 2016-09-07 | 2017-02-15 | 同济大学 | 一种基于CRISPR/Cas9高通量技术筛选药物靶点基因的方法 |
US20180105806A1 (en) | 2016-09-07 | 2018-04-19 | Massachusetts Institute Of Technology | Method for rna-guided endonuclease-based dna assembly |
CN106399311A (zh) | 2016-09-07 | 2017-02-15 | 同济大学 | 用于Chip‑seq全基因组结合谱的内源蛋白标记的方法 |
CN106367435B (zh) | 2016-09-07 | 2019-11-08 | 电子科技大学 | 一种水稻miRNA定向敲除的方法 |
EP3510151A4 (en) | 2016-09-09 | 2020-04-15 | The Board of Trustees of the Leland Stanford Junior University | HIGH-THROUGHPUT PRECISION GENE EDITING |
CN107574179B (zh) | 2016-09-09 | 2018-07-10 | 康码(上海)生物科技有限公司 | 一种为克鲁维酵母优化的CRISPR/Cas9高效基因编辑系统 |
US11485971B2 (en) | 2016-09-14 | 2022-11-01 | Yeda Research And Development Co. Ltd. | CRISP-seq, an integrated method for massively parallel single cell RNA-seq and CRISPR pooled screens |
CN106318934B (zh) | 2016-09-21 | 2020-06-05 | 上海交通大学 | 胡萝卜β(1,2)木糖转移酶的基因全序列及用于转染双子叶植物的CRISPR/CAS9的质粒构建 |
CN106957858A (zh) | 2016-09-23 | 2017-07-18 | 西北农林科技大学 | 一种利用CRISPR/Cas9系统共同敲除绵羊MSTN、ASIP、BCO2基因的方法 |
US20180127786A1 (en) | 2016-09-23 | 2018-05-10 | Casebia Therapeutics Limited Liability Partnership | Compositions and methods for gene editing |
WO2017216392A1 (en) | 2016-09-23 | 2017-12-21 | Dsm Ip Assets B.V. | A guide-rna expression system for a host cell |
EP3497215B1 (en) | 2016-09-28 | 2024-01-10 | Cellivery Therapeutics, Inc. | Cell-permeable (cp)-cas9 recombinant protein and uses thereof |
EP3523426A4 (en) | 2016-09-30 | 2020-01-22 | The Regents of The University of California | RNA GUIDED NUCLEIC ACID MODIFYING ENZYMES AND METHOD FOR USE THEREOF |
CN107881184B (zh) | 2016-09-30 | 2021-08-27 | 中国科学院分子植物科学卓越创新中心 | 一种基于Cpf1的DNA体外拼接方法 |
EP3518656A4 (en) | 2016-09-30 | 2020-09-30 | Monsanto Technology LLC | METHOD OF SELECTING TARGETS FOR SITE-SPECIFIC GENOME MODIFICATION IN PLANTS |
CN107880132B (zh) | 2016-09-30 | 2022-06-17 | 北京大学 | 一种融合蛋白及使用其进行同源重组的方法 |
GB2569734B (en) | 2016-09-30 | 2022-09-07 | Univ California | RNA-guided nucleic acid modifying enzymes and methods of use thereof |
CN106480027A (zh) | 2016-09-30 | 2017-03-08 | 重庆高圣生物医药有限责任公司 | CRISPR/Cas9 靶向敲除人PD‑1基因及其特异性gRNA |
EP3518981A4 (en) | 2016-10-03 | 2020-06-10 | President and Fellows of Harvard College | DELIVERING THERAPEUTIC RNAS VIA ARRDC1-MEDIATED MICROVESICLES |
WO2018067846A1 (en) | 2016-10-05 | 2018-04-12 | President And Fellows Of Harvard College | Methods of crispr mediated genome modulation in v. natriegens |
US10669539B2 (en) | 2016-10-06 | 2020-06-02 | Pioneer Biolabs, Llc | Methods and compositions for generating CRISPR guide RNA libraries |
CN110462034A (zh) | 2016-10-07 | 2019-11-15 | 综合Dna技术公司 | 化脓链球菌cas9突变基因和由其编码的多肽 |
CN106479985A (zh) | 2016-10-09 | 2017-03-08 | 上海吉玛制药技术有限公司 | 病毒介导的Cpf1蛋白在CRISPR/Cpf1基因编辑系统中的应用 |
WO2018071623A2 (en) | 2016-10-12 | 2018-04-19 | Temple University - Of The Commonwealth System Of Higher Education | Combination therapies for eradicating flavivirus infections in subjects |
IT201600102542A1 (it) | 2016-10-12 | 2018-04-12 | Univ Degli Studi Di Trento | Plasmide e sistema lentivirale contenente un circuito autolimitante della Cas9 che ne incrementa la sicurezza. |
CN106434663A (zh) | 2016-10-12 | 2017-02-22 | 遵义医学院 | CRISPR/Cas9靶向敲除人ezrin基因增强子关键区的方法及其特异性gRNA |
US20190330620A1 (en) | 2016-10-14 | 2019-10-31 | Emendobio Inc. | Rna compositions for genome editing |
AU2017341926B2 (en) | 2016-10-14 | 2022-06-30 | The General Hospital Corporation | Epigenetically regulated site-specific nucleases |
KR102622411B1 (ko) | 2016-10-14 | 2024-01-10 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
CN106434782B (zh) | 2016-10-14 | 2020-01-10 | 南京工业大学 | 一种产顺式-4-羟脯氨酸的方法 |
US11840694B2 (en) | 2016-10-17 | 2023-12-12 | Nanyang Technological University | Truncated CRISPR-Cas proteins for DNA targeting |
US10640810B2 (en) | 2016-10-19 | 2020-05-05 | Drexel University | Methods of specifically labeling nucleic acids using CRISPR/Cas |
WO2018081535A2 (en) | 2016-10-28 | 2018-05-03 | Massachusetts Institute Of Technology | Dynamic genome engineering |
WO2018080573A1 (en) | 2016-10-28 | 2018-05-03 | Massachusetts Institute Of Technology | Crispr/cas global regulator screening platform |
EP3532616A1 (en) | 2016-10-28 | 2019-09-04 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating herpes simplex virus |
WO2018079134A1 (ja) | 2016-10-31 | 2018-05-03 | 株式会社江口高周波 | リアクトル |
US11795453B2 (en) | 2016-10-31 | 2023-10-24 | Emendobio, Inc. | Compositions for genome editing |
WO2018085288A1 (en) | 2016-11-01 | 2018-05-11 | President And Fellows Of Harvard College | Inhibitors of rna guided nucleases and uses thereof |
WO2018083606A1 (en) | 2016-11-01 | 2018-05-11 | Novartis Ag | Methods and compositions for enhancing gene editing |
GB201618507D0 (en) | 2016-11-02 | 2016-12-14 | Stichting Voor De Technische Wetenschappen And Wageningen Univ | Microbial genome editing |
CN106544353A (zh) | 2016-11-08 | 2017-03-29 | 宁夏医科大学总医院 | 一种利用CRISPR‑Cas9清除鲍曼不动杆菌耐药性基因的方法 |
CN106755088A (zh) | 2016-11-11 | 2017-05-31 | 广东万海细胞生物科技有限公司 | 一种自体car‑t细胞制备方法及应用 |
EP3538561A4 (en) | 2016-11-11 | 2020-10-21 | The Regents of The University of California | RNA-GUIDED POLYPEPTIDE VARIANTS AND METHOD OF USING |
CN106566838B (zh) | 2016-11-14 | 2019-11-01 | 上海伯豪生物技术有限公司 | 一种基于CRISPR-Cas9技术的miR-126全长基因敲除试剂盒及其应用 |
JP2019523011A (ja) | 2016-11-14 | 2019-08-22 | インスティテュート・オブ・ジェネティクス・アンド・ディヴェロプメンタル・バイオロジー、チャイニーズ・アカデミー・オブ・サイエンシズInstitute of Genetics and Developmental Biology, Chinese Academy of Sciences | 植物における塩基編集のための方法 |
CN106554969A (zh) | 2016-11-15 | 2017-04-05 | 陕西理工学院 | 基于抑菌杀菌的多靶点CRISPR/Cas9表达载体 |
CN106754912B (zh) | 2016-11-16 | 2019-11-08 | 上海交通大学 | 一类定向清除肝细胞中HBVcccDNA的质粒及制剂 |
CN110139927A (zh) | 2016-11-16 | 2019-08-16 | 加利福尼亚大学董事会 | Crispr-cas9抑制剂 |
US20180282722A1 (en) | 2016-11-21 | 2018-10-04 | Massachusetts Institute Of Technology | Chimeric DNA:RNA Guide for High Accuracy Cas9 Genome Editing |
CN106480067A (zh) | 2016-11-21 | 2017-03-08 | 中国农业科学院烟草研究所 | 烟草NtNAC096基因控制烟草衰老的应用 |
JP2019535287A (ja) | 2016-11-22 | 2019-12-12 | インテグレイテツド・デイー・エヌ・エイ・テクノロジーズ・インコーポレイテツド | Crispr/cpf1システム及び方法 |
CN106755091A (zh) | 2016-11-28 | 2017-05-31 | 中国人民解放军第三军医大学第附属医院 | 基因敲除载体,mh7a细胞nlrp1基因敲除方法 |
MX2019006157A (es) | 2016-11-28 | 2019-11-21 | Univ Texas | Prevención de la distrofia muscular mediante la edición de genes mediada por crispr/cpf1. |
CN106480036B (zh) | 2016-11-30 | 2019-04-09 | 华南理工大学 | 一种具有启动子功能的dna片段及其应用 |
CN107043779B (zh) | 2016-12-01 | 2020-05-12 | 中国农业科学院作物科学研究所 | 一种CRISPR/nCas9介导的定点碱基替换在植物中的应用 |
CN106834323A (zh) | 2016-12-01 | 2017-06-13 | 安徽大学 | 一种基于维吉尼亚链霉菌IBL14基因cas7‑5‑3的基因编辑方法 |
CA3045335A1 (en) | 2016-12-01 | 2018-06-07 | Universite Laval | Crispr-based treatment of friedreich ataxia |
US9816093B1 (en) | 2016-12-06 | 2017-11-14 | Caribou Biosciences, Inc. | Engineered nucleic acid-targeting nucleic acids |
CN106701830B (zh) | 2016-12-07 | 2020-01-03 | 湖南人文科技学院 | 一种敲除猪胚胎p66shc基因的方法 |
WO2018103686A1 (zh) | 2016-12-07 | 2018-06-14 | 中国科学院上海生命科学研究院 | 叶绿体基因组编辑方法 |
CN106544351B (zh) | 2016-12-08 | 2019-09-10 | 江苏省农业科学院 | CRISPR-Cas9体外敲除耐药基因mcr-1的方法及其专用细胞穿透肽 |
IL267024B2 (en) | 2016-12-08 | 2023-12-01 | Intellia Therapeutics Inc | Adapted leader RNAs for genomic editing |
US11192929B2 (en) | 2016-12-08 | 2021-12-07 | Regents Of The University Of Minnesota | Site-specific DNA base editing using modified APOBEC enzymes |
WO2018107103A1 (en) | 2016-12-09 | 2018-06-14 | The Broad Institute, Inc. | Crispr-systems for modifying a trait of interest in a plant |
SI3551753T1 (sl) | 2016-12-09 | 2022-09-30 | The Broad Institute, Inc. | Diagnostika, temelječa na sistemu CRISPR EFFECTOR |
US11293022B2 (en) | 2016-12-12 | 2022-04-05 | Integrated Dna Technologies, Inc. | Genome editing enhancement |
WO2018111946A1 (en) | 2016-12-12 | 2018-06-21 | Integrated Dna Technologies, Inc. | Genome editing detection |
CN107893074A (zh) | 2016-12-13 | 2018-04-10 | 广东赤萌医疗科技有限公司 | 一种用于敲除CXCR4基因的gRNA、表达载体、敲除系统、试剂盒 |
US11242513B2 (en) | 2016-12-14 | 2022-02-08 | Wageningen Universiteit | Thermostable Cas9 nucleases |
WO2018109101A1 (en) | 2016-12-14 | 2018-06-21 | Wageningen Universiteit | Thermostable cas9 nucleases |
KR101748575B1 (ko) | 2016-12-16 | 2017-06-20 | 주식회사 엠젠플러스 | Ins 유전자 녹아웃 당뇨병 또는 당뇨병 합병증 동물모델 및 이의 제조방법 |
WO2018112336A1 (en) | 2016-12-16 | 2018-06-21 | Ohio State Innovation Foundation | Systems and methods for dna-guided rna cleavage |
WO2018112446A2 (en) | 2016-12-18 | 2018-06-21 | Selonterra, Inc. | Use of apoe4 motif-mediated genes for diagnosis and treatment of alzheimer's disease |
CN106755026A (zh) | 2016-12-18 | 2017-05-31 | 吉林大学 | sgRNA表达载体的构建及牙釉质钙化不全模型的建立 |
GB2605925B (en) | 2016-12-23 | 2023-02-22 | Harvard College | Gene editing of PCSK9 |
WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
CN106755424B (zh) | 2016-12-26 | 2020-11-06 | 郑州大学 | 一种基于crispr的大肠杆菌st131系菌株检测引物、试剂盒及检测方法 |
CN107354173A (zh) | 2016-12-26 | 2017-11-17 | 浙江省医学科学院 | 基于crispr技术和水动力尾静脉注射建立肝脏特异性敲除小鼠模型的方法 |
CN106834347A (zh) | 2016-12-27 | 2017-06-13 | 安徽省农业科学院畜牧兽医研究所 | 一种山羊cdk2基因敲除载体及其构建方法 |
CN106755097A (zh) | 2016-12-27 | 2017-05-31 | 安徽省农业科学院畜牧兽医研究所 | 一种山羊tlr4基因敲除载体及其构建方法 |
CN106868008A (zh) | 2016-12-30 | 2017-06-20 | 重庆高圣生物医药有限责任公司 | CRISPR/Cas9靶向敲除人Lin28A基因及其特异性gRNA |
CN106701763B (zh) | 2016-12-30 | 2019-07-19 | 重庆高圣生物医药有限责任公司 | CRISPR/Cas9靶向敲除人乙肝病毒P基因及其特异性gRNA |
CN106755077A (zh) | 2016-12-30 | 2017-05-31 | 华智水稻生物技术有限公司 | 利用crispr‑cas9技术对水稻cenh3基因定点突变的方法 |
CN106834341B (zh) | 2016-12-30 | 2020-06-16 | 中国农业大学 | 一种基因定点突变载体及其构建方法和应用 |
CN106701818B (zh) | 2017-01-09 | 2020-04-24 | 湖南杂交水稻研究中心 | 一种培育水稻普通核不育系的方法 |
US20190352634A1 (en) | 2017-01-11 | 2019-11-21 | Oxford University Innovation Limited | Crispr rna |
CN107012164B (zh) | 2017-01-11 | 2023-03-03 | 电子科技大学 | CRISPR/Cpf1植物基因组定向修饰功能单元、包含该功能单元的载体及其应用 |
WO2018135838A2 (ko) | 2017-01-17 | 2018-07-26 | 기초과학연구원 | Dna 단일가닥 절단에 의한 염기 교정 비표적 위치 확인 방법 |
CN106701823A (zh) | 2017-01-18 | 2017-05-24 | 上海交通大学 | 生产无岩藻糖单克隆抗体的cho细胞系建立及其应用 |
CN107058372A (zh) | 2017-01-18 | 2017-08-18 | 四川农业大学 | 一种应用于植物上的CRISPR/Cas9载体的构建方法 |
US20180201921A1 (en) | 2017-01-18 | 2018-07-19 | Excision Biotherapeutics, Inc. | CRISPRs |
CN106801056A (zh) | 2017-01-24 | 2017-06-06 | 中国科学院广州生物医药与健康研究院 | 一种sgRNA及其构建的慢病毒载体和应用 |
JP2020505074A (ja) | 2017-01-30 | 2020-02-20 | カー・ヴェー・エス ザート エス・エー ウント コー. カー・ゲー・アー・アーKWS SAAT SE & Co. KGaA | ゲノム工学のためのエンドヌクレアーゼに対する修復鋳型の結合 |
TWI608100B (zh) | 2017-02-03 | 2017-12-11 | 國立清華大學 | Cas9表達質體、大腸桿菌基因剪輯系統及其方法 |
US20190345501A1 (en) | 2017-02-07 | 2019-11-14 | Massachusetts Institute Of Technology | Methods and compositions for rna-guided genetic circuits |
AU2018218280A1 (en) | 2017-02-07 | 2019-08-29 | The Regents Of The University Of California | Gene therapy for haploinsufficiency |
WO2018148647A2 (en) | 2017-02-10 | 2018-08-16 | Lajoie Marc Joseph | Genome editing reagents and their use |
IT201700016321A1 (it) | 2017-02-14 | 2018-08-14 | Univ Degli Studi Di Trento | Mutanti di cas9 ad alta specificita' e loro applicazioni. |
US20200063127A1 (en) | 2017-02-15 | 2020-02-27 | Massachusetts Institute Of Technology | Dna writers, molecular recorders and uses thereof |
CN106957855B (zh) | 2017-02-16 | 2020-04-17 | 上海市农业科学院 | 使用CRISPR/Cas9技术靶向敲除水稻矮杆基因SD1的方法 |
WO2018152418A1 (en) | 2017-02-17 | 2018-08-23 | Temple University - Of The Commonwealth System Of Higher Education | Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5 |
CA3053861A1 (en) | 2017-02-20 | 2018-08-23 | Institute Of Genetics And Developmental Biology, Chinese Academy Of Sciences | Genome editing method |
JP2020508056A (ja) | 2017-02-22 | 2020-03-19 | クリスパー・セラピューティクス・アクチェンゲゼルシャフトCRISPR Therapeutics AG | 遺伝子編集のための組成物および方法 |
US20190365929A1 (en) | 2017-02-22 | 2019-12-05 | Crispr Therapeutics Ag | Materials and methods for treatment of dystrophic epidermolysis bullosa (deb) and other collagen type vii alpha 1 chain (col7a1) gene related conditions or disorders |
US20200248168A1 (en) | 2017-02-22 | 2020-08-06 | Crispr Therapeutics Ag | Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (pcsk9)-related disorders |
WO2018156372A1 (en) | 2017-02-22 | 2018-08-30 | The Regents Of The University Of California | Genetically modified non-human animals and products thereof |
US20200040061A1 (en) | 2017-02-22 | 2020-02-06 | Crispr Therapeutics Ag | Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders |
EP3585899A1 (en) | 2017-02-22 | 2020-01-01 | CRISPR Therapeutics AG | Materials and methods for treatment of primary hyperoxaluria type 1 (ph1) and other alanine-glyoxylate aminotransferase (agxt) gene related conditions or disorders |
US20200216857A1 (en) | 2017-02-22 | 2020-07-09 | Crispr Therapeutics Ag | Materials and methods for treatment of spinocerebellar ataxia type 2 (sca2) and other spinocerebellar ataxia type 2 protein (atxn2) gene related conditions or disorders |
EP3585896A1 (en) | 2017-02-22 | 2020-01-01 | CRISPR Therapeutics AG | Materials and methods for treatment of merosin-deficient cogenital muscular dystrophy (mdcmd) and other laminin, alpha 2 (lama2) gene related conditions or disorders |
EP3585898A1 (en) | 2017-02-22 | 2020-01-01 | CRISPR Therapeutics AG | Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders |
CN106868031A (zh) | 2017-02-24 | 2017-06-20 | 北京大学 | 一种基于分级组装的多个sgRNA串联并行表达的克隆方法及应用 |
WO2018161009A1 (en) | 2017-03-03 | 2018-09-07 | Yale University | Aav-mediated direct in vivo crispr screen in glioblastoma |
US11111492B2 (en) | 2017-03-06 | 2021-09-07 | Florida State University Research Foundation, Inc. | Genome engineering methods using a cytosine-specific Cas9 |
US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
WO2018165629A1 (en) | 2017-03-10 | 2018-09-13 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
EP3595694A4 (en) | 2017-03-14 | 2021-06-09 | The Regents of The University of California | CONSTRUCTION OF CAS9 CRISPR IMMUNE FURTIF |
CN106978428A (zh) | 2017-03-15 | 2017-07-25 | 上海吐露港生物科技有限公司 | 一种Cas蛋白特异结合靶标DNA、调控靶标基因转录的方法及试剂盒 |
EP3596218B1 (en) | 2017-03-15 | 2023-08-23 | The Broad Institute, Inc. | Crispr effector system based diagnostics for virus detection |
CN106906242A (zh) | 2017-03-16 | 2017-06-30 | 重庆高圣生物医药有限责任公司 | 一种提高CRIPSR/Cas9靶向敲除基因产生非同源性末端接合效率的方法 |
CA3057330A1 (en) | 2017-03-21 | 2018-09-27 | Anthony P. Shuber | Treating cancer with cas endonuclease complexes |
EP3601562A1 (en) | 2017-03-23 | 2020-02-05 | President and Fellows of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
CN107012213A (zh) | 2017-03-24 | 2017-08-04 | 南开大学 | 结直肠癌的生物标记物 |
CN106947780A (zh) | 2017-03-28 | 2017-07-14 | 扬州大学 | 一种兔mstn基因的编辑方法 |
US10876101B2 (en) | 2017-03-28 | 2020-12-29 | Locanabio, Inc. | CRISPR-associated (Cas) protein |
CN106906240A (zh) | 2017-03-29 | 2017-06-30 | 浙江大学 | 运用CRISPR‑Cas9系统敲除大麦VE合成通路中的关键基因HPT的方法 |
CA3058584A1 (en) | 2017-03-30 | 2018-10-04 | Kyoto University | Method for inducing exon skipping by genome editing |
CN108660161B (zh) | 2017-03-31 | 2023-05-09 | 中国科学院脑科学与智能技术卓越创新中心 | 基于CRISPR/Cas9技术的制备无嵌合基因敲除动物的方法 |
CN107058358B (zh) | 2017-04-01 | 2020-06-09 | 中国科学院微生物研究所 | 一种双spacer序列识别切割CRISPR-Cas9载体构建及其在疣孢菌中的应用 |
US9938288B1 (en) | 2017-04-05 | 2018-04-10 | President And Fellows Of Harvard College | Macrocyclic compound and uses thereof |
CN106967726B (zh) | 2017-04-05 | 2020-12-29 | 华南农业大学 | 一种创建亚洲栽培稻与非洲栽培稻种间杂种亲和系的方法和应用 |
CN107142282A (zh) | 2017-04-06 | 2017-09-08 | 中山大学 | 一种利用CRISPR/Cas9在哺乳动物细胞中实现大片段DNA定点整合的方法 |
CN107034229A (zh) | 2017-04-07 | 2017-08-11 | 江苏贝瑞利生物科技有限公司 | 一种植物中高效筛选CRISPR/CAS9基因编辑系统候选sgRNA系统及应用 |
CN107058320B (zh) | 2017-04-12 | 2019-08-02 | 南开大学 | Il7r基因缺失斑马鱼突变体的制备及其应用 |
CN106916852B (zh) | 2017-04-13 | 2020-12-04 | 上海科技大学 | 一种碱基编辑系统及其构建和应用方法 |
CN108728476A (zh) | 2017-04-14 | 2018-11-02 | 复旦大学 | 一种利用crispr系统产生多样性抗体文库的方法 |
CN107298701B (zh) | 2017-04-18 | 2020-10-30 | 上海大学 | 玉米转录因子ZmbZIP22及其应用 |
CN106957844A (zh) | 2017-04-20 | 2017-07-18 | 华侨大学 | 一种能有效敲除HTLV‑1病毒基因组的CRISPR/Cas9的gRNA序列 |
WO2018195402A1 (en) | 2017-04-20 | 2018-10-25 | Egenesis, Inc. | Methods for generating genetically modified animals |
US11591589B2 (en) | 2017-04-21 | 2023-02-28 | The General Hospital Corporation | Variants of Cpf1 (Cas12a) with altered PAM specificity |
WO2018195555A1 (en) | 2017-04-21 | 2018-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Crispr/cas 9-mediated integration of polynucleotides by sequential homologous recombination of aav donor vectors |
EP3615665A1 (en) | 2017-04-24 | 2020-03-04 | DuPont Nutrition Biosciences ApS | Novel anti-crispr genes and proteins and methods of use |
CN107043775B (zh) | 2017-04-24 | 2020-06-16 | 中国农业科学院生物技术研究所 | 一种能促进棉花侧根发育的sgRNA及其应用 |
CN206970581U (zh) | 2017-04-26 | 2018-02-06 | 重庆威斯腾生物医药科技有限责任公司 | 一种用于辅助CRISPR/cas9基因敲除的试剂盒 |
WO2018197020A1 (en) | 2017-04-27 | 2018-11-01 | Novozymes A/S | Genome editing by crispr-cas9 using short donor oligonucleotides |
EP3619305A1 (en) | 2017-05-03 | 2020-03-11 | KWS SAAT SE & Co. KGaA | Use of crispr-cas endonucleases for plant genome engineering |
CN110785489A (zh) | 2017-05-04 | 2020-02-11 | 宾夕法尼亚大学董事会 | 使用CRISPR/Cpf1在T细胞中进行基因编辑的组合物和方法 |
CN107012174A (zh) | 2017-05-04 | 2017-08-04 | 昆明理工大学 | CRISPR/Cas9技术在获得家蚕锌指蛋白基因突变体中的应用 |
CN107254485A (zh) | 2017-05-08 | 2017-10-17 | 南京农业大学 | 一种能够快速构建植物基因定点敲除载体的新反应体系 |
CN107129999A (zh) | 2017-05-09 | 2017-09-05 | 福建省农业科学院畜牧兽医研究所 | 利用稳转CRISPR/Cas9系统对病毒基因组进行靶向编辑的方法 |
WO2018208755A1 (en) | 2017-05-09 | 2018-11-15 | The Regents Of The University Of California | Compositions and methods for tagging target proteins in proximity to a nucleotide sequence of interest |
EP3622070A2 (en) | 2017-05-10 | 2020-03-18 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
CN110869498A (zh) | 2017-05-10 | 2020-03-06 | 加利福尼亚大学董事会 | 经由核递送crispr/cas9导向编辑细胞rna |
WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
CN107130000B (zh) | 2017-05-12 | 2019-12-17 | 浙江卫未生物医药科技有限公司 | 一种同时敲除KRAS基因和EGFR基因的CRISPR-Cas9系统及其应用 |
CN106987570A (zh) | 2017-05-16 | 2017-07-28 | 上海交通大学 | 一种Cas9核酸酶R780A及其用途 |
CN106957831B (zh) | 2017-05-16 | 2021-03-12 | 上海交通大学 | 一种Cas9核酸酶K918A及其用途 |
CN106939303B (zh) | 2017-05-16 | 2021-02-23 | 上海交通大学 | 一种Cas9核酸酶R919P及其用途 |
CN106947750B (zh) | 2017-05-16 | 2020-12-08 | 上海交通大学 | 一种Cas9核酸酶Q920P及其用途 |
CN106967697B (zh) | 2017-05-16 | 2021-03-26 | 上海交通大学 | 一种Cas9核酸酶G915F及其用途 |
CN106916820B (zh) | 2017-05-16 | 2019-09-27 | 吉林大学 | 能有效编辑猪ROSA26基因的sgRNA及其应用 |
CN106957830B (zh) | 2017-05-16 | 2020-12-25 | 上海交通大学 | 一种Cas9核酸酶ΔF916及其用途 |
CN107012250B (zh) | 2017-05-16 | 2021-01-29 | 上海交通大学 | 一种适用于CRISPR/Cas9系统的基因组DNA片段编辑精准度的分析方法及应用 |
CN107326042A (zh) | 2017-05-16 | 2017-11-07 | 上海交通大学 | 水稻tms10基因的定点敲除系统及其应用 |
EP3625340A4 (en) | 2017-05-18 | 2021-02-24 | Cargill, Incorporated | GENOME EDITING SYSTEM |
AU2018270088B2 (en) | 2017-05-18 | 2024-05-16 | Massachusetts Institute Of Technology | Systems, methods, and compositions for targeted nucleic acid editing |
WO2018213726A1 (en) | 2017-05-18 | 2018-11-22 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
EP3625359A4 (en) | 2017-05-18 | 2021-03-03 | Children's National Medical Center | APTAMERIC AND NUCLEIC ACID PAYLOAD COMPOSITIONS AND METHODS OF USE |
CN107236737A (zh) | 2017-05-19 | 2017-10-10 | 上海交通大学 | 特异靶向拟南芥ILK2基因的sgRNA序列及其应用 |
CN107043787B (zh) | 2017-05-19 | 2017-12-26 | 南京医科大学 | 一种基于CRISPR/Cas9获得MARF1定点突变小鼠模型的构建方法和应用 |
WO2018217852A1 (en) | 2017-05-23 | 2018-11-29 | Gettysburg College | Crispr based tool for characterizing bacterial serovar diversity |
CN107034188B (zh) | 2017-05-24 | 2018-07-24 | 中山大学附属口腔医院 | 一种靶向骨的外泌体载体、CRISPR/Cas9基因编辑系统及应用 |
CN110997728A (zh) | 2017-05-25 | 2020-04-10 | 通用医疗公司 | 二分型碱基编辑器(bbe)结构和ii-型-cas9锌指编辑 |
WO2018217981A1 (en) | 2017-05-26 | 2018-11-29 | North Carolina State University | Altered guide rnas for modulating cas9 activity and methods of use |
CN107287245B (zh) | 2017-05-27 | 2020-03-17 | 南京农业大学 | 一种基于CRISPR/Cas9技术的Glrx1基因敲除动物模型的构建方法 |
CN107142272A (zh) | 2017-06-05 | 2017-09-08 | 南京金斯瑞生物科技有限公司 | 一种控制大肠杆菌中质粒复制的方法 |
CN107034218A (zh) | 2017-06-07 | 2017-08-11 | 浙江大学 | 用于猪APN基因编辑的靶向sgRNA、修饰载体及其制备方法和应用 |
CN107177595A (zh) | 2017-06-07 | 2017-09-19 | 浙江大学 | 用于猪CD163基因编辑的靶向sgRNA、修饰载体及其制备方法和应用 |
CN107119071A (zh) | 2017-06-07 | 2017-09-01 | 江苏三黍生物科技有限公司 | 一种降低植物直链淀粉含量的方法及应用 |
CN107236739A (zh) | 2017-06-12 | 2017-10-10 | 上海捷易生物科技有限公司 | CRISPR/SaCas9特异性敲除人CXCR4基因的方法 |
CN106987757A (zh) | 2017-06-12 | 2017-07-28 | 苏州双金实业有限公司 | 一种耐腐蚀型奥氏体镍基合金 |
CN107227352A (zh) | 2017-06-13 | 2017-10-03 | 西安医学院 | 基于eGFP的GPR120基因表达的检测方法及应用 |
CN107083392B (zh) | 2017-06-13 | 2020-09-08 | 中国医学科学院病原生物学研究所 | 一种CRISPR/Cpf1基因编辑系统及其在分枝杆菌中的应用 |
CN107245502B (zh) | 2017-06-14 | 2020-11-03 | 中国科学院武汉病毒研究所 | Cd2结合蛋白(cd2ap)和其相互作用蛋白 |
CN107312798B (zh) | 2017-06-16 | 2020-06-23 | 武汉大学 | 含特异靶向CCR5基因的gRNA序列的CRISPR/Cas9重组慢病毒载体及应用 |
CN107099850B (zh) | 2017-06-19 | 2018-05-04 | 东北农业大学 | 一种通过酶切基因组构建CRISPR/Cas9基因组敲除文库的方法 |
CN107266541B (zh) | 2017-06-20 | 2021-06-04 | 上海大学 | 玉米转录因子ZmbHLH167及其应用 |
CN107446951B (zh) | 2017-06-20 | 2021-01-08 | 温氏食品集团股份有限公司 | 一种通过CRISPR/Cas9系统快速筛选重组鸡痘病毒的方法及其应用 |
CN107058328A (zh) | 2017-06-22 | 2017-08-18 | 江苏三黍生物科技有限公司 | 一种提高植物直链淀粉含量的方法及应用 |
CN107099533A (zh) | 2017-06-23 | 2017-08-29 | 东北农业大学 | 一种特异靶向猪IGFBP3基因的sgRNA导向序列及应用 |
US9982279B1 (en) | 2017-06-23 | 2018-05-29 | Inscripta, Inc. | Nucleic acid-guided nucleases |
CN107227307A (zh) | 2017-06-23 | 2017-10-03 | 东北农业大学 | 一种特异靶向猪IRS1基因的sgRNA导向序列及其应用 |
CN107119053A (zh) | 2017-06-23 | 2017-09-01 | 东北农业大学 | 一种特异靶向猪MC4R基因的sgRNA导向序列及其应用 |
WO2019005886A1 (en) | 2017-06-26 | 2019-01-03 | The Broad Institute, Inc. | CRISPR / CAS-CYTIDINE DEAMINASE COMPOSITIONS, SYSTEMS AND METHODS FOR TARGETED EDITING OF NUCLEIC ACIDS |
CN107177631B (zh) | 2017-06-26 | 2020-11-24 | 中国农业大学 | 利用CRISPR-CAS9技术敲除NRK细胞Slc22a2基因的方法 |
CN107217075B (zh) | 2017-06-28 | 2021-07-02 | 西安交通大学医学院第一附属医院 | 一种构建epo基因敲除斑马鱼动物模型的方法及引物、质粒与制备方法 |
CN107356793A (zh) | 2017-07-01 | 2017-11-17 | 合肥东玖电气有限公司 | 一种防火电表箱 |
CN107312793A (zh) | 2017-07-05 | 2017-11-03 | 新疆农业科学院园艺作物研究所 | Cas9介导的番茄基因编辑载体及其应用 |
CN107190006A (zh) | 2017-07-07 | 2017-09-22 | 南通大学附属医院 | 一种靶向IGF‑IR基因的sgRNA及其应用 |
CN107354156B (zh) | 2017-07-19 | 2021-02-09 | 广州医科大学附属第五医院 | 一种敲除野生型T细胞TCR beta链的gRNA及方法 |
CN107236741A (zh) | 2017-07-19 | 2017-10-10 | 广州医科大学附属第五医院 | 一种敲除野生型T细胞TCR alpha链的gRNA及方法 |
CN107400677B (zh) | 2017-07-19 | 2020-05-22 | 江南大学 | 一种基于CRISPR-Cas9系统的地衣芽孢杆菌基因组编辑载体及其制备方法 |
CN107190008A (zh) | 2017-07-19 | 2017-09-22 | 苏州吉赛基因测序科技有限公司 | 一种基于Crispr/cas9的捕获基因组目标序列的方法及其在高通量测序中的应用 |
CN107435051B (zh) | 2017-07-28 | 2020-06-02 | 新乡医学院 | 一种通过CRISPR/Cas9系统快速获得大片段缺失的细胞系基因敲除方法 |
CN107418974A (zh) | 2017-07-28 | 2017-12-01 | 新乡医学院 | 一种利用单克隆细胞分选快速获得CRISPR/Cas9基因敲除稳定细胞株的方法 |
CN107446954A (zh) | 2017-07-28 | 2017-12-08 | 新乡医学院 | 一种sd大鼠t细胞缺失遗传模型的制备方法 |
CN107435069A (zh) | 2017-07-28 | 2017-12-05 | 新乡医学院 | 一种细胞系CRISPR/Cas9基因敲除的快速检测方法 |
CN107267515B (zh) | 2017-07-28 | 2020-08-25 | 重庆医科大学附属儿童医院 | CRISPR/Cas9靶向敲除人CNE10基因及其特异性gRNA |
US11732274B2 (en) | 2017-07-28 | 2023-08-22 | President And Fellows Of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE) |
CN107384922A (zh) | 2017-07-28 | 2017-11-24 | 重庆医科大学附属儿童医院 | CRISPR/Cas9靶向敲除人CNE9基因及其特异性gRNA |
CN107217042B (zh) | 2017-07-31 | 2020-03-06 | 江苏东抗生物医药科技有限公司 | 一种生产无岩藻糖基化蛋白的基因工程细胞系及其建立方法 |
CN107446922A (zh) | 2017-08-03 | 2017-12-08 | 无锡市第二人民医院 | 一种敲除人成骨细胞株中hepcidin基因的gRNA序列及其使用方法 |
CN107502618B (zh) | 2017-08-08 | 2021-03-12 | 中国科学院微生物研究所 | 可控载体消除方法及易用型CRISPR-Cas9工具 |
CN107312785B (zh) | 2017-08-09 | 2019-12-06 | 四川农业大学 | OsKTN80b基因在降低水稻株高方面的应用 |
CN107384926B (zh) | 2017-08-13 | 2020-06-26 | 中国人民解放军疾病预防控制所 | 一种靶向清除细菌耐药性质粒的CRISPR-Cas9系统及应用 |
CN107365804B (zh) | 2017-08-13 | 2019-12-20 | 中国人民解放军疾病预防控制所 | 一种使用温和噬菌体载体包装CRISPR-Cas9系统的方法 |
CN107446923B (zh) | 2017-08-13 | 2019-12-31 | 中国人民解放军疾病预防控制所 | rAAV8-CRISPR-SaCas9系统及在制备乙肝治疗药物中的应用 |
CN107815463A (zh) | 2017-08-15 | 2018-03-20 | 西南大学 | CRISPR/Cas9技术介导miR167前体序列编辑体系的建立方法 |
CN107446924B (zh) | 2017-08-16 | 2020-01-14 | 中国科学院华南植物园 | 一种基于CRISPR-Cas9的猕猴桃基因AcPDS编辑载体及其构建方法和应用 |
CN108034656A (zh) | 2017-08-16 | 2018-05-15 | 四川省农业科学院生物技术核技术研究所 | 与水稻红褐色颖壳性状有关的sgRNA、CRISPR/Cas9载体、载体构建、应用 |
CN107384894B (zh) | 2017-08-21 | 2019-10-22 | 华南师范大学 | 功能化氧化石墨烯高效运载CRISPR/Cas9用于基因编辑的方法 |
CN107557393B (zh) | 2017-08-23 | 2020-05-08 | 中国科学院上海应用物理研究所 | 一种磁性纳米材料介导的CRISPR/Cas9 T细胞内递送系统及其制备方法和应用 |
CN107299114B (zh) | 2017-08-23 | 2021-08-27 | 中国科学院分子植物科学卓越创新中心 | 一种高效的酵母菌染色体融合方法 |
CN107312795A (zh) | 2017-08-24 | 2017-11-03 | 浙江省农业科学院 | 运用CRISPR/Cas9系统创制粉色果实番茄的基因编辑方法 |
CN107460196A (zh) | 2017-08-25 | 2017-12-12 | 同济大学 | 一种免疫缺陷小鼠动物模型的构建方法及应用 |
CN107488649A (zh) | 2017-08-25 | 2017-12-19 | 南方医科大学 | 一种Cpf1和p300核心结构域的融合蛋白、相应的DNA靶向激活系统和应用 |
CN107541525B (zh) | 2017-08-26 | 2021-12-10 | 内蒙古大学 | 一种基于CRISPR/Cas9技术介导山羊Tβ4基因定点敲入的方法 |
CN107446932B (zh) | 2017-08-29 | 2020-02-21 | 江西省农业科学院 | 一个控制水稻雄性生殖发育基因及其应用 |
EP3676376A2 (en) | 2017-08-30 | 2020-07-08 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
CN107519492B (zh) | 2017-09-06 | 2019-01-25 | 武汉迈特维尔生物科技有限公司 | 使用CRISPR技术敲除miR-3187-3p在冠状动脉粥样硬化性心脏病中的应用 |
CN107641631A (zh) | 2017-09-07 | 2018-01-30 | 浙江工业大学 | 一种由化学转化介导的基于CRISPR/Cas9系统敲除大肠杆菌基因的方法 |
CN107362372B (zh) | 2017-09-07 | 2019-01-11 | 佛山波若恩生物科技有限公司 | 使用crispr技术在冠状动脉粥样硬化性心脏病中的应用 |
CN107502608B (zh) | 2017-09-08 | 2020-10-16 | 中山大学 | 用于敲除人ALDH2基因的sgRNA、ALDH2基因缺失细胞株的构建方法及应用 |
CN107557455A (zh) | 2017-09-15 | 2018-01-09 | 国家纳米科学中心 | 一种基于CRISPR‑Cas13a的特异性核酸片段的检测方法 |
CN107475300B (zh) | 2017-09-18 | 2020-04-21 | 上海市同济医院 | Ifit3-eKO1基因敲除小鼠动物模型的构建方法和应用 |
CN107557390A (zh) | 2017-09-18 | 2018-01-09 | 江南大学 | 一种筛选cho细胞系高表达位点的方法 |
CN107630042A (zh) | 2017-09-19 | 2018-01-26 | 安徽大学 | 一种源于I型Cas系统4个cas基因的原核生物基因编辑方法 |
CN107557378A (zh) | 2017-09-19 | 2018-01-09 | 安徽大学 | 一种基于I型CRISPR‑Cas系统中基因cas7‑3的真核基因编辑方法 |
CN107523583A (zh) | 2017-09-19 | 2017-12-29 | 安徽大学 | 一种源于I型CRISPR‑Cas系统中基因cas5‑3的原核基因编辑方法 |
CN107557373A (zh) | 2017-09-19 | 2018-01-09 | 安徽大学 | 一种基于I‑B型CRISPR‑Cas系统基因cas3的基因编辑方法 |
CN107630041A (zh) | 2017-09-19 | 2018-01-26 | 安徽大学 | 一种基于维吉尼亚链霉菌IBL14 I‑B型Cas系统的真核基因编辑方法 |
CN107619837A (zh) | 2017-09-20 | 2018-01-23 | 西北农林科技大学 | 利用Cas9切割核酸酶介导Ipr1定点插入获取转基因牛胎儿成纤维细胞的方法 |
CN107513531B (zh) | 2017-09-21 | 2020-02-21 | 无锡市妇幼保健院 | 用于内源性过表达lncRNA-XIST的gRNA靶点序列及其应用 |
CN107686848A (zh) | 2017-09-26 | 2018-02-13 | 中山大学孙逸仙纪念医院 | 转座子协同CRISPR/Cas9系统的稳定敲除单质粒载体及其应用 |
CN107557394A (zh) | 2017-09-29 | 2018-01-09 | 南京鼓楼医院 | 降低CRISPR/Cas9介导的胚胎基因编辑脱靶率的方法 |
CN107760652A (zh) | 2017-09-29 | 2018-03-06 | 华南理工大学 | CRISPR/CAS9介导药物转运体靶向性敲除的caco‑2细胞模型及其方法 |
CN107828794A (zh) | 2017-09-30 | 2018-03-23 | 上海市农业生物基因中心 | 一种水稻耐盐基因OsRR22突变体、其编码的氨基酸序列、植株及该突变体的创制方法 |
CN107760663A (zh) | 2017-09-30 | 2018-03-06 | 新疆大学 | 油莎草pepc基因的克隆及表达载体的构建和应用 |
CN107630006B (zh) | 2017-09-30 | 2020-09-11 | 山东兴瑞生物科技有限公司 | 一种制备tcr与hla双基因敲除的t细胞的方法 |
CN107604003A (zh) | 2017-10-10 | 2018-01-19 | 南方医科大学 | 一种基于线性化crispr‑cas9慢病毒载体基因敲除试剂盒及其应用 |
CN108102940B (zh) | 2017-10-12 | 2021-07-13 | 中石化上海工程有限公司 | 一株利用CRISPR/Cas9系统敲除XKS1基因的工业酿酒酵母菌株及构建方法 |
CN107474129B (zh) | 2017-10-12 | 2018-10-19 | 江西汉氏联合干细胞科技有限公司 | 特异性增强crispr-cas系统基因编辑效率的方法 |
CN107557381A (zh) | 2017-10-12 | 2018-01-09 | 南京农业大学 | 一种白菜CRISPR‑Cas9基因编辑体系的建立及其应用 |
CN108103586A (zh) | 2017-10-13 | 2018-06-01 | 上海科技大学 | 一种CRISPR/Cas9随机文库及其构建和应用 |
CN107619829B (zh) | 2017-10-14 | 2018-08-24 | 南京平港生物技术有限公司 | 使用crispr-cas系统对间充质干细胞进行gins2基因敲除的方法 |
CN107586779B (zh) | 2017-10-14 | 2018-08-28 | 天津金匙生物科技有限公司 | 使用crispr-cas系统对间充质干细胞进行casp3基因敲除的方法 |
KR20200121782A (ko) | 2017-10-16 | 2020-10-26 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
CN107523567A (zh) | 2017-10-16 | 2017-12-29 | 遵义医学院 | 一种敲除人ezrin基因增强子的食管癌细胞株的构建方法 |
CN107760715B (zh) | 2017-10-17 | 2021-12-10 | 张业胜 | 一种转基因载体及其构建方法和应用 |
CN107937427A (zh) | 2017-10-20 | 2018-04-20 | 广东石油化工学院 | 一种基于CRISPR/Cas9体系的同源修复载体构建方法 |
CN107893086B (zh) | 2017-10-24 | 2021-09-03 | 中国科学院武汉植物园 | 快速构建配对sgRNA的Cas9双元表达载体文库的方法 |
CN107760684B (zh) | 2017-11-03 | 2018-09-25 | 上海拉德钫斯生物科技有限公司 | 使用crispr-cas系统对间充质干细胞进行rbm17基因敲除的方法 |
CN107858346B (zh) | 2017-11-06 | 2020-06-16 | 天津大学 | 一种敲除酿酒酵母染色体的方法 |
CN107794276A (zh) | 2017-11-08 | 2018-03-13 | 中国农业科学院作物科学研究所 | 一种crispr介导快速有效的农作物定点基因片段或等位基因替换方法和体系 |
CN107630043A (zh) | 2017-11-14 | 2018-01-26 | 吉林大学 | 采用敲除技术建立Gadd45a敲除兔模型的方法 |
CN108441519A (zh) | 2017-11-15 | 2018-08-24 | 中国农业大学 | 在crispr/cas9基因编辑中提高同源修复效率的方法 |
CN107858373B (zh) | 2017-11-16 | 2020-03-17 | 山东省千佛山医院 | 内皮细胞条件性敲除ccr5基因小鼠模型的构建方法 |
CN108192956B (zh) | 2017-11-17 | 2021-06-01 | 东南大学 | 一种基于Cas9核酸酶的DNA检测分析方法及其应用 |
CN107893075A (zh) | 2017-11-17 | 2018-04-10 | 和元生物技术(上海)股份有限公司 | CRISPR‑Cas9靶向敲除人肠癌细胞RITA基因及其特异性的sgRNA |
CN107828874B (zh) | 2017-11-20 | 2020-10-16 | 东南大学 | 一种基于crispr的dna检测和分型方法及其应用 |
CN107904261A (zh) | 2017-11-21 | 2018-04-13 | 福州大学 | CRISPR/Cas9纳米基因系统的制备及其在转染方面的应用 |
CN107653256A (zh) | 2017-11-21 | 2018-02-02 | 云南省烟草农业科学研究院 | 一种烟草多酚氧化酶基因NtPPO1及其定点突变方法与应用 |
CN107893076A (zh) | 2017-11-23 | 2018-04-10 | 和元生物技术(上海)股份有限公司 | CRISPR‑Cas9靶向敲除人乳腺癌细胞RASSF2基因及其特异性的sgRNA |
CN107937432B (zh) | 2017-11-24 | 2020-05-01 | 华中农业大学 | 一种基于crispr系统的基因组编辑方法及其应用 |
CN107937501A (zh) | 2017-11-24 | 2018-04-20 | 安徽师范大学 | 一种快速简便的筛选CRISPR/Cas基因编辑阳性对象的方法 |
CN107828738A (zh) | 2017-11-28 | 2018-03-23 | 新乡医学院 | 一种dna甲基转移酶缺陷型cho细胞系及其制备方法及应用 |
CN107988256B (zh) | 2017-12-01 | 2020-07-28 | 暨南大学 | 人亨廷顿基因敲入用重组载体及其构建方法和在模型猪构建中的应用 |
CN108570479B (zh) | 2017-12-06 | 2020-04-03 | 内蒙古大学 | 一种基于CRISPR/Cas9技术介导绒山羊VEGF基因定点敲入的方法 |
CN108148873A (zh) | 2017-12-06 | 2018-06-12 | 南方医科大学 | 一种cav-1基因缺失斑马鱼及其制备方法 |
CN107974466B (zh) | 2017-12-07 | 2020-09-29 | 中国科学院水生生物研究所 | 一种鲟鱼CRISPR/Cas9基因编辑方法 |
CN108315330B (zh) | 2017-12-07 | 2020-05-19 | 嘉兴市第一医院 | CRISPR-Cas9系统特异性靶向人RSPO2基因的sgRNA及敲除方法和应用 |
CN108251423B (zh) | 2017-12-07 | 2020-11-06 | 嘉兴市第一医院 | CRISPR-Cas9系统特异性靶向人RSPO2基因的sgRNA及激活方法和应用 |
CN108148835A (zh) | 2017-12-07 | 2018-06-12 | 和元生物技术(上海)股份有限公司 | CRISPR-Cas9靶向敲除SLC30A1基因及其特异性的sgRNA |
CN107828826A (zh) | 2017-12-12 | 2018-03-23 | 南开大学 | 一种体外高效获得神经干细胞的方法 |
CN108103098B (zh) | 2017-12-14 | 2020-07-28 | 华南理工大学 | 一种化合物皮肤致敏体外评估细胞模型及其构建方法 |
WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
CN107988268A (zh) | 2017-12-18 | 2018-05-04 | 湖南师范大学 | 一种基因敲除选育tcf25基因缺失型斑马鱼的方法 |
CN108018316A (zh) | 2017-12-20 | 2018-05-11 | 湖南师范大学 | 一种基因敲除选育rmnd5b基因缺失型斑马鱼的方法 |
CN108048466B (zh) | 2017-12-21 | 2020-02-07 | 嘉兴市第一医院 | CRISPR-Cas13a系统特异性靶向人RSPO2基因的crRNA及系统和应用 |
RU2652899C1 (ru) | 2017-12-28 | 2018-05-03 | Федеральное бюджетное учреждение науки "Центральный научно-исследовательский институт эпидемиологии" Федеральной службы по надзору в сфере защиты прав потребителей и благополучия человека (ФБУН ЦНИИ Эпидемиологии Роспотребнадзора) | РНК-проводники для подавления репликации вируса гепатита B и для элиминации вируса гепатита B из клетки-хозяина |
CN107893080A (zh) | 2017-12-29 | 2018-04-10 | 江苏省农业科学院 | 一种靶向大鼠Inhba基因的sgRNA及其应用 |
CN108103092B (zh) | 2018-01-05 | 2021-02-12 | 中国农业科学院作物科学研究所 | 利用CRISPR-Cas系统修饰OsHPH基因获得矮化水稻的系统及其应用 |
CN107988229B (zh) | 2018-01-05 | 2020-01-07 | 中国农业科学院作物科学研究所 | 一种利用CRISPR-Cas修饰OsTAC1基因获得分蘖改变的水稻的方法 |
CN107988246A (zh) | 2018-01-05 | 2018-05-04 | 汕头大学医学院 | 一种基因敲除载体及其斑马鱼胶质瘤模型 |
CN108559760A (zh) | 2018-01-09 | 2018-09-21 | 陕西师范大学 | 基于CRISPR靶向基因组修饰技术建立荧光素酶knock-in细胞系的方法 |
CN108148837A (zh) | 2018-01-12 | 2018-06-12 | 南京医科大学 | ApoE-CRISPR/Cas9载体及其在敲除ApoE基因中的应用 |
CN108559730B (zh) | 2018-01-12 | 2021-09-24 | 中国人民解放军第四军医大学 | 利用CRISPR/Cas9技术构建Hutat2:Fc基因敲入单核细胞的实验方法 |
CN108251451A (zh) | 2018-01-16 | 2018-07-06 | 西南大学 | HTT的CRISPR/Cas9-gRNA打靶序列对、质粒及其应用 |
CN108251452A (zh) | 2018-01-17 | 2018-07-06 | 扬州大学 | 一种表达Cas9基因的转基因斑马鱼及其构建方法和应用 |
CN208034188U (zh) | 2018-02-09 | 2018-11-02 | 衡阳市振洋汽车配件有限公司 | 一种快速定位的加工孔用夹具 |
CN108359712B (zh) | 2018-02-09 | 2020-06-26 | 广东省农业科学院农业生物基因研究中心 | 一种快速高效筛选SgRNA靶向DNA序列的方法 |
CN108559745A (zh) | 2018-02-10 | 2018-09-21 | 和元生物技术(上海)股份有限公司 | 基于CRISPR-Cas9技术提高B16F10细胞转染效率的方法 |
CN108486145A (zh) | 2018-02-12 | 2018-09-04 | 中国科学院遗传与发育生物学研究所 | 基于CRISPR/Cas9的植物高效同源重组方法 |
CN108359691B (zh) | 2018-02-12 | 2021-09-28 | 中国科学院重庆绿色智能技术研究院 | 利用mito-CRISPR/Cas9系统敲除异常线粒体DNA的试剂盒及方法 |
CN109021111B (zh) | 2018-02-23 | 2021-12-07 | 上海科技大学 | 一种基因碱基编辑器 |
CN108396027A (zh) | 2018-02-27 | 2018-08-14 | 和元生物技术(上海)股份有限公司 | CRISPR-Cas9靶向敲除人肠癌细胞DEAF1基因及其特异性的sgRNA |
CN108486159B (zh) | 2018-03-01 | 2021-10-22 | 南通大学附属医院 | 一种敲除GRIN2D基因的CRISPR-Cas9系统及其应用 |
CN108342480B (zh) | 2018-03-05 | 2022-03-01 | 北京医院 | 一种基因变异检测质控物及其制备方法 |
CN108410906A (zh) | 2018-03-05 | 2018-08-17 | 淮海工学院 | 一种适用于海洋甲壳类线粒体基因组的CRISPR/Cpf1基因编辑方法 |
CN108410907B (zh) | 2018-03-08 | 2021-08-27 | 湖南农业大学 | 一种基于CRISPR/Cas9技术实现HMGCR基因敲除的方法 |
CN108410911B (zh) | 2018-03-09 | 2021-08-20 | 广西医科大学 | 基于CRISPR/Cas9技术构建的LMNA基因敲除的细胞系 |
CN108486146B (zh) | 2018-03-16 | 2021-02-19 | 中国农业科学院作物科学研究所 | LbCpf1-RR突变体用于CRISPR/Cpf1系统在植物基因编辑中的应用 |
CN108486108B (zh) | 2018-03-16 | 2020-10-09 | 华南农业大学 | 一种敲除人hmgb1基因的细胞株及其应用 |
CN108384784A (zh) | 2018-03-23 | 2018-08-10 | 广西医科大学 | 一种利用CRISPR/Cas9技术敲除Endoglin基因的方法 |
CN108504685A (zh) | 2018-03-27 | 2018-09-07 | 宜明细胞生物科技有限公司 | 一种利用CRISPR/Cas9系统同源重组修复IL-2RG缺陷基因的方法 |
CN108410877A (zh) | 2018-03-27 | 2018-08-17 | 和元生物技术(上海)股份有限公司 | CRISPR-Cas9靶向敲除人细胞SANIL1基因及其特异性的sgRNA |
CN108486234B (zh) | 2018-03-29 | 2022-02-11 | 东南大学 | 一种crispr分型pcr的方法及其应用 |
CN108424931A (zh) | 2018-03-29 | 2018-08-21 | 内蒙古大学 | CRISPR/Cas9技术介导山羊VEGF基因定点整合的方法 |
CN108753772B (zh) | 2018-04-04 | 2020-10-30 | 南华大学 | 基于CRISPR/Cas技术敲除CAPNS1基因的人神经母细胞瘤细胞系的构建方法 |
CN108504693A (zh) | 2018-04-04 | 2018-09-07 | 首都医科大学附属北京朝阳医院 | 利用Crispr技术敲除T合酶基因构建的O-型糖基化异常的结肠癌细胞系 |
CN108486154A (zh) | 2018-04-04 | 2018-09-04 | 福州大学 | 一种唾液酸酶基因敲除小鼠模型的构建方法及其应用 |
CN108486111A (zh) | 2018-04-04 | 2018-09-04 | 山西医科大学 | CRISPR-Cas9靶向敲除人SMYD3基因的方法及其特异性sgRNA |
CN108441520B (zh) | 2018-04-04 | 2020-07-31 | 苏州大学 | 利用CRISPR/Cas9系统构建的基因条件性敲除方法 |
CN108504657B (zh) | 2018-04-12 | 2019-06-14 | 中南民族大学 | 利用crispr-cas9技术敲除hek293t细胞kdm2a基因的方法 |
CN108588182A (zh) | 2018-04-13 | 2018-09-28 | 中国科学院深圳先进技术研究院 | 基于crispr-链取代的等温扩增及检测技术 |
CN108753817A (zh) | 2018-04-13 | 2018-11-06 | 北京华伟康信生物科技有限公司 | 增强细胞的抗癌能力的方法及采用该方法获得的增强型细胞 |
CN108753832A (zh) | 2018-04-20 | 2018-11-06 | 中山大学 | 一种利用CRISPR/Cas9编辑大白猪CD163基因的方法 |
CN108823248A (zh) | 2018-04-20 | 2018-11-16 | 中山大学 | 一种利用CRISPR/Cas9编辑陆川猪CD163基因的方法 |
CN108588071A (zh) | 2018-04-25 | 2018-09-28 | 和元生物技术(上海)股份有限公司 | CRISPR-Cas9靶向敲除人肠癌细胞CNR1基因及其特异性的sgRNA |
CN108588128A (zh) | 2018-04-26 | 2018-09-28 | 南昌大学 | 一种高效率大豆CRISPR/Cas9系统的构建方法及应用 |
CN108707621B (zh) | 2018-04-26 | 2021-02-12 | 中国农业科学院作物科学研究所 | 一种CRISPR/Cpf1系统介导的以RNA转录本为修复模板的同源重组方法 |
CN108546712B (zh) | 2018-04-26 | 2020-08-07 | 中国农业科学院作物科学研究所 | 一种利用CRISPR/LbCpf1系统实现目的基因在植物中同源重组的方法 |
CN108642053A (zh) | 2018-04-28 | 2018-10-12 | 和元生物技术(上海)股份有限公司 | CRISPR-Cas9靶向敲除人肠癌细胞PPP1R1C基因及其特异性的sgRNA |
CN108611364A (zh) | 2018-05-03 | 2018-10-02 | 南京农业大学 | 一种非转基因crispr突变体的制备方法 |
CN108588123A (zh) | 2018-05-07 | 2018-09-28 | 南京医科大学 | CRISPR/Cas9载体组合在制备基因敲除猪的血液制品中的应用 |
CN108610399B (zh) | 2018-05-14 | 2019-09-27 | 河北万玛生物医药有限公司 | 特异性增强crispr-cas系统在表皮干细胞中进行基因编辑效率的方法 |
CN108546717A (zh) | 2018-05-15 | 2018-09-18 | 吉林大学 | 反义lncRNA介导顺式调控抑制靶基因表达的方法 |
CN108546718B (zh) | 2018-05-16 | 2021-07-09 | 康春生 | crRNA介导的CRISPR/Cas13a基因编辑系统在肿瘤细胞中的应用 |
CN108624622A (zh) | 2018-05-16 | 2018-10-09 | 湖南艾佳生物科技股份有限公司 | 一种基于CRISPR-Cas9系统构建的能分泌小鼠白细胞介素-6的基因工程细胞株 |
CN108642055B (zh) | 2018-05-17 | 2021-12-03 | 吉林大学 | 能有效编辑猪miR-17-92基因簇的sgRNA |
CN108642077A (zh) | 2018-05-18 | 2018-10-12 | 江苏省农业科学院 | 基于CRISPR/Cas9基因编辑技术选育绿豆不育突变体的方法及专用gRNA |
CN108642090A (zh) | 2018-05-18 | 2018-10-12 | 中国人民解放军总医院 | 基于CRISPR/Cas9技术获得Nogo-B敲除模式小鼠的方法及应用 |
CN108642078A (zh) | 2018-05-18 | 2018-10-12 | 江苏省农业科学院 | 基于CRISPR/Cas9基因编辑技术选育绿豆开花传粉突变体的方法及专用gRNA |
CN108559732A (zh) | 2018-05-21 | 2018-09-21 | 陕西师范大学 | 基于CRISPR/Cas9靶向基因组修饰技术建立KI-T2A-luciferase细胞系的方法 |
CN108707620A (zh) | 2018-05-22 | 2018-10-26 | 西北农林科技大学 | 一种Gene drive载体及构建方法 |
US20210198330A1 (en) | 2018-05-23 | 2021-07-01 | The Broad Institute, Inc. | Base editors and uses thereof |
CN108690844B (zh) | 2018-05-25 | 2021-10-15 | 西南大学 | HTT的CRISPR/Cas9-gRNA打靶序列对、质粒及HD细胞模型 |
CN108823249A (zh) | 2018-05-28 | 2018-11-16 | 上海海洋大学 | CRISPR/Cas9构建notch1a突变体斑马鱼的方法 |
CN108707629A (zh) | 2018-05-28 | 2018-10-26 | 上海海洋大学 | 斑马鱼notch1b基因突变体的制备方法 |
CN108707628B (zh) | 2018-05-28 | 2021-11-23 | 上海海洋大学 | 斑马鱼notch2基因突变体的制备方法 |
CN108753835A (zh) | 2018-05-30 | 2018-11-06 | 中山大学 | 一种利用CRISPR/Cas9编辑猪BMP15基因的方法 |
CN108707604B (zh) | 2018-05-30 | 2019-07-23 | 江西汉氏联合干细胞科技有限公司 | 表皮干细胞中采用CRISPR-Cas系统进行CNE10基因敲除 |
CN108753836B (zh) | 2018-06-04 | 2021-10-12 | 北京大学 | 一种利用rna干扰机制的基因调控或编辑系统 |
CN108715850B (zh) | 2018-06-05 | 2020-10-23 | 艾一生命科技(广东)有限公司 | 表皮干细胞中采用CRISPR-Cas系统进行GING2基因敲除 |
CN108753813B (zh) | 2018-06-08 | 2021-08-24 | 中国水稻研究所 | 获得无标记转基因植物的方法 |
CN108753783A (zh) | 2018-06-13 | 2018-11-06 | 上海市同济医院 | Sqstm1全基因敲除小鼠动物模型的构建方法和应用 |
CN108728486A (zh) | 2018-06-20 | 2018-11-02 | 江苏省农业科学院 | 一种茄子CRISPR/Cas9基因敲除载体的构建方法和应用 |
CN108841845A (zh) | 2018-06-21 | 2018-11-20 | 广东石油化工学院 | 一种带有筛选标记的CRISPR/Cas9载体及其构建方法 |
CN108893529A (zh) | 2018-06-25 | 2018-11-27 | 武汉博杰生物医学科技有限公司 | 一种基于CRISPR技术特异性检测人KRAS基因2号及3号外显子突变的crRNA |
CN108866093B (zh) | 2018-07-04 | 2021-07-09 | 广东三杰牧草生物科技有限公司 | 一种利用CRISPR/Cas9系统对紫花苜蓿基因定点突变的方法 |
CN108913714A (zh) | 2018-07-05 | 2018-11-30 | 江西省超级水稻研究发展中心 | 一种利用CRISPR/Cas9系统敲除BADH2基因创制香稻的方法 |
CN108795902A (zh) | 2018-07-05 | 2018-11-13 | 深圳三智医学科技有限公司 | 一种安全高效的CRISPR/Cas9基因编辑技术 |
EP3820495A4 (en) | 2018-07-09 | 2022-07-20 | The Broad Institute Inc. | RNA PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES |
CN108913691B (zh) | 2018-07-16 | 2020-09-01 | 山东华御生物科技有限公司 | 表皮干细胞中采用CRISPR-Cas系统进行Card3基因敲除 |
CN108913664B (zh) | 2018-07-20 | 2020-09-04 | 嘉兴学院 | 一种CRISPR/Cas9基因编辑方法敲除卵巢癌细胞中CFP1基因的方法 |
CN108853133A (zh) | 2018-07-25 | 2018-11-23 | 福州大学 | 一种PAMAM与CRISPR/Cas9系统重组质粒递送纳米粒的制备方法 |
CN108823291B (zh) | 2018-07-25 | 2022-04-12 | 领航医学科技(深圳)有限公司 | 基于crispr技术的特异性核酸片段定量检测方法 |
CN108913717A (zh) | 2018-08-01 | 2018-11-30 | 河南农业大学 | 一种利用CRISPR/Cas9系统对水稻PHYB基因定点突变的方法 |
EP3841203A4 (en) | 2018-08-23 | 2022-11-02 | The Broad Institute Inc. | CAS9 VARIANTS WITH NON-CANONICAL PAM SPECIFICITIES AND USES OF THEM |
WO2020051360A1 (en) | 2018-09-05 | 2020-03-12 | The Broad Institute, Inc. | Base editing for treating hutchinson-gilford progeria syndrome |
WO2020086908A1 (en) | 2018-10-24 | 2020-04-30 | The Broad Institute, Inc. | Constructs for improved hdr-dependent genomic editing |
US20220389395A1 (en) | 2018-10-29 | 2022-12-08 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
US20220282275A1 (en) | 2018-11-15 | 2022-09-08 | The Broad Institute, Inc. | G-to-t base editors and uses thereof |
WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
WO2020181180A1 (en) | 2019-03-06 | 2020-09-10 | The Broad Institute, Inc. | A:t to c:g base editors and uses thereof |
WO2020181178A1 (en) | 2019-03-06 | 2020-09-10 | The Broad Institute, Inc. | T:a to a:t base editing through thymine alkylation |
US20220170013A1 (en) | 2019-03-06 | 2022-06-02 | The Broad Institute, Inc. | T:a to a:t base editing through adenosine methylation |
WO2020181202A1 (en) | 2019-03-06 | 2020-09-10 | The Broad Institute, Inc. | A:t to t:a base editing through adenine deamination and oxidation |
WO2020181195A1 (en) | 2019-03-06 | 2020-09-10 | The Broad Institute, Inc. | T:a to a:t base editing through adenine excision |
BR112021018606A2 (pt) | 2019-03-19 | 2021-11-23 | Harvard College | Métodos e composições para editar sequências de nucleotídeos |
US20220204975A1 (en) | 2019-04-12 | 2022-06-30 | President And Fellows Of Harvard College | System for genome editing |
WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
EP3973054A1 (en) | 2019-05-20 | 2022-03-30 | The Broad Institute Inc. | Aav delivery of nucleobase editors |
WO2021025750A1 (en) | 2019-08-08 | 2021-02-11 | The Broad Institute, Inc. | Base editors with diversified targeting scope |
WO2021030666A1 (en) | 2019-08-15 | 2021-02-18 | The Broad Institute, Inc. | Base editing by transglycosylation |
-
2014
- 2014-07-08 US US14/326,109 patent/US9840699B2/en active Active
- 2014-07-08 US US14/326,303 patent/US20150165054A1/en not_active Abandoned
- 2014-07-08 US US14/326,269 patent/US9068179B1/en active Active
- 2014-07-08 US US14/326,290 patent/US20150166984A1/en not_active Abandoned
- 2014-07-08 US US14/326,140 patent/US20150166982A1/en not_active Abandoned
- 2014-07-08 US US14/326,318 patent/US20150166985A1/en not_active Abandoned
- 2014-07-08 US US14/325,815 patent/US11053481B2/en active Active
- 2014-12-12 EP EP19181479.7A patent/EP3604511B1/en active Active
- 2014-12-12 JP JP2016539218A patent/JP2017500035A/ja active Pending
- 2014-12-12 HU HUE14825518A patent/HUE046398T2/hu unknown
- 2014-12-12 CN CN202210053406.0A patent/CN114516920A/zh active Pending
- 2014-12-12 EP EP24155880.8A patent/EP4375373A2/en active Pending
- 2014-12-12 ES ES14825518T patent/ES2754433T3/es active Active
- 2014-12-12 US US15/103,608 patent/US10465176B2/en active Active
- 2014-12-12 PL PL14825518T patent/PL3080265T3/pl unknown
- 2014-12-12 WO PCT/US2014/070038 patent/WO2015089406A1/en active Application Filing
- 2014-12-12 DK DK14825518T patent/DK3080265T3/da active
- 2014-12-12 CN CN201480072550.2A patent/CN105934516B/zh active Active
- 2014-12-12 CA CA2933625A patent/CA2933625C/en active Active
- 2014-12-12 PT PT148255185T patent/PT3080265T/pt unknown
- 2014-12-12 EP EP14825518.5A patent/EP3080265B1/en active Active
- 2014-12-12 AU AU2014362208A patent/AU2014362208B2/en active Active
-
2019
- 2019-04-03 US US16/374,634 patent/US11124782B2/en active Active
-
2020
- 2020-05-07 JP JP2020082201A patent/JP2020164529A/ja active Pending
-
2021
- 2021-01-20 AU AU2021200375A patent/AU2021200375B2/en active Active
- 2021-08-20 US US17/408,306 patent/US20220119785A1/en active Pending
- 2021-11-19 JP JP2021188589A patent/JP2022043042A/ja active Pending
-
2023
- 2023-10-26 AU AU2023254972A patent/AU2023254972A1/en active Pending
-
2024
- 2024-01-04 JP JP2024000252A patent/JP2024061716A/ja active Pending
Patent Citations (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2010132092A2 (en) * | 2009-05-12 | 2010-11-18 | The Scripps Research Institute | Cytidine deaminase fusions and related methods |
US20110104787A1 (en) * | 2009-11-05 | 2011-05-05 | President And Fellows Of Harvard College | Fusion Peptides That Bind to and Modify Target Nucleic Acid Sequences |
WO2013176772A1 (en) * | 2012-05-25 | 2013-11-28 | The Regents Of The University Of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
WO2014144288A1 (en) * | 2013-03-15 | 2014-09-18 | The General Hospital Corporation | Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing |
Non-Patent Citations (7)
Title |
---|
3 BIOTECH., vol. 3, JPN6019005546, 18 September 2012 (2012-09-18), pages 225 - 234, ISSN: 0004510908 * |
BRITISH J. HAEMATOL., vol. 82, JPN6019005552, 1992, pages 66 - 72, ISSN: 0004510910 * |
FAMILIAN CANCER, vol. 9, JPN6019005543, 2010, pages 125 - 129, ISSN: 0004510907 * |
J. ALZHEIMER'S DISEASE, vol. 25, JPN6019005551, 2011, pages 425 - 431, ISSN: 0004510909 * |
NATURE METHODS, vol. 10, no. 10, JPN5016012357, 1 October 2013 (2013-10-01), pages 957 - 963, ISSN: 0004696548 * |
PROTEIN SCI., vol. 13, JPN6019005553, 2004, pages 694 - 702, ISSN: 0004510911 * |
SCIENCE, vol. 333, JPN6015052349, 2011, pages 1843 - 1846, ISSN: 0004696549 * |
Also Published As
Similar Documents
Publication | Publication Date | Title |
---|---|---|
JP2020164529A (ja) | 遺伝子編集用のcas多様体 | |
JP7201153B2 (ja) | プログラム可能cas9-リコンビナーゼ融合タンパク質およびその使用 | |
EP3365356B1 (en) | Nucleobase editors and uses thereof | |
CN110914426A (zh) | 包含核酸可编程dna结合蛋白的核碱基编辑器 | |
CA2956224A1 (en) | Cas9 proteins including ligand-dependent inteins |
Legal Events
Date | Code | Title | Description |
---|---|---|---|
A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20200608 |
|
A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20200828 |
|
A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20210520 |
|
A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20210816 |
|
A02 | Decision of refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A02 Effective date: 20220201 |