KR20160034901A - 서열 조작에 최적화된 crispr-cas 이중 닉카아제 시스템, 방법 및 조성물 - Google Patents
서열 조작에 최적화된 crispr-cas 이중 닉카아제 시스템, 방법 및 조성물 Download PDFInfo
- Publication number
- KR20160034901A KR20160034901A KR1020167001293A KR20167001293A KR20160034901A KR 20160034901 A KR20160034901 A KR 20160034901A KR 1020167001293 A KR1020167001293 A KR 1020167001293A KR 20167001293 A KR20167001293 A KR 20167001293A KR 20160034901 A KR20160034901 A KR 20160034901A
- Authority
- KR
- South Korea
- Prior art keywords
- sequence
- crispr
- tracr
- sequences
- target
- 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.)
- Ceased
Links
- 238000000034 method Methods 0.000 title claims abstract description 384
- 239000000203 mixture Substances 0.000 title claims abstract description 50
- 108010008532 Deoxyribonuclease I Proteins 0.000 title description 12
- 102000007260 Deoxyribonuclease I Human genes 0.000 title description 12
- 108091033409 CRISPR Proteins 0.000 claims abstract description 302
- 239000013598 vector Substances 0.000 claims abstract description 200
- 230000000694 effects Effects 0.000 claims abstract description 64
- 210000003527 eukaryotic cell Anatomy 0.000 claims abstract description 47
- 238000010354 CRISPR gene editing Methods 0.000 claims abstract 20
- 108090000623 proteins and genes Proteins 0.000 claims description 340
- 210000004027 cell Anatomy 0.000 claims description 272
- 102000004190 Enzymes Human genes 0.000 claims description 261
- 108090000790 Enzymes Proteins 0.000 claims description 261
- 102000040430 polynucleotide Human genes 0.000 claims description 250
- 108091033319 polynucleotide Proteins 0.000 claims description 250
- 239000002157 polynucleotide Substances 0.000 claims description 250
- 108020004414 DNA Proteins 0.000 claims description 202
- 230000014509 gene expression Effects 0.000 claims description 169
- 230000035772 mutation Effects 0.000 claims description 169
- 102000004169 proteins and genes Human genes 0.000 claims description 159
- 108020005004 Guide RNA Proteins 0.000 claims description 106
- 238000003776 cleavage reaction Methods 0.000 claims description 95
- 230000007017 scission Effects 0.000 claims description 95
- 230000001105 regulatory effect Effects 0.000 claims description 62
- 101000910035 Streptococcus pyogenes serotype M1 CRISPR-associated endonuclease Cas9/Csn1 Proteins 0.000 claims description 58
- 108091092236 Chimeric RNA Proteins 0.000 claims description 57
- 241000282414 Homo sapiens Species 0.000 claims description 57
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 48
- 201000010099 disease Diseases 0.000 claims description 45
- 239000013603 viral vector Substances 0.000 claims description 40
- 230000035897 transcription Effects 0.000 claims description 39
- 238000013518 transcription Methods 0.000 claims description 39
- 108020004705 Codon Proteins 0.000 claims description 36
- 238000001727 in vivo Methods 0.000 claims description 34
- 230000008439 repair process Effects 0.000 claims description 34
- 230000008685 targeting Effects 0.000 claims description 34
- 102000053602 DNA Human genes 0.000 claims description 32
- 230000006870 function Effects 0.000 claims description 31
- 238000009396 hybridization Methods 0.000 claims description 27
- 230000001404 mediated effect Effects 0.000 claims description 26
- 230000009870 specific binding Effects 0.000 claims description 24
- 230000000295 complement effect Effects 0.000 claims description 22
- 230000001939 inductive effect Effects 0.000 claims description 22
- 210000001808 exosome Anatomy 0.000 claims description 20
- 230000030648 nucleus localization Effects 0.000 claims description 19
- 230000002829 reductive effect Effects 0.000 claims description 18
- 239000002105 nanoparticle Substances 0.000 claims description 17
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 15
- 230000005782 double-strand break Effects 0.000 claims description 15
- 210000004962 mammalian cell Anatomy 0.000 claims description 15
- 230000006780 non-homologous end joining Effects 0.000 claims description 14
- 125000006850 spacer group Chemical group 0.000 claims description 14
- 230000003197 catalytic effect Effects 0.000 claims description 13
- 210000005260 human cell Anatomy 0.000 claims description 13
- 241000282412 Homo Species 0.000 claims description 11
- 239000003814 drug Substances 0.000 claims description 11
- 108091092195 Intron Proteins 0.000 claims description 10
- 238000004519 manufacturing process Methods 0.000 claims description 10
- 239000012634 fragment Substances 0.000 claims description 9
- 230000009466 transformation Effects 0.000 claims description 9
- 238000011282 treatment Methods 0.000 claims description 9
- 230000003412 degenerative effect Effects 0.000 claims description 8
- 238000002744 homologous recombination Methods 0.000 claims description 8
- 230000006801 homologous recombination Effects 0.000 claims description 8
- 108091034117 Oligonucleotide Proteins 0.000 claims description 7
- 230000007547 defect Effects 0.000 claims description 7
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims description 5
- 230000001737 promoting effect Effects 0.000 claims description 5
- 230000006378 damage Effects 0.000 claims description 4
- 229940046166 oligodeoxynucleotide Drugs 0.000 claims description 4
- 108091008146 restriction endonucleases Proteins 0.000 claims description 4
- 230000004075 alteration Effects 0.000 claims description 3
- 230000007423 decrease Effects 0.000 claims description 3
- 230000002265 prevention Effects 0.000 claims description 3
- 238000000844 transformation Methods 0.000 claims description 2
- 102220605874 Cytosolic arginine sensor for mTORC1 subunit 2_D10A_mutation Human genes 0.000 claims 15
- 210000004088 microvessel Anatomy 0.000 claims 1
- 238000012384 transportation and delivery Methods 0.000 abstract description 57
- 230000015572 biosynthetic process Effects 0.000 abstract description 30
- 238000005457 optimization Methods 0.000 abstract description 15
- 210000001236 prokaryotic cell Anatomy 0.000 abstract description 8
- 230000001976 improved effect Effects 0.000 abstract description 7
- 231100000331 toxic Toxicity 0.000 abstract description 2
- 230000002588 toxic effect Effects 0.000 abstract description 2
- 229940088598 enzyme Drugs 0.000 description 258
- 235000018102 proteins Nutrition 0.000 description 117
- 150000007523 nucleic acids Chemical class 0.000 description 77
- 108091027544 Subgenomic mRNA Proteins 0.000 description 73
- 239000000047 product Substances 0.000 description 66
- 125000003729 nucleotide group Chemical group 0.000 description 65
- 239000002773 nucleotide Substances 0.000 description 63
- 102000039446 nucleic acids Human genes 0.000 description 61
- 108020004707 nucleic acids Proteins 0.000 description 61
- 241000196324 Embryophyta Species 0.000 description 39
- 238000012217 deletion Methods 0.000 description 39
- 206010057190 Respiratory tract infections Diseases 0.000 description 38
- 108020004999 messenger RNA Proteins 0.000 description 38
- 241000700605 Viruses Species 0.000 description 36
- 241001465754 Metazoa Species 0.000 description 33
- 235000001014 amino acid Nutrition 0.000 description 33
- 230000037430 deletion Effects 0.000 description 33
- 238000003780 insertion Methods 0.000 description 33
- 230000037431 insertion Effects 0.000 description 33
- 150000001413 amino acids Chemical class 0.000 description 31
- 239000013612 plasmid Substances 0.000 description 29
- 230000027455 binding Effects 0.000 description 28
- 238000009739 binding Methods 0.000 description 28
- 239000002245 particle Substances 0.000 description 28
- 241000701161 unidentified adenovirus Species 0.000 description 28
- 210000001519 tissue Anatomy 0.000 description 26
- 108010077850 Nuclear Localization Signals Proteins 0.000 description 25
- 208000009869 Neu-Laxova syndrome Diseases 0.000 description 24
- 238000003556 assay Methods 0.000 description 24
- 210000004556 brain Anatomy 0.000 description 23
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 description 23
- 239000013604 expression vector Substances 0.000 description 23
- 238000000338 in vitro Methods 0.000 description 23
- 239000000523 sample Substances 0.000 description 23
- 108091028043 Nucleic acid sequence Proteins 0.000 description 21
- 230000008238 biochemical pathway Effects 0.000 description 21
- 230000011664 signaling Effects 0.000 description 21
- 210000004940 nucleus Anatomy 0.000 description 20
- 108090000765 processed proteins & peptides Proteins 0.000 description 19
- 238000011144 upstream manufacturing Methods 0.000 description 19
- 101710163270 Nuclease Proteins 0.000 description 18
- 241000193996 Streptococcus pyogenes Species 0.000 description 18
- 238000004458 analytical method Methods 0.000 description 18
- 238000010362 genome editing Methods 0.000 description 17
- 230000008569 process Effects 0.000 description 17
- 238000006467 substitution reaction Methods 0.000 description 17
- 241000699666 Mus <mouse, genus> Species 0.000 description 16
- 230000001965 increasing effect Effects 0.000 description 16
- 230000004048 modification Effects 0.000 description 16
- 238000012986 modification Methods 0.000 description 16
- 102000004196 processed proteins & peptides Human genes 0.000 description 16
- 230000003612 virological effect Effects 0.000 description 16
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 15
- 238000013459 approach Methods 0.000 description 15
- 230000000875 corresponding effect Effects 0.000 description 15
- 210000002569 neuron Anatomy 0.000 description 15
- 238000004806 packaging method and process Methods 0.000 description 15
- 241000124008 Mammalia Species 0.000 description 14
- 241000699670 Mus sp. Species 0.000 description 14
- -1 RecBCD Proteins 0.000 description 14
- 108020001778 catalytic domains Proteins 0.000 description 14
- 241000894007 species Species 0.000 description 14
- 230000009261 transgenic effect Effects 0.000 description 14
- 201000003883 Cystic fibrosis Diseases 0.000 description 13
- 238000009472 formulation Methods 0.000 description 13
- 239000004055 small Interfering RNA Substances 0.000 description 13
- 238000012360 testing method Methods 0.000 description 13
- 239000013607 AAV vector Substances 0.000 description 12
- 108020004459 Small interfering RNA Proteins 0.000 description 12
- 239000003795 chemical substances by application Substances 0.000 description 12
- 238000001415 gene therapy Methods 0.000 description 12
- 229920001184 polypeptide Polymers 0.000 description 12
- 102100036279 DNA (cytosine-5)-methyltransferase 1 Human genes 0.000 description 11
- 230000007018 DNA scission Effects 0.000 description 11
- 108010043121 Green Fluorescent Proteins Proteins 0.000 description 11
- 102000004144 Green Fluorescent Proteins Human genes 0.000 description 11
- 230000004913 activation Effects 0.000 description 11
- 238000006243 chemical reaction Methods 0.000 description 11
- 230000004927 fusion Effects 0.000 description 11
- 239000005090 green fluorescent protein Substances 0.000 description 11
- 238000001890 transfection Methods 0.000 description 11
- 238000012546 transfer Methods 0.000 description 11
- 241000894006 Bacteria Species 0.000 description 10
- 238000004422 calculation algorithm Methods 0.000 description 10
- 238000005516 engineering process Methods 0.000 description 10
- 230000010354 integration Effects 0.000 description 10
- 101000931098 Homo sapiens DNA (cytosine-5)-methyltransferase 1 Proteins 0.000 description 9
- 101001048956 Homo sapiens Homeobox protein EMX1 Proteins 0.000 description 9
- 108091028113 Trans-activating crRNA Proteins 0.000 description 9
- 230000003321 amplification Effects 0.000 description 9
- 239000000499 gel Substances 0.000 description 9
- 230000002068 genetic effect Effects 0.000 description 9
- 230000003834 intracellular effect Effects 0.000 description 9
- 239000002502 liposome Substances 0.000 description 9
- 210000004185 liver Anatomy 0.000 description 9
- 238000003199 nucleic acid amplification method Methods 0.000 description 9
- 230000010076 replication Effects 0.000 description 9
- 238000010361 transduction Methods 0.000 description 9
- 230000026683 transduction Effects 0.000 description 9
- 108091026890 Coding region Proteins 0.000 description 8
- 150000001875 compounds Chemical class 0.000 description 8
- 102000037865 fusion proteins Human genes 0.000 description 8
- 108020001507 fusion proteins Proteins 0.000 description 8
- 244000052769 pathogen Species 0.000 description 8
- 231100000419 toxicity Toxicity 0.000 description 8
- 230000001988 toxicity Effects 0.000 description 8
- 230000004568 DNA-binding Effects 0.000 description 7
- 241000588724 Escherichia coli Species 0.000 description 7
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 description 7
- 108010008281 Recombinant Fusion Proteins Proteins 0.000 description 7
- 102000007056 Recombinant Fusion Proteins Human genes 0.000 description 7
- 241000194020 Streptococcus thermophilus Species 0.000 description 7
- 125000003275 alpha amino acid group Chemical group 0.000 description 7
- 230000008859 change Effects 0.000 description 7
- 238000001514 detection method Methods 0.000 description 7
- 239000003623 enhancer Substances 0.000 description 7
- 238000003209 gene knockout Methods 0.000 description 7
- 229920000642 polymer Polymers 0.000 description 7
- 238000012216 screening Methods 0.000 description 7
- 238000013519 translation Methods 0.000 description 7
- 235000007627 Caesalpinia Nutrition 0.000 description 6
- 241000522234 Caesalpinia Species 0.000 description 6
- 102000012605 Cystic Fibrosis Transmembrane Conductance Regulator Human genes 0.000 description 6
- 108010079245 Cystic Fibrosis Transmembrane Conductance Regulator Proteins 0.000 description 6
- 241000701022 Cytomegalovirus Species 0.000 description 6
- 108091029865 Exogenous DNA Proteins 0.000 description 6
- 241000238631 Hexapoda Species 0.000 description 6
- 102100023823 Homeobox protein EMX1 Human genes 0.000 description 6
- 241000713666 Lentivirus Species 0.000 description 6
- 241000194017 Streptococcus Species 0.000 description 6
- 230000008901 benefit Effects 0.000 description 6
- 239000000872 buffer Substances 0.000 description 6
- 150000002632 lipids Chemical class 0.000 description 6
- 230000011987 methylation Effects 0.000 description 6
- 238000007069 methylation reaction Methods 0.000 description 6
- 231100000350 mutagenesis Toxicity 0.000 description 6
- 239000013600 plasmid vector Substances 0.000 description 6
- 108020001580 protein domains Proteins 0.000 description 6
- 238000000746 purification Methods 0.000 description 6
- 230000001603 reducing effect Effects 0.000 description 6
- 230000001177 retroviral effect Effects 0.000 description 6
- 239000000126 substance Substances 0.000 description 6
- 241001634120 Adeno-associated virus - 5 Species 0.000 description 5
- 108010088751 Albumins Proteins 0.000 description 5
- 102000009027 Albumins Human genes 0.000 description 5
- 108700010070 Codon Usage Proteins 0.000 description 5
- 241000702421 Dependoparvovirus Species 0.000 description 5
- PEDCQBHIVMGVHV-UHFFFAOYSA-N Glycerine Chemical compound OCC(O)CO PEDCQBHIVMGVHV-UHFFFAOYSA-N 0.000 description 5
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 description 5
- 108700011259 MicroRNAs Proteins 0.000 description 5
- 241000283973 Oryctolagus cuniculus Species 0.000 description 5
- 240000004808 Saccharomyces cerevisiae Species 0.000 description 5
- 239000002671 adjuvant Substances 0.000 description 5
- 125000000539 amino acid group Chemical group 0.000 description 5
- 230000001580 bacterial effect Effects 0.000 description 5
- 239000003153 chemical reaction reagent Substances 0.000 description 5
- 230000003247 decreasing effect Effects 0.000 description 5
- 230000009977 dual effect Effects 0.000 description 5
- 238000001476 gene delivery Methods 0.000 description 5
- 229910052739 hydrogen Inorganic materials 0.000 description 5
- 239000001257 hydrogen Substances 0.000 description 5
- 230000005847 immunogenicity Effects 0.000 description 5
- 230000002401 inhibitory effect Effects 0.000 description 5
- 238000001638 lipofection Methods 0.000 description 5
- 239000003550 marker Substances 0.000 description 5
- 239000011159 matrix material Substances 0.000 description 5
- 230000001225 therapeutic effect Effects 0.000 description 5
- 241000701447 unidentified baculovirus Species 0.000 description 5
- 241001430294 unidentified retrovirus Species 0.000 description 5
- 238000001262 western blot Methods 0.000 description 5
- 241000702423 Adeno-associated virus - 2 Species 0.000 description 4
- 208000002267 Anti-neutrophil cytoplasmic antibody-associated vasculitis Diseases 0.000 description 4
- 102000052510 DNA-Binding Proteins Human genes 0.000 description 4
- 108010070675 Glutathione transferase Proteins 0.000 description 4
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 description 4
- 102100029100 Hematopoietic prostaglandin D synthase Human genes 0.000 description 4
- 108010033040 Histones Proteins 0.000 description 4
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 description 4
- 241000700159 Rattus Species 0.000 description 4
- 235000014680 Saccharomyces cerevisiae Nutrition 0.000 description 4
- 102000001435 Synapsin Human genes 0.000 description 4
- 108050009621 Synapsin Proteins 0.000 description 4
- 230000000692 anti-sense effect Effects 0.000 description 4
- 239000000427 antigen Substances 0.000 description 4
- 108091007433 antigens Proteins 0.000 description 4
- 102000036639 antigens Human genes 0.000 description 4
- 229960002685 biotin Drugs 0.000 description 4
- 239000011616 biotin Substances 0.000 description 4
- 230000015556 catabolic process Effects 0.000 description 4
- 230000003915 cell function Effects 0.000 description 4
- 230000002759 chromosomal effect Effects 0.000 description 4
- 238000010367 cloning Methods 0.000 description 4
- 238000004590 computer program Methods 0.000 description 4
- 230000021615 conjugation Effects 0.000 description 4
- 238000012350 deep sequencing Methods 0.000 description 4
- 238000002716 delivery method Methods 0.000 description 4
- 230000001419 dependent effect Effects 0.000 description 4
- 238000013461 design Methods 0.000 description 4
- 239000000975 dye Substances 0.000 description 4
- 108010025678 empty spiracles homeobox proteins Proteins 0.000 description 4
- 238000010353 genetic engineering Methods 0.000 description 4
- 230000006698 induction Effects 0.000 description 4
- 230000005764 inhibitory process Effects 0.000 description 4
- 238000002372 labelling Methods 0.000 description 4
- 239000002679 microRNA Substances 0.000 description 4
- 102000042567 non-coding RNA Human genes 0.000 description 4
- 230000003287 optical effect Effects 0.000 description 4
- 210000000056 organ Anatomy 0.000 description 4
- 230000004481 post-translational protein modification Effects 0.000 description 4
- 230000002285 radioactive effect Effects 0.000 description 4
- 238000003259 recombinant expression Methods 0.000 description 4
- 230000006798 recombination Effects 0.000 description 4
- 238000005215 recombination Methods 0.000 description 4
- 238000011160 research Methods 0.000 description 4
- 238000012163 sequencing technique Methods 0.000 description 4
- 239000000243 solution Substances 0.000 description 4
- 210000000130 stem cell Anatomy 0.000 description 4
- 208000024891 symptom Diseases 0.000 description 4
- 241001529453 unidentified herpesvirus Species 0.000 description 4
- 241000589158 Agrobacterium Species 0.000 description 3
- 238000010356 CRISPR-Cas9 genome editing Methods 0.000 description 3
- 101000909256 Caldicellulosiruptor bescii (strain ATCC BAA-1888 / DSM 6725 / Z-1320) DNA polymerase I Proteins 0.000 description 3
- 241000282472 Canis lupus familiaris Species 0.000 description 3
- 108010077544 Chromatin Proteins 0.000 description 3
- 241000195493 Cryptophyta Species 0.000 description 3
- 101710177611 DNA polymerase II large subunit Proteins 0.000 description 3
- 101710184669 DNA polymerase II small subunit Proteins 0.000 description 3
- 101710096438 DNA-binding protein Proteins 0.000 description 3
- 102000016928 DNA-directed DNA polymerase Human genes 0.000 description 3
- 101150065780 DNMT gene Proteins 0.000 description 3
- 241000206602 Eukaryota Species 0.000 description 3
- HVLSXIKZNLPZJJ-TXZCQADKSA-N HA peptide Chemical compound C([C@@H](C(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](C(C)C)C(=O)N1[C@@H](CCC1)C(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](CC=1C=CC(O)=CC=1)C(=O)N[C@@H](C)C(O)=O)NC(=O)[C@H]1N(CCC1)C(=O)[C@@H](N)CC=1C=CC(O)=CC=1)C1=CC=C(O)C=C1 HVLSXIKZNLPZJJ-TXZCQADKSA-N 0.000 description 3
- 101000852815 Homo sapiens Insulin receptor Proteins 0.000 description 3
- 241000713772 Human immunodeficiency virus 1 Species 0.000 description 3
- 108020004684 Internal Ribosome Entry Sites Proteins 0.000 description 3
- AHLPHDHHMVZTML-BYPYZUCNSA-N L-Ornithine Chemical compound NCCC[C@H](N)C(O)=O AHLPHDHHMVZTML-BYPYZUCNSA-N 0.000 description 3
- OKKJLVBELUTLKV-UHFFFAOYSA-N Methanol Chemical compound OC OKKJLVBELUTLKV-UHFFFAOYSA-N 0.000 description 3
- AHLPHDHHMVZTML-UHFFFAOYSA-N Orn-delta-NH2 Natural products NCCCC(N)C(O)=O AHLPHDHHMVZTML-UHFFFAOYSA-N 0.000 description 3
- UTJLXEIPEHZYQJ-UHFFFAOYSA-N Ornithine Natural products OC(=O)C(C)CCCN UTJLXEIPEHZYQJ-UHFFFAOYSA-N 0.000 description 3
- 108010043958 Peptoids Proteins 0.000 description 3
- 241000288906 Primates Species 0.000 description 3
- 101000902592 Pyrococcus furiosus (strain ATCC 43587 / DSM 3638 / JCM 8422 / Vc1) DNA polymerase Proteins 0.000 description 3
- 108020004511 Recombinant DNA Proteins 0.000 description 3
- 241000714474 Rous sarcoma virus Species 0.000 description 3
- 241000700618 Vaccinia virus Species 0.000 description 3
- 108700005077 Viral Genes Proteins 0.000 description 3
- 238000009825 accumulation Methods 0.000 description 3
- DZBUGLKDJFMEHC-UHFFFAOYSA-N acridine Chemical compound C1=CC=CC2=CC3=CC=CC=C3N=C21 DZBUGLKDJFMEHC-UHFFFAOYSA-N 0.000 description 3
- 230000009471 action Effects 0.000 description 3
- 235000004279 alanine Nutrition 0.000 description 3
- 210000004102 animal cell Anatomy 0.000 description 3
- 108010005774 beta-Galactosidase Proteins 0.000 description 3
- 239000008280 blood Substances 0.000 description 3
- 210000000234 capsid Anatomy 0.000 description 3
- 238000004113 cell culture Methods 0.000 description 3
- 230000010307 cell transformation Effects 0.000 description 3
- 230000005754 cellular signaling Effects 0.000 description 3
- 210000003483 chromatin Anatomy 0.000 description 3
- 230000004186 co-expression Effects 0.000 description 3
- 238000012790 confirmation Methods 0.000 description 3
- 201000006815 congenital muscular dystrophy Diseases 0.000 description 3
- 230000007123 defense Effects 0.000 description 3
- 230000002950 deficient Effects 0.000 description 3
- 238000006731 degradation reaction Methods 0.000 description 3
- 208000035475 disorder Diseases 0.000 description 3
- 229940079593 drug Drugs 0.000 description 3
- 210000001671 embryonic stem cell Anatomy 0.000 description 3
- 238000011156 evaluation Methods 0.000 description 3
- 210000002950 fibroblast Anatomy 0.000 description 3
- 238000001943 fluorescence-activated cell sorting Methods 0.000 description 3
- 230000036541 health Effects 0.000 description 3
- 210000002443 helper t lymphocyte Anatomy 0.000 description 3
- 102000047882 human INSR Human genes 0.000 description 3
- 230000003308 immunostimulating effect Effects 0.000 description 3
- 238000011534 incubation Methods 0.000 description 3
- 239000004615 ingredient Substances 0.000 description 3
- 238000002347 injection Methods 0.000 description 3
- 239000007924 injection Substances 0.000 description 3
- 238000001990 intravenous administration Methods 0.000 description 3
- 210000003734 kidney Anatomy 0.000 description 3
- 239000003446 ligand Substances 0.000 description 3
- 230000000670 limiting effect Effects 0.000 description 3
- 239000000463 material Substances 0.000 description 3
- 244000005700 microbiome Species 0.000 description 3
- 238000010369 molecular cloning Methods 0.000 description 3
- 108091027963 non-coding RNA Proteins 0.000 description 3
- 229960003104 ornithine Drugs 0.000 description 3
- 108010079892 phosphoglycerol kinase Proteins 0.000 description 3
- 238000012545 processing Methods 0.000 description 3
- 102000005912 ran GTP Binding Protein Human genes 0.000 description 3
- 150000003839 salts Chemical class 0.000 description 3
- 238000010186 staining Methods 0.000 description 3
- 239000000758 substrate Substances 0.000 description 3
- 230000001131 transforming effect Effects 0.000 description 3
- 239000003981 vehicle Substances 0.000 description 3
- YBJHBAHKTGYVGT-ZKWXMUAHSA-N (+)-Biotin Chemical compound N1C(=O)N[C@@H]2[C@H](CCCCC(=O)O)SC[C@@H]21 YBJHBAHKTGYVGT-ZKWXMUAHSA-N 0.000 description 2
- KCYOZNARADAZIZ-CWBQGUJCSA-N 2-[(2e,4e,6e,8e,10e,12e,14e)-15-(4,4,7a-trimethyl-2,5,6,7-tetrahydro-1-benzofuran-2-yl)-6,11-dimethylhexadeca-2,4,6,8,10,12,14-heptaen-2-yl]-4,4,7a-trimethyl-2,5,6,7-tetrahydro-1-benzofuran-6-ol Chemical compound O1C2(C)CC(O)CC(C)(C)C2=CC1C(\C)=C\C=C\C(\C)=C\C=C\C=C(/C)\C=C\C=C(/C)C1C=C2C(C)(C)CCCC2(C)O1 KCYOZNARADAZIZ-CWBQGUJCSA-N 0.000 description 2
- WRMNZCZEMHIOCP-UHFFFAOYSA-N 2-phenylethanol Chemical compound OCCC1=CC=CC=C1 WRMNZCZEMHIOCP-UHFFFAOYSA-N 0.000 description 2
- FWBHETKCLVMNFS-UHFFFAOYSA-N 4',6-Diamino-2-phenylindol Chemical compound C1=CC(C(=N)N)=CC=C1C1=CC2=CC=C(C(N)=N)C=C2N1 FWBHETKCLVMNFS-UHFFFAOYSA-N 0.000 description 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 description 2
- 108091093088 Amplicon Proteins 0.000 description 2
- 241000203069 Archaea Species 0.000 description 2
- 206010003805 Autism Diseases 0.000 description 2
- 208000020706 Autistic disease Diseases 0.000 description 2
- 102100026189 Beta-galactosidase Human genes 0.000 description 2
- 241000283690 Bos taurus Species 0.000 description 2
- 241000282693 Cercopithecidae Species 0.000 description 2
- 108010035563 Chloramphenicol O-acetyltransferase Proteins 0.000 description 2
- 108091029523 CpG island Proteins 0.000 description 2
- KCYOZNARADAZIZ-PPBBKLJYSA-N Cryptochrome Natural products O[C@@H]1CC(C)(C)C=2[C@@](C)(O[C@H](/C(=C\C=C\C(=C/C=C/C=C(\C=C\C=C(\C)/[C@H]3O[C@@]4(C)C(C(C)(C)CCC4)=C3)/C)\C)/C)C=2)C1 KCYOZNARADAZIZ-PPBBKLJYSA-N 0.000 description 2
- 108010037139 Cryptochromes Proteins 0.000 description 2
- 108010009540 DNA (Cytosine-5-)-Methyltransferase 1 Proteins 0.000 description 2
- 108010014303 DNA-directed DNA polymerase Proteins 0.000 description 2
- 238000002965 ELISA Methods 0.000 description 2
- 108700024394 Exon Proteins 0.000 description 2
- ZHNUHDYFZUAESO-UHFFFAOYSA-N Formamide Chemical compound NC=O ZHNUHDYFZUAESO-UHFFFAOYSA-N 0.000 description 2
- 241000223221 Fusarium oxysporum Species 0.000 description 2
- 108091092584 GDNA Proteins 0.000 description 2
- 101150106478 GPS1 gene Proteins 0.000 description 2
- 108010010803 Gelatin Proteins 0.000 description 2
- 206010064571 Gene mutation Diseases 0.000 description 2
- 229940123611 Genome editing Drugs 0.000 description 2
- 241000713813 Gibbon ape leukemia virus Species 0.000 description 2
- 239000004471 Glycine Substances 0.000 description 2
- 101710154606 Hemagglutinin Proteins 0.000 description 2
- 108091027305 Heteroduplex Proteins 0.000 description 2
- 108010001336 Horseradish Peroxidase Proteins 0.000 description 2
- 241000725303 Human immunodeficiency virus Species 0.000 description 2
- VEXZGXHMUGYJMC-UHFFFAOYSA-N Hydrochloric acid Chemical compound Cl VEXZGXHMUGYJMC-UHFFFAOYSA-N 0.000 description 2
- 208000026350 Inborn Genetic disease Diseases 0.000 description 2
- 102000004310 Ion Channels Human genes 0.000 description 2
- 108090000862 Ion Channels Proteins 0.000 description 2
- QNAYBMKLOCPYGJ-REOHCLBHSA-N L-alanine Chemical compound C[C@H](N)C(O)=O QNAYBMKLOCPYGJ-REOHCLBHSA-N 0.000 description 2
- 108060001084 Luciferase Proteins 0.000 description 2
- 239000005089 Luciferase Substances 0.000 description 2
- 235000007688 Lycopersicon esculentum Nutrition 0.000 description 2
- 241000282560 Macaca mulatta Species 0.000 description 2
- 101710175625 Maltose/maltodextrin-binding periplasmic protein Proteins 0.000 description 2
- 241000699660 Mus musculus Species 0.000 description 2
- 101100219625 Mus musculus Casd1 gene Proteins 0.000 description 2
- 101001033765 Mus musculus Methyl-CpG-binding protein 2 Proteins 0.000 description 2
- 206010028980 Neoplasm Diseases 0.000 description 2
- 108020004485 Nonsense Codon Proteins 0.000 description 2
- 238000000636 Northern blotting Methods 0.000 description 2
- 108020004711 Nucleic Acid Probes Proteins 0.000 description 2
- 102000002488 Nucleoplasmin Human genes 0.000 description 2
- 101710093908 Outer capsid protein VP4 Proteins 0.000 description 2
- 101710135467 Outer capsid protein sigma-1 Proteins 0.000 description 2
- ISWSIDIOOBJBQZ-UHFFFAOYSA-N Phenol Chemical compound OC1=CC=CC=C1 ISWSIDIOOBJBQZ-UHFFFAOYSA-N 0.000 description 2
- 101710176177 Protein A56 Proteins 0.000 description 2
- 101100344443 Rattus norvegicus Map1b gene Proteins 0.000 description 2
- 108010091086 Recombinases Proteins 0.000 description 2
- 102000018120 Recombinases Human genes 0.000 description 2
- 108020005091 Replication Origin Proteins 0.000 description 2
- 108700008625 Reporter Genes Proteins 0.000 description 2
- 241000700584 Simplexvirus Species 0.000 description 2
- 108091027967 Small hairpin RNA Proteins 0.000 description 2
- CDBYLPFSWZWCQE-UHFFFAOYSA-L Sodium Carbonate Chemical compound [Na+].[Na+].[O-]C([O-])=O CDBYLPFSWZWCQE-UHFFFAOYSA-L 0.000 description 2
- UIIMBOGNXHQVGW-UHFFFAOYSA-M Sodium bicarbonate Chemical compound [Na+].OC([O-])=O UIIMBOGNXHQVGW-UHFFFAOYSA-M 0.000 description 2
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 2
- 240000003768 Solanum lycopersicum Species 0.000 description 2
- 241000191940 Staphylococcus Species 0.000 description 2
- 241000193998 Streptococcus pneumoniae Species 0.000 description 2
- RAHZWNYVWXNFOC-UHFFFAOYSA-N Sulphur dioxide Chemical compound O=S=O RAHZWNYVWXNFOC-UHFFFAOYSA-N 0.000 description 2
- 108020005038 Terminator Codon Proteins 0.000 description 2
- 102100036407 Thioredoxin Human genes 0.000 description 2
- 241000723873 Tobacco mosaic virus Species 0.000 description 2
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 description 2
- 108020004566 Transfer RNA Proteins 0.000 description 2
- 108700019146 Transgenes Proteins 0.000 description 2
- 102000004243 Tubulin Human genes 0.000 description 2
- 108090000704 Tubulin 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
- 230000021736 acetylation Effects 0.000 description 2
- 238000006640 acetylation reaction Methods 0.000 description 2
- 239000002253 acid Substances 0.000 description 2
- 150000007513 acids Chemical class 0.000 description 2
- 230000003213 activating effect Effects 0.000 description 2
- 206010002022 amyloidosis Diseases 0.000 description 2
- 238000010171 animal model Methods 0.000 description 2
- 238000003491 array Methods 0.000 description 2
- 210000004436 artificial bacterial chromosome Anatomy 0.000 description 2
- 210000001106 artificial yeast chromosome Anatomy 0.000 description 2
- 230000004888 barrier function Effects 0.000 description 2
- KCYOZNARADAZIZ-XZOHMNSDSA-N beta-cryptochrome Natural products CC(=C/C=C/C=C(C)/C=C/C=C(C)/C1OC2(C)CC(O)CC(C)(C)C2=C1)C=CC=C(/C)C3OC4(C)CCCC(C)(C)C4=C3 KCYOZNARADAZIZ-XZOHMNSDSA-N 0.000 description 2
- 239000011230 binding agent Substances 0.000 description 2
- 239000002551 biofuel Substances 0.000 description 2
- 239000013060 biological fluid Substances 0.000 description 2
- 230000008827 biological function Effects 0.000 description 2
- 239000012472 biological sample Substances 0.000 description 2
- 230000005540 biological transmission Effects 0.000 description 2
- 238000001574 biopsy Methods 0.000 description 2
- 210000004369 blood Anatomy 0.000 description 2
- 230000008499 blood brain barrier function Effects 0.000 description 2
- 108091005948 blue fluorescent proteins Proteins 0.000 description 2
- 210000004958 brain cell Anatomy 0.000 description 2
- 210000005013 brain tissue Anatomy 0.000 description 2
- 239000006172 buffering agent Substances 0.000 description 2
- 229910000389 calcium phosphate Inorganic materials 0.000 description 2
- 201000011510 cancer Diseases 0.000 description 2
- 101150055766 cat gene Proteins 0.000 description 2
- 150000001768 cations Chemical class 0.000 description 2
- 230000001413 cellular effect Effects 0.000 description 2
- OSASVXMJTNOKOY-UHFFFAOYSA-N chlorobutanol Chemical compound CC(C)(O)C(Cl)(Cl)Cl OSASVXMJTNOKOY-UHFFFAOYSA-N 0.000 description 2
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 description 2
- 210000000349 chromosome Anatomy 0.000 description 2
- 230000009918 complex formation Effects 0.000 description 2
- 238000012937 correction Methods 0.000 description 2
- 239000013078 crystal Substances 0.000 description 2
- 108010082025 cyan fluorescent protein Proteins 0.000 description 2
- 238000003745 diagnosis Methods 0.000 description 2
- 238000010586 diagram Methods 0.000 description 2
- 238000009826 distribution Methods 0.000 description 2
- 238000011143 downstream manufacturing Methods 0.000 description 2
- 238000009510 drug design Methods 0.000 description 2
- 238000007877 drug screening Methods 0.000 description 2
- 239000012636 effector Substances 0.000 description 2
- CTSPAMFJBXKSOY-UHFFFAOYSA-N ellipticine Chemical compound N1=CC=C2C(C)=C(NC=3C4=CC=CC=3)C4=C(C)C2=C1 CTSPAMFJBXKSOY-UHFFFAOYSA-N 0.000 description 2
- 108010048367 enhanced green fluorescent protein Proteins 0.000 description 2
- 230000002255 enzymatic effect Effects 0.000 description 2
- 210000000981 epithelium Anatomy 0.000 description 2
- CBOQJANXLMLOSS-UHFFFAOYSA-N ethyl vanillin Chemical compound CCOC1=CC(C=O)=CC=C1O CBOQJANXLMLOSS-UHFFFAOYSA-N 0.000 description 2
- 238000000605 extraction Methods 0.000 description 2
- 239000008273 gelatin Substances 0.000 description 2
- 229920000159 gelatin Polymers 0.000 description 2
- 235000019322 gelatine Nutrition 0.000 description 2
- 235000011852 gelatine desserts Nutrition 0.000 description 2
- 208000016361 genetic disease Diseases 0.000 description 2
- 238000012268 genome sequencing Methods 0.000 description 2
- 235000011187 glycerol Nutrition 0.000 description 2
- 230000013595 glycosylation Effects 0.000 description 2
- 238000006206 glycosylation reaction Methods 0.000 description 2
- PCHJSUWPFVWCPO-UHFFFAOYSA-N gold Chemical compound [Au] PCHJSUWPFVWCPO-UHFFFAOYSA-N 0.000 description 2
- 210000005003 heart tissue Anatomy 0.000 description 2
- 238000010438 heat treatment Methods 0.000 description 2
- 239000000185 hemagglutinin Substances 0.000 description 2
- 230000028993 immune response Effects 0.000 description 2
- 238000003018 immunoassay Methods 0.000 description 2
- 238000001114 immunoprecipitation Methods 0.000 description 2
- 230000002779 inactivation Effects 0.000 description 2
- 208000015181 infectious disease Diseases 0.000 description 2
- 208000032839 leukemia Diseases 0.000 description 2
- 230000029226 lipidation Effects 0.000 description 2
- 244000144972 livestock Species 0.000 description 2
- 239000012528 membrane Substances 0.000 description 2
- 210000004379 membrane Anatomy 0.000 description 2
- 208000030159 metabolic disease Diseases 0.000 description 2
- 238000000520 microinjection Methods 0.000 description 2
- 238000002703 mutagenesis Methods 0.000 description 2
- 239000003471 mutagenic agent Substances 0.000 description 2
- 239000013642 negative control Substances 0.000 description 2
- 230000001537 neural effect Effects 0.000 description 2
- 210000000633 nuclear envelope Anatomy 0.000 description 2
- 239000002853 nucleic acid probe Substances 0.000 description 2
- 108060005597 nucleoplasmin Proteins 0.000 description 2
- 239000003921 oil Substances 0.000 description 2
- 235000019198 oils Nutrition 0.000 description 2
- 230000036961 partial effect Effects 0.000 description 2
- 239000000546 pharmaceutical excipient Substances 0.000 description 2
- 230000026731 phosphorylation Effects 0.000 description 2
- 238000006366 phosphorylation reaction Methods 0.000 description 2
- 229920002401 polyacrylamide Polymers 0.000 description 2
- 230000029279 positive regulation of transcription, DNA-dependent Effects 0.000 description 2
- OXCMYAYHXIHQOA-UHFFFAOYSA-N potassium;[2-butyl-5-chloro-3-[[4-[2-(1,2,4-triaza-3-azanidacyclopenta-1,4-dien-5-yl)phenyl]phenyl]methyl]imidazol-4-yl]methanol Chemical compound [K+].CCCCC1=NC(Cl)=C(CO)N1CC1=CC=C(C=2C(=CC=CC=2)C2=N[N-]N=N2)C=C1 OXCMYAYHXIHQOA-UHFFFAOYSA-N 0.000 description 2
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 description 2
- 238000004393 prognosis Methods 0.000 description 2
- 125000001436 propyl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])[H] 0.000 description 2
- 238000011002 quantification Methods 0.000 description 2
- 210000000463 red nucleus Anatomy 0.000 description 2
- 230000009467 reduction Effects 0.000 description 2
- 230000009711 regulatory function Effects 0.000 description 2
- 230000003362 replicative effect Effects 0.000 description 2
- 230000002441 reversible effect Effects 0.000 description 2
- 238000005070 sampling Methods 0.000 description 2
- 238000002864 sequence alignment Methods 0.000 description 2
- 239000000344 soap Substances 0.000 description 2
- 238000010561 standard procedure Methods 0.000 description 2
- 239000006228 supernatant Substances 0.000 description 2
- 238000001356 surgical procedure Methods 0.000 description 2
- 239000000375 suspending agent Substances 0.000 description 2
- 238000003786 synthesis reaction Methods 0.000 description 2
- 230000009897 systematic effect Effects 0.000 description 2
- 108060008226 thioredoxin Proteins 0.000 description 2
- 230000002103 transcriptional effect Effects 0.000 description 2
- 238000011830 transgenic mouse model Methods 0.000 description 2
- 230000010474 transient expression Effects 0.000 description 2
- 230000005945 translocation Effects 0.000 description 2
- 238000002054 transplantation Methods 0.000 description 2
- 230000032258 transport Effects 0.000 description 2
- QORWJWZARLRLPR-UHFFFAOYSA-H tricalcium bis(phosphate) Chemical compound [Ca+2].[Ca+2].[Ca+2].[O-]P([O-])([O-])=O.[O-]P([O-])([O-])=O QORWJWZARLRLPR-UHFFFAOYSA-H 0.000 description 2
- 238000005406 washing Methods 0.000 description 2
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 2
- 229910001868 water Inorganic materials 0.000 description 2
- 239000000080 wetting agent Substances 0.000 description 2
- 108091005957 yellow fluorescent proteins Proteins 0.000 description 2
- OPCHFPHZPIURNA-MFERNQICSA-N (2s)-2,5-bis(3-aminopropylamino)-n-[2-(dioctadecylamino)acetyl]pentanamide Chemical compound CCCCCCCCCCCCCCCCCCN(CC(=O)NC(=O)[C@H](CCCNCCCN)NCCCN)CCCCCCCCCCCCCCCCCC OPCHFPHZPIURNA-MFERNQICSA-N 0.000 description 1
- IYKLZBIWFXPUCS-VIFPVBQESA-N (2s)-2-(naphthalen-1-ylamino)propanoic acid Chemical compound C1=CC=C2C(N[C@@H](C)C(O)=O)=CC=CC2=C1 IYKLZBIWFXPUCS-VIFPVBQESA-N 0.000 description 1
- PNBMXFUTFSFLNU-BXRBKJIMSA-N (2s)-2-aminobutanedioic acid;(2s)-2-aminopropanoic acid Chemical group C[C@H](N)C(O)=O.OC(=O)[C@@H](N)CC(O)=O PNBMXFUTFSFLNU-BXRBKJIMSA-N 0.000 description 1
- SGKRLCUYIXIAHR-AKNGSSGZSA-N (4s,4ar,5s,5ar,6r,12ar)-4-(dimethylamino)-1,5,10,11,12a-pentahydroxy-6-methyl-3,12-dioxo-4a,5,5a,6-tetrahydro-4h-tetracene-2-carboxamide Chemical compound C1=CC=C2[C@H](C)[C@@H]([C@H](O)[C@@H]3[C@](C(O)=C(C(N)=O)C(=O)[C@H]3N(C)C)(O)C3=O)C3=C(O)C2=C1O SGKRLCUYIXIAHR-AKNGSSGZSA-N 0.000 description 1
- OWEGMIWEEQEYGQ-UHFFFAOYSA-N 100676-05-9 Natural products OC1C(O)C(O)C(CO)OC1OCC1C(O)C(O)C(O)C(OC2C(OC(O)C(O)C2O)CO)O1 OWEGMIWEEQEYGQ-UHFFFAOYSA-N 0.000 description 1
- BLCJBICVQSYOIF-UHFFFAOYSA-N 2,2-diaminobutanoic acid Chemical compound CCC(N)(N)C(O)=O BLCJBICVQSYOIF-UHFFFAOYSA-N 0.000 description 1
- NHBKXEKEPDILRR-UHFFFAOYSA-N 2,3-bis(butanoylsulfanyl)propyl butanoate Chemical compound CCCC(=O)OCC(SC(=O)CCC)CSC(=O)CCC NHBKXEKEPDILRR-UHFFFAOYSA-N 0.000 description 1
- CHHHXKFHOYLYRE-UHFFFAOYSA-M 2,4-Hexadienoic acid, potassium salt (1:1), (2E,4E)- Chemical compound [K+].CC=CC=CC([O-])=O CHHHXKFHOYLYRE-UHFFFAOYSA-M 0.000 description 1
- JKMHFZQWWAIEOD-UHFFFAOYSA-N 2-[4-(2-hydroxyethyl)piperazin-1-yl]ethanesulfonic acid Chemical compound OCC[NH+]1CCN(CCS([O-])(=O)=O)CC1 JKMHFZQWWAIEOD-UHFFFAOYSA-N 0.000 description 1
- WTOFYLAWDLQMBZ-UHFFFAOYSA-N 2-azaniumyl-3-thiophen-2-ylpropanoate Chemical compound OC(=O)C(N)CC1=CC=CS1 WTOFYLAWDLQMBZ-UHFFFAOYSA-N 0.000 description 1
- BMYNFMYTOJXKLE-UHFFFAOYSA-N 3-azaniumyl-2-hydroxypropanoate Chemical compound NCC(O)C(O)=O BMYNFMYTOJXKLE-UHFFFAOYSA-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
- STQGQHZAVUOBTE-UHFFFAOYSA-N 7-Cyan-hept-2t-en-4,6-diinsaeure Natural products C1=2C(O)=C3C(=O)C=4C(OC)=CC=CC=4C(=O)C3=C(O)C=2CC(O)(C(C)=O)CC1OC1CC(N)C(O)C(C)O1 STQGQHZAVUOBTE-UHFFFAOYSA-N 0.000 description 1
- QTBSBXVTEAMEQO-UHFFFAOYSA-M Acetate Chemical compound CC([O-])=O QTBSBXVTEAMEQO-UHFFFAOYSA-M 0.000 description 1
- 241000589291 Acinetobacter Species 0.000 description 1
- 241001502050 Acis Species 0.000 description 1
- 102100022900 Actin, cytoplasmic 1 Human genes 0.000 description 1
- 108010085238 Actins Proteins 0.000 description 1
- 102100033647 Activity-regulated cytoskeleton-associated protein Human genes 0.000 description 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 description 1
- 102100032157 Adenylate cyclase type 10 Human genes 0.000 description 1
- 241000567147 Aeropyrum Species 0.000 description 1
- 229920001817 Agar Polymers 0.000 description 1
- BYXHQQCXAJARLQ-ZLUOBGJFSA-N Ala-Ala-Ala Chemical compound C[C@H](N)C(=O)N[C@@H](C)C(=O)N[C@@H](C)C(O)=O BYXHQQCXAJARLQ-ZLUOBGJFSA-N 0.000 description 1
- 108020004774 Alkaline Phosphatase Proteins 0.000 description 1
- 102000002260 Alkaline Phosphatase Human genes 0.000 description 1
- GUBGYTABKSRVRQ-XLOQQCSPSA-N Alpha-Lactose Chemical compound O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@H]1O[C@@H]1[C@@H](CO)O[C@H](O)[C@H](O)[C@H]1O GUBGYTABKSRVRQ-XLOQQCSPSA-N 0.000 description 1
- 241000192542 Anabaena Species 0.000 description 1
- 208000009575 Angelman syndrome Diseases 0.000 description 1
- 241000207208 Aquifex Species 0.000 description 1
- 241000219195 Arabidopsis thaliana Species 0.000 description 1
- 241000205046 Archaeoglobus Species 0.000 description 1
- 108090001008 Avidin Proteins 0.000 description 1
- 241000193830 Bacillus <bacterium> Species 0.000 description 1
- 206010061692 Benign muscle neoplasm Diseases 0.000 description 1
- 108060000903 Beta-catenin Proteins 0.000 description 1
- BTBUEUYNUDRHOZ-UHFFFAOYSA-N Borate Chemical compound [O-]B([O-])[O-] BTBUEUYNUDRHOZ-UHFFFAOYSA-N 0.000 description 1
- 240000002791 Brassica napus Species 0.000 description 1
- 235000004977 Brassica sinapistrum Nutrition 0.000 description 1
- 101150029409 CFTR gene Proteins 0.000 description 1
- 108091079001 CRISPR RNA Proteins 0.000 description 1
- 101150069031 CSN2 gene Proteins 0.000 description 1
- 102000004657 Calcium-Calmodulin-Dependent Protein Kinase Type 2 Human genes 0.000 description 1
- 108010003721 Calcium-Calmodulin-Dependent Protein Kinase Type 2 Proteins 0.000 description 1
- 240000001432 Calendula officinalis Species 0.000 description 1
- 235000005881 Calendula officinalis Nutrition 0.000 description 1
- 241000589876 Campylobacter Species 0.000 description 1
- 101150044789 Cap gene Proteins 0.000 description 1
- 241000283707 Capra Species 0.000 description 1
- 101710132601 Capsid protein Proteins 0.000 description 1
- 102000014914 Carrier Proteins Human genes 0.000 description 1
- 108090000994 Catalytic RNA Proteins 0.000 description 1
- 102000053642 Catalytic RNA Human genes 0.000 description 1
- 102100025064 Cellular tumor antigen p53 Human genes 0.000 description 1
- 241000191366 Chlorobium Species 0.000 description 1
- 241000588881 Chromobacterium Species 0.000 description 1
- 241000193403 Clostridium Species 0.000 description 1
- 101710094648 Coat protein Proteins 0.000 description 1
- 101100007328 Cocos nucifera COS-1 gene Proteins 0.000 description 1
- 108091028732 Concatemer Proteins 0.000 description 1
- 241000186216 Corynebacterium Species 0.000 description 1
- 102100024300 Cryptic protein Human genes 0.000 description 1
- 101710185307 Cryptic protein Proteins 0.000 description 1
- 102100028908 Cullin-3 Human genes 0.000 description 1
- 108010052832 Cytochromes Proteins 0.000 description 1
- 102000018832 Cytochromes Human genes 0.000 description 1
- 102000004127 Cytokines Human genes 0.000 description 1
- 108090000695 Cytokines Proteins 0.000 description 1
- 238000000116 DAPI staining Methods 0.000 description 1
- 108010017826 DNA Polymerase I Proteins 0.000 description 1
- 102000004594 DNA Polymerase I Human genes 0.000 description 1
- 239000012625 DNA intercalator Substances 0.000 description 1
- 230000007067 DNA methylation Effects 0.000 description 1
- 230000008265 DNA repair mechanism Effects 0.000 description 1
- 241000450599 DNA viruses Species 0.000 description 1
- 108700020911 DNA-Binding Proteins Proteins 0.000 description 1
- 101150086683 DYRK1A gene Proteins 0.000 description 1
- WEAHRLBPCANXCN-UHFFFAOYSA-N Daunomycin Natural products CCC1(O)CC(OC2CC(N)C(O)C(C)O2)c3cc4C(=O)c5c(OC)cccc5C(=O)c4c(O)c3C1 WEAHRLBPCANXCN-UHFFFAOYSA-N 0.000 description 1
- 241000605716 Desulfovibrio Species 0.000 description 1
- 229920002307 Dextran Polymers 0.000 description 1
- 101100118093 Drosophila melanogaster eEF1alpha2 gene Proteins 0.000 description 1
- 102100031780 Endonuclease Human genes 0.000 description 1
- 108010013369 Enteropeptidase Proteins 0.000 description 1
- 102100029727 Enteropeptidase Human genes 0.000 description 1
- 241000283086 Equidae Species 0.000 description 1
- 241000588698 Erwinia Species 0.000 description 1
- 102000003951 Erythropoietin Human genes 0.000 description 1
- 102100031939 Erythropoietin Human genes 0.000 description 1
- 108090000394 Erythropoietin Proteins 0.000 description 1
- 241000588722 Escherichia Species 0.000 description 1
- 101150030532 F7 gene Proteins 0.000 description 1
- 108010074860 Factor Xa Proteins 0.000 description 1
- 241000282326 Felis catus Species 0.000 description 1
- 102000008857 Ferritin Human genes 0.000 description 1
- 108050000784 Ferritin Proteins 0.000 description 1
- 238000008416 Ferritin Methods 0.000 description 1
- 241000223218 Fusarium Species 0.000 description 1
- 241000611205 Fusarium oxysporum f. sp. lycopersici Species 0.000 description 1
- 241000605909 Fusobacterium Species 0.000 description 1
- 101150014889 Gad1 gene Proteins 0.000 description 1
- 102100039556 Galectin-4 Human genes 0.000 description 1
- 108700028146 Genetic Enhancer Elements Proteins 0.000 description 1
- 241001135750 Geobacter Species 0.000 description 1
- 208000010412 Glaucoma Diseases 0.000 description 1
- 102000053187 Glucuronidase Human genes 0.000 description 1
- 108010060309 Glucuronidase Proteins 0.000 description 1
- 102100035902 Glutamate decarboxylase 1 Human genes 0.000 description 1
- 102100035857 Glutamate decarboxylase 2 Human genes 0.000 description 1
- 244000068988 Glycine max Species 0.000 description 1
- 235000010469 Glycine max Nutrition 0.000 description 1
- 102100021181 Golgi phosphoprotein 3 Human genes 0.000 description 1
- 102100036698 Golgi reassembly-stacking protein 1 Human genes 0.000 description 1
- 241000288105 Grus Species 0.000 description 1
- ZIXGXMMUKPLXBB-UHFFFAOYSA-N Guatambuinine Natural products N1C2=CC=CC=C2C2=C1C(C)=C1C=CN=C(C)C1=C2 ZIXGXMMUKPLXBB-UHFFFAOYSA-N 0.000 description 1
- 239000007995 HEPES buffer Substances 0.000 description 1
- 241000204988 Haloferax mediterranei Species 0.000 description 1
- 102100021519 Hemoglobin subunit beta Human genes 0.000 description 1
- 108091005904 Hemoglobin subunit beta Proteins 0.000 description 1
- 208000007514 Herpes zoster Diseases 0.000 description 1
- 101001023784 Heteractis crispa GFP-like non-fluorescent chromoprotein Proteins 0.000 description 1
- 101000775498 Homo sapiens Adenylate cyclase type 10 Proteins 0.000 description 1
- 101000721661 Homo sapiens Cellular tumor antigen p53 Proteins 0.000 description 1
- 101000883545 Homo sapiens Chromodomain-helicase-DNA-binding protein 8 Proteins 0.000 description 1
- 101000916238 Homo sapiens Cullin-3 Proteins 0.000 description 1
- 101000907783 Homo sapiens Cystic fibrosis transmembrane conductance regulator Proteins 0.000 description 1
- 101000608765 Homo sapiens Galectin-4 Proteins 0.000 description 1
- 101000873786 Homo sapiens Glutamate decarboxylase 2 Proteins 0.000 description 1
- 101000843809 Homo sapiens Hydroxycarboxylic acid receptor 2 Proteins 0.000 description 1
- 101000975108 Homo sapiens Katanin p60 ATPase-containing subunit A-like 2 Proteins 0.000 description 1
- 101000614988 Homo sapiens Mediator of RNA polymerase II transcription subunit 12 Proteins 0.000 description 1
- 101001057159 Homo sapiens Melanoma-associated antigen C3 Proteins 0.000 description 1
- 101000954986 Homo sapiens Merlin Proteins 0.000 description 1
- 101000615488 Homo sapiens Methyl-CpG-binding domain protein 2 Proteins 0.000 description 1
- 101000635854 Homo sapiens Myoglobin Proteins 0.000 description 1
- 101000706121 Homo sapiens Parvalbumin alpha Proteins 0.000 description 1
- 101000600434 Homo sapiens Putative uncharacterized protein encoded by MIR7-3HG Proteins 0.000 description 1
- 101000738771 Homo sapiens Receptor-type tyrosine-protein phosphatase C Proteins 0.000 description 1
- 101000984753 Homo sapiens Serine/threonine-protein kinase B-raf Proteins 0.000 description 1
- 101000684826 Homo sapiens Sodium channel protein type 2 subunit alpha Proteins 0.000 description 1
- 101000575685 Homo sapiens Synembryn-B Proteins 0.000 description 1
- 101000625338 Homo sapiens Transcriptional adapter 1 Proteins 0.000 description 1
- 101000626636 Homo sapiens Transcriptional adapter 2-beta Proteins 0.000 description 1
- 101000772888 Homo sapiens Ubiquitin-protein ligase E3A Proteins 0.000 description 1
- 101000742373 Homo sapiens Vesicular inhibitory amino acid transporter Proteins 0.000 description 1
- 101000804921 Homo sapiens X-ray repair cross-complementing protein 5 Proteins 0.000 description 1
- 101001074035 Homo sapiens Zinc finger protein GLI2 Proteins 0.000 description 1
- 206010020460 Human T-cell lymphotropic virus type I infection Diseases 0.000 description 1
- 241000714260 Human T-lymphotropic virus 1 Species 0.000 description 1
- 241000701109 Human adenovirus 2 Species 0.000 description 1
- 208000023105 Huntington disease Diseases 0.000 description 1
- 206010061598 Immunodeficiency Diseases 0.000 description 1
- 208000029462 Immunodeficiency disease Diseases 0.000 description 1
- 108060003951 Immunoglobulin Proteins 0.000 description 1
- 102100022971 Katanin p60 ATPase-containing subunit A-like 2 Human genes 0.000 description 1
- ZGUNAGUHMKGQNY-ZETCQYMHSA-N L-alpha-phenylglycine zwitterion Chemical compound OC(=O)[C@@H](N)C1=CC=CC=C1 ZGUNAGUHMKGQNY-ZETCQYMHSA-N 0.000 description 1
- CKLJMWTZIZZHCS-REOHCLBHSA-N L-aspartic acid Chemical compound OC(=O)[C@@H](N)CC(O)=O CKLJMWTZIZZHCS-REOHCLBHSA-N 0.000 description 1
- COLNVLDHVKWLRT-QMMMGPOBSA-N L-phenylalanine Chemical compound OC(=O)[C@@H](N)CC1=CC=CC=C1 COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 description 1
- GUBGYTABKSRVRQ-QKKXKWKRSA-N Lactose Natural products OC[C@H]1O[C@@H](O[C@H]2[C@H](O)[C@@H](O)C(O)O[C@@H]2CO)[C@H](O)[C@@H](O)[C@H]1O GUBGYTABKSRVRQ-QKKXKWKRSA-N 0.000 description 1
- 208000005870 Lafora disease Diseases 0.000 description 1
- 208000014161 Lafora myoclonic epilepsy Diseases 0.000 description 1
- 101710128836 Large T antigen Proteins 0.000 description 1
- 241000589248 Legionella Species 0.000 description 1
- 208000007764 Legionnaires' Disease Diseases 0.000 description 1
- 241000186781 Listeria Species 0.000 description 1
- UPYKUZBSLRQECL-UKMVMLAPSA-N Lycopene Natural products CC(=C/C=C/C=C(C)/C=C/C=C(C)/C=C/C1C(=C)CCCC1(C)C)C=CC=C(/C)C=CC2C(=C)CCCC2(C)C UPYKUZBSLRQECL-UKMVMLAPSA-N 0.000 description 1
- JEVVKJMRZMXFBT-XWDZUXABSA-N Lycophyll Natural products OC/C(=C/CC/C(=C\C=C\C(=C/C=C/C(=C\C=C\C=C(/C=C/C=C(\C=C\C=C(/CC/C=C(/CO)\C)\C)/C)\C)/C)\C)/C)/C JEVVKJMRZMXFBT-XWDZUXABSA-N 0.000 description 1
- 239000007993 MOPS buffer Substances 0.000 description 1
- 101710125418 Major capsid protein Proteins 0.000 description 1
- OFOBLEOULBTSOW-UHFFFAOYSA-L Malonate Chemical compound [O-]C(=O)CC([O-])=O OFOBLEOULBTSOW-UHFFFAOYSA-L 0.000 description 1
- GUBGYTABKSRVRQ-PICCSMPSSA-N Maltose Natural products O[C@@H]1[C@@H](O)[C@H](O)[C@@H](CO)O[C@@H]1O[C@@H]1[C@@H](CO)OC(O)[C@H](O)[C@H]1O GUBGYTABKSRVRQ-PICCSMPSSA-N 0.000 description 1
- 102100021070 Mediator of RNA polymerase II transcription subunit 12 Human genes 0.000 description 1
- 102000018697 Membrane Proteins Human genes 0.000 description 1
- 108010052285 Membrane Proteins Proteins 0.000 description 1
- 102100037106 Merlin Human genes 0.000 description 1
- 241000202974 Methanobacterium Species 0.000 description 1
- 241000203353 Methanococcus Species 0.000 description 1
- 241000204675 Methanopyrus Species 0.000 description 1
- 241000205276 Methanosarcina Species 0.000 description 1
- 102100021299 Methyl-CpG-binding domain protein 2 Human genes 0.000 description 1
- 241000589345 Methylococcus Species 0.000 description 1
- 108060004795 Methyltransferase Proteins 0.000 description 1
- 229920000168 Microcrystalline cellulose Polymers 0.000 description 1
- 101100445099 Mus musculus Emx1 gene Proteins 0.000 description 1
- 101000981253 Mus musculus GPI-linked NAD(P)(+)-arginine ADP-ribosyltransferase 1 Proteins 0.000 description 1
- 101100452019 Mus musculus Icam2 gene Proteins 0.000 description 1
- 101100076422 Mus musculus Mecp2 gene Proteins 0.000 description 1
- 101100078999 Mus musculus Mx1 gene Proteins 0.000 description 1
- 241000186359 Mycobacterium Species 0.000 description 1
- 241000187479 Mycobacterium tuberculosis Species 0.000 description 1
- 241000204031 Mycoplasma Species 0.000 description 1
- 102100030856 Myoglobin Human genes 0.000 description 1
- 201000004458 Myoma Diseases 0.000 description 1
- 241000863420 Myxococcus Species 0.000 description 1
- 108091061960 Naked DNA Proteins 0.000 description 1
- 241000588653 Neisseria Species 0.000 description 1
- 101100058191 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) bcp-1 gene Proteins 0.000 description 1
- 101100385413 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) csm-3 gene Proteins 0.000 description 1
- 239000000020 Nitrocellulose Substances 0.000 description 1
- 241000605122 Nitrosomonas Species 0.000 description 1
- 101710141454 Nucleoprotein Proteins 0.000 description 1
- 108700020796 Oncogene Proteins 0.000 description 1
- 102000043276 Oncogene Human genes 0.000 description 1
- 229910019142 PO4 Inorganic materials 0.000 description 1
- 241000606860 Pasteurella Species 0.000 description 1
- 235000019483 Peanut oil Nutrition 0.000 description 1
- 241001494479 Pecora Species 0.000 description 1
- 208000037581 Persistent Infection Diseases 0.000 description 1
- 108091000080 Phosphotransferase Proteins 0.000 description 1
- 241000607568 Photobacterium Species 0.000 description 1
- 241000204826 Picrophilus Species 0.000 description 1
- RVGRUAULSDPKGF-UHFFFAOYSA-N Poloxamer Chemical compound C1CO1.CC1CO1 RVGRUAULSDPKGF-UHFFFAOYSA-N 0.000 description 1
- 102000012338 Poly(ADP-ribose) Polymerases Human genes 0.000 description 1
- 108010061844 Poly(ADP-ribose) Polymerases Proteins 0.000 description 1
- 229920000776 Poly(Adenosine diphosphate-ribose) polymerase Polymers 0.000 description 1
- 241000605894 Porphyromonas Species 0.000 description 1
- 101710083689 Probable capsid protein Proteins 0.000 description 1
- 108010049395 Prokaryotic Initiation Factor-2 Proteins 0.000 description 1
- XBDQKXXYIPTUBI-UHFFFAOYSA-M Propionate Chemical compound CCC([O-])=O XBDQKXXYIPTUBI-UHFFFAOYSA-M 0.000 description 1
- 101800001494 Protease 2A Proteins 0.000 description 1
- 101800001066 Protein 2A Proteins 0.000 description 1
- 102000001253 Protein Kinase Human genes 0.000 description 1
- 108700040121 Protein Methyltransferases Proteins 0.000 description 1
- 102000055027 Protein Methyltransferases Human genes 0.000 description 1
- 241000221300 Puccinia Species 0.000 description 1
- 102100037401 Putative uncharacterized protein encoded by MIR7-3HG Human genes 0.000 description 1
- 241000205226 Pyrobaculum Species 0.000 description 1
- 241000205160 Pyrococcus Species 0.000 description 1
- 102000014450 RNA Polymerase III Human genes 0.000 description 1
- 108010078067 RNA Polymerase III Proteins 0.000 description 1
- 230000007022 RNA scission Effects 0.000 description 1
- 108010092799 RNA-directed DNA polymerase Proteins 0.000 description 1
- 108091030071 RNAI Proteins 0.000 description 1
- 101100047461 Rattus norvegicus Trpm8 gene Proteins 0.000 description 1
- 102000001218 Rec A Recombinases Human genes 0.000 description 1
- 108010055016 Rec A Recombinases Proteins 0.000 description 1
- 102100037422 Receptor-type tyrosine-protein phosphatase C Human genes 0.000 description 1
- 108091081062 Repeated sequence (DNA) Proteins 0.000 description 1
- 108091028664 Ribonucleotide Proteins 0.000 description 1
- KJTLSVCANCCWHF-UHFFFAOYSA-N Ruthenium Chemical compound [Ru] KJTLSVCANCCWHF-UHFFFAOYSA-N 0.000 description 1
- SUYXJDLXGFPMCQ-INIZCTEOSA-N SJ000287331 Natural products CC1=c2cnccc2=C(C)C2=Nc3ccccc3[C@H]12 SUYXJDLXGFPMCQ-INIZCTEOSA-N 0.000 description 1
- 101100170553 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) DLD2 gene Proteins 0.000 description 1
- 241000607142 Salmonella Species 0.000 description 1
- 102100027103 Serine/threonine-protein kinase B-raf Human genes 0.000 description 1
- 102100023150 Sodium channel protein type 2 subunit alpha Human genes 0.000 description 1
- 101100166144 Staphylococcus aureus cas9 gene Proteins 0.000 description 1
- 241000320123 Streptococcus pyogenes M1 GAS Species 0.000 description 1
- 241000187747 Streptomyces Species 0.000 description 1
- CZMRCDWAGMRECN-UGDNZRGBSA-N Sucrose Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 CZMRCDWAGMRECN-UGDNZRGBSA-N 0.000 description 1
- 229930006000 Sucrose Natural products 0.000 description 1
- 241000282887 Suidae Species 0.000 description 1
- 241000205101 Sulfolobus Species 0.000 description 1
- 102100026014 Synembryn-B Human genes 0.000 description 1
- 102000043123 TET family Human genes 0.000 description 1
- 108091084976 TET family Proteins 0.000 description 1
- 102000018679 Tacrolimus Binding Proteins Human genes 0.000 description 1
- 108010027179 Tacrolimus Binding Proteins Proteins 0.000 description 1
- 239000004098 Tetracycline Substances 0.000 description 1
- 241000186339 Thermoanaerobacter Species 0.000 description 1
- 241000204667 Thermoplasma Species 0.000 description 1
- 241000204652 Thermotoga Species 0.000 description 1
- 241000589596 Thermus Species 0.000 description 1
- 108010022394 Threonine synthase Proteins 0.000 description 1
- 108090000190 Thrombin Proteins 0.000 description 1
- 241000283907 Tragelaphus oryx Species 0.000 description 1
- 102100025043 Transcriptional adapter 1 Human genes 0.000 description 1
- 102100024858 Transcriptional adapter 2-beta Human genes 0.000 description 1
- 108010020764 Transposases Proteins 0.000 description 1
- 102000008579 Transposases Human genes 0.000 description 1
- 241000589886 Treponema Species 0.000 description 1
- 239000007983 Tris buffer Substances 0.000 description 1
- 235000021307 Triticum Nutrition 0.000 description 1
- 244000098338 Triticum aestivum Species 0.000 description 1
- VGQOVCHZGQWAOI-UHFFFAOYSA-N UNPD55612 Natural products N1C(O)C2CC(C=CC(N)=O)=CN2C(=O)C2=CC=C(C)C(O)=C12 VGQOVCHZGQWAOI-UHFFFAOYSA-N 0.000 description 1
- 108010046334 Urease Proteins 0.000 description 1
- 102100038170 Vesicular inhibitory amino acid transporter Human genes 0.000 description 1
- 108020005202 Viral DNA Proteins 0.000 description 1
- 208000036142 Viral infection Diseases 0.000 description 1
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 description 1
- 239000005862 Whey Substances 0.000 description 1
- 108010046377 Whey Proteins Proteins 0.000 description 1
- 102000007544 Whey Proteins Human genes 0.000 description 1
- 241000605941 Wolinella Species 0.000 description 1
- 102100036973 X-ray repair cross-complementing protein 5 Human genes 0.000 description 1
- 102100036976 X-ray repair cross-complementing protein 6 Human genes 0.000 description 1
- 101710124907 X-ray repair cross-complementing protein 6 Proteins 0.000 description 1
- 241000589634 Xanthomonas Species 0.000 description 1
- 241000607734 Yersinia <bacteria> Species 0.000 description 1
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 description 1
- 102100035558 Zinc finger protein GLI2 Human genes 0.000 description 1
- 244000273928 Zingiber officinale Species 0.000 description 1
- 235000006886 Zingiber officinale Nutrition 0.000 description 1
- HMNZFMSWFCAGGW-XPWSMXQVSA-N [3-[hydroxy(2-hydroxyethoxy)phosphoryl]oxy-2-[(e)-octadec-9-enoyl]oxypropyl] (e)-octadec-9-enoate Chemical compound CCCCCCCC\C=C\CCCCCCCC(=O)OCC(COP(O)(=O)OCCO)OC(=O)CCCCCCC\C=C\CCCCCCCC HMNZFMSWFCAGGW-XPWSMXQVSA-N 0.000 description 1
- 238000010521 absorption reaction Methods 0.000 description 1
- DPXJVFZANSGRMM-UHFFFAOYSA-N acetic acid;2,3,4,5,6-pentahydroxyhexanal;sodium Chemical compound [Na].CC(O)=O.OCC(O)C(O)C(O)C(O)C=O DPXJVFZANSGRMM-UHFFFAOYSA-N 0.000 description 1
- 230000002378 acidificating effect Effects 0.000 description 1
- DPKHZNPWBDQZCN-UHFFFAOYSA-N acridine orange free base Chemical compound C1=CC(N(C)C)=CC2=NC3=CC(N(C)C)=CC=C3C=C21 DPKHZNPWBDQZCN-UHFFFAOYSA-N 0.000 description 1
- PEJLNXHANOHNSU-UHFFFAOYSA-N acridine-3,6-diamine;10-methylacridin-10-ium-3,6-diamine;chloride Chemical compound [Cl-].C1=CC(N)=CC2=NC3=CC(N)=CC=C3C=C21.C1=C(N)C=C2[N+](C)=C(C=C(N)C=C3)C3=CC2=C1 PEJLNXHANOHNSU-UHFFFAOYSA-N 0.000 description 1
- 239000013543 active substance Substances 0.000 description 1
- GFFGJBXGBJISGV-UHFFFAOYSA-N adenyl group Chemical group N1=CN=C2N=CNC2=C1N GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 description 1
- 239000000443 aerosol Substances 0.000 description 1
- 238000001261 affinity purification Methods 0.000 description 1
- 239000008272 agar Substances 0.000 description 1
- 235000010419 agar Nutrition 0.000 description 1
- 239000000556 agonist Substances 0.000 description 1
- 230000009418 agronomic effect Effects 0.000 description 1
- 150000001295 alanines Chemical class 0.000 description 1
- 150000001298 alcohols Chemical class 0.000 description 1
- 125000000217 alkyl group Chemical group 0.000 description 1
- 238000003016 alphascreen Methods 0.000 description 1
- WNROFYMDJYEPJX-UHFFFAOYSA-K aluminium hydroxide Chemical compound [OH-].[OH-].[OH-].[Al+3] WNROFYMDJYEPJX-UHFFFAOYSA-K 0.000 description 1
- ILRRQNADMUWWFW-UHFFFAOYSA-K aluminium phosphate Chemical compound O1[Al]2OP1(=O)O2 ILRRQNADMUWWFW-UHFFFAOYSA-K 0.000 description 1
- 238000000137 annealing Methods 0.000 description 1
- VGQOVCHZGQWAOI-HYUHUPJXSA-N anthramycin Chemical compound N1[C@@H](O)[C@@H]2CC(\C=C\C(N)=O)=CN2C(=O)C2=CC=C(C)C(O)=C12 VGQOVCHZGQWAOI-HYUHUPJXSA-N 0.000 description 1
- 210000000628 antibody-producing cell Anatomy 0.000 description 1
- 239000002518 antifoaming agent Substances 0.000 description 1
- 239000003963 antioxidant agent Substances 0.000 description 1
- 230000006907 apoptotic process Effects 0.000 description 1
- 235000003704 aspartic acid Nutrition 0.000 description 1
- 239000005441 aurora Substances 0.000 description 1
- WPYMKLBDIGXBTP-UHFFFAOYSA-N benzoic acid Chemical compound OC(=O)C1=CC=CC=C1 WPYMKLBDIGXBTP-UHFFFAOYSA-N 0.000 description 1
- UCMIRNVEIXFBKS-UHFFFAOYSA-N beta-alanine Chemical group NCCC(O)=O UCMIRNVEIXFBKS-UHFFFAOYSA-N 0.000 description 1
- OQFSQFPPLPISGP-UHFFFAOYSA-N beta-carboxyaspartic acid Natural products OC(=O)C(N)C(C(O)=O)C(O)=O OQFSQFPPLPISGP-UHFFFAOYSA-N 0.000 description 1
- 108091008324 binding proteins Proteins 0.000 description 1
- 230000004071 biological effect Effects 0.000 description 1
- 230000033228 biological regulation Effects 0.000 description 1
- 235000020958 biotin Nutrition 0.000 description 1
- 210000001218 blood-brain barrier Anatomy 0.000 description 1
- 230000037396 body weight Effects 0.000 description 1
- 238000006664 bond formation reaction Methods 0.000 description 1
- 210000000988 bone and bone Anatomy 0.000 description 1
- 210000000621 bronchi Anatomy 0.000 description 1
- 210000004900 c-terminal fragment Anatomy 0.000 description 1
- 210000004899 c-terminal region Anatomy 0.000 description 1
- 102220354910 c.4C>G Human genes 0.000 description 1
- 239000001506 calcium phosphate Substances 0.000 description 1
- 235000011010 calcium phosphates Nutrition 0.000 description 1
- 238000004364 calculation method Methods 0.000 description 1
- 150000001720 carbohydrates Chemical class 0.000 description 1
- 229910052799 carbon Inorganic materials 0.000 description 1
- 229920003123 carboxymethyl cellulose sodium Polymers 0.000 description 1
- 229940063834 carboxymethylcellulose sodium Drugs 0.000 description 1
- 239000000969 carrier Substances 0.000 description 1
- 230000001364 causal effect Effects 0.000 description 1
- 230000022131 cell cycle Effects 0.000 description 1
- 230000032823 cell division Effects 0.000 description 1
- 210000000170 cell membrane Anatomy 0.000 description 1
- 210000003855 cell nucleus Anatomy 0.000 description 1
- 230000003833 cell viability Effects 0.000 description 1
- 239000002738 chelating agent Substances 0.000 description 1
- 239000007795 chemical reaction product Substances 0.000 description 1
- 229960004926 chlorobutanol Drugs 0.000 description 1
- 235000012000 cholesterol Nutrition 0.000 description 1
- ZYVSOIYQKUDENJ-WKSBCEQHSA-N chromomycin A3 Chemical compound O([C@@H]1C[C@@H](O[C@H](C)[C@@H]1OC(C)=O)OC=1C=C2C=C3C[C@H]([C@@H](C(=O)C3=C(O)C2=C(O)C=1C)O[C@@H]1O[C@H](C)[C@@H](O)[C@H](O[C@@H]2O[C@H](C)[C@@H](O)[C@H](O[C@@H]3O[C@@H](C)[C@H](OC(C)=O)[C@@](C)(O)C3)C2)C1)[C@H](OC)C(=O)[C@@H](O)[C@@H](C)O)[C@@H]1C[C@@H](O)[C@@H](OC)[C@@H](C)O1 ZYVSOIYQKUDENJ-WKSBCEQHSA-N 0.000 description 1
- 230000001684 chronic effect Effects 0.000 description 1
- 238000000576 coating method Methods 0.000 description 1
- 239000003086 colorant Substances 0.000 description 1
- 239000002299 complementary DNA Substances 0.000 description 1
- 239000002131 composite material Substances 0.000 description 1
- 239000012141 concentrate Substances 0.000 description 1
- 235000008504 concentrate Nutrition 0.000 description 1
- 239000000470 constituent Substances 0.000 description 1
- 238000010276 construction Methods 0.000 description 1
- 238000011109 contamination Methods 0.000 description 1
- 238000007796 conventional method Methods 0.000 description 1
- 101150055601 cops2 gene Proteins 0.000 description 1
- 239000011258 core-shell material Substances 0.000 description 1
- 230000002596 correlated effect Effects 0.000 description 1
- 210000003618 cortical neuron Anatomy 0.000 description 1
- 210000000805 cytoplasm Anatomy 0.000 description 1
- STQGQHZAVUOBTE-VGBVRHCVSA-N daunorubicin Chemical compound O([C@H]1C[C@@](O)(CC=2C(O)=C3C(=O)C=4C=CC=C(C=4C(=O)C3=C(O)C=21)OC)C(C)=O)[C@H]1C[C@H](N)[C@H](O)[C@H](C)O1 STQGQHZAVUOBTE-VGBVRHCVSA-N 0.000 description 1
- 230000007850 degeneration Effects 0.000 description 1
- 230000002939 deleterious effect Effects 0.000 description 1
- 238000000432 density-gradient centrifugation Methods 0.000 description 1
- 239000005547 deoxyribonucleotide Substances 0.000 description 1
- 125000002637 deoxyribonucleotide group Chemical group 0.000 description 1
- 238000009795 derivation Methods 0.000 description 1
- 238000011161 development Methods 0.000 description 1
- 230000018109 developmental process Effects 0.000 description 1
- 235000014113 dietary fatty acids Nutrition 0.000 description 1
- 102000004419 dihydrofolate reductase Human genes 0.000 description 1
- 239000003085 diluting agent Substances 0.000 description 1
- LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical compound P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 description 1
- 208000037765 diseases and disorders Diseases 0.000 description 1
- 239000002552 dosage form Substances 0.000 description 1
- 231100000673 dose–response relationship Toxicity 0.000 description 1
- 229960003722 doxycycline Drugs 0.000 description 1
- 239000003937 drug carrier Substances 0.000 description 1
- 238000007876 drug discovery Methods 0.000 description 1
- 241001493065 dsRNA viruses Species 0.000 description 1
- 238000010894 electron beam technology Methods 0.000 description 1
- 238000004520 electroporation Methods 0.000 description 1
- 230000008030 elimination Effects 0.000 description 1
- 238000003379 elimination reaction Methods 0.000 description 1
- 210000002257 embryonic structure Anatomy 0.000 description 1
- 239000003995 emulsifying agent Substances 0.000 description 1
- 238000005538 encapsulation Methods 0.000 description 1
- 230000012202 endocytosis Effects 0.000 description 1
- 210000002889 endothelial cell Anatomy 0.000 description 1
- 230000002708 enhancing effect Effects 0.000 description 1
- 238000001952 enzyme assay Methods 0.000 description 1
- 229940105423 erythropoietin Drugs 0.000 description 1
- BEFDCLMNVWHSGT-UHFFFAOYSA-N ethenylcyclopentane Chemical compound C=CC1CCCC1 BEFDCLMNVWHSGT-UHFFFAOYSA-N 0.000 description 1
- ZMMJGEGLRURXTF-UHFFFAOYSA-N ethidium bromide Chemical compound [Br-].C12=CC(N)=CC=C2C2=CC=C(N)C=C2[N+](CC)=C1C1=CC=CC=C1 ZMMJGEGLRURXTF-UHFFFAOYSA-N 0.000 description 1
- 229960005542 ethidium bromide Drugs 0.000 description 1
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 description 1
- 229940073505 ethyl vanillin Drugs 0.000 description 1
- 230000002964 excitative effect Effects 0.000 description 1
- 230000029142 excretion Effects 0.000 description 1
- 239000013613 expression plasmid Substances 0.000 description 1
- 230000008622 extracellular signaling Effects 0.000 description 1
- 210000001508 eye Anatomy 0.000 description 1
- 239000000194 fatty acid Substances 0.000 description 1
- 229930195729 fatty acid Natural products 0.000 description 1
- 150000004665 fatty acids Chemical class 0.000 description 1
- 230000001605 fetal effect Effects 0.000 description 1
- 239000000945 filler Substances 0.000 description 1
- 239000000796 flavoring agent Substances 0.000 description 1
- 108010021843 fluorescent protein 583 Proteins 0.000 description 1
- 102000034287 fluorescent proteins Human genes 0.000 description 1
- 108091006047 fluorescent proteins Proteins 0.000 description 1
- 235000013305 food Nutrition 0.000 description 1
- 235000013355 food flavoring agent Nutrition 0.000 description 1
- 125000002485 formyl group Chemical group [H]C(*)=O 0.000 description 1
- 238000010230 functional analysis Methods 0.000 description 1
- 125000000524 functional group Chemical group 0.000 description 1
- 210000001222 gaba-ergic neuron Anatomy 0.000 description 1
- ZXQYGBMAQZUVMI-GCMPRSNUSA-N gamma-cyhalothrin Chemical compound CC1(C)[C@@H](\C=C(/Cl)C(F)(F)F)[C@H]1C(=O)O[C@H](C#N)C1=CC=CC(OC=2C=CC=CC=2)=C1 ZXQYGBMAQZUVMI-GCMPRSNUSA-N 0.000 description 1
- 238000003500 gene array Methods 0.000 description 1
- 238000012239 gene modification Methods 0.000 description 1
- 230000030279 gene silencing Effects 0.000 description 1
- 230000009368 gene silencing by RNA Effects 0.000 description 1
- 238000010363 gene targeting Methods 0.000 description 1
- 230000005017 genetic modification Effects 0.000 description 1
- 238000010448 genetic screening Methods 0.000 description 1
- 238000012248 genetic selection Methods 0.000 description 1
- 230000007614 genetic variation Effects 0.000 description 1
- 235000013617 genetically modified food Nutrition 0.000 description 1
- 210000004602 germ cell Anatomy 0.000 description 1
- 235000008397 ginger Nutrition 0.000 description 1
- 239000011521 glass Substances 0.000 description 1
- 239000003862 glucocorticoid Substances 0.000 description 1
- 229910052737 gold Inorganic materials 0.000 description 1
- 239000010931 gold Substances 0.000 description 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 description 1
- 230000011132 hemopoiesis Effects 0.000 description 1
- 208000006454 hepatitis Diseases 0.000 description 1
- 231100000283 hepatitis Toxicity 0.000 description 1
- 210000004295 hippocampal neuron Anatomy 0.000 description 1
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 description 1
- 230000001744 histochemical effect Effects 0.000 description 1
- 102000056427 human CFTR Human genes 0.000 description 1
- 210000005119 human aortic smooth muscle cell Anatomy 0.000 description 1
- 125000004435 hydrogen atom Chemical group [H]* 0.000 description 1
- 210000002865 immune cell Anatomy 0.000 description 1
- 210000000987 immune system Anatomy 0.000 description 1
- 230000003053 immunization Effects 0.000 description 1
- 238000003119 immunoblot Methods 0.000 description 1
- 230000000984 immunochemical effect Effects 0.000 description 1
- 230000007813 immunodeficiency Effects 0.000 description 1
- 238000010185 immunofluorescence analysis Methods 0.000 description 1
- 102000018358 immunoglobulin Human genes 0.000 description 1
- 238000003364 immunohistochemistry Methods 0.000 description 1
- 238000007901 in situ hybridization Methods 0.000 description 1
- 238000003017 in situ immunoassay Methods 0.000 description 1
- 230000000415 inactivating effect Effects 0.000 description 1
- 206010022000 influenza Diseases 0.000 description 1
- 108700032552 influenza virus INS1 Proteins 0.000 description 1
- 230000000977 initiatory effect Effects 0.000 description 1
- 229910052500 inorganic mineral Inorganic materials 0.000 description 1
- 238000002743 insertional mutagenesis Methods 0.000 description 1
- 239000012212 insulator Substances 0.000 description 1
- 230000003993 interaction Effects 0.000 description 1
- 239000000138 intercalating agent Substances 0.000 description 1
- 230000004068 intracellular signaling Effects 0.000 description 1
- 238000010255 intramuscular injection Methods 0.000 description 1
- 239000007927 intramuscular injection Substances 0.000 description 1
- 238000010253 intravenous injection Methods 0.000 description 1
- 238000011835 investigation Methods 0.000 description 1
- 150000002500 ions Chemical class 0.000 description 1
- 238000011005 laboratory method Methods 0.000 description 1
- 239000008101 lactose Substances 0.000 description 1
- 230000004807 localization Effects 0.000 description 1
- 230000007774 longterm Effects 0.000 description 1
- 231100000053 low toxicity Toxicity 0.000 description 1
- 210000004072 lung Anatomy 0.000 description 1
- 229960004999 lycopene Drugs 0.000 description 1
- OAIJSZIZWZSQBC-GYZMGTAESA-N lycopene Chemical compound CC(C)=CCC\C(C)=C\C=C\C(\C)=C\C=C\C(\C)=C\C=C\C=C(/C)\C=C\C=C(/C)\C=C\C=C(/C)CCC=C(C)C OAIJSZIZWZSQBC-GYZMGTAESA-N 0.000 description 1
- 235000012661 lycopene Nutrition 0.000 description 1
- 239000001751 lycopene Substances 0.000 description 1
- 210000002751 lymph Anatomy 0.000 description 1
- 210000004698 lymphocyte Anatomy 0.000 description 1
- 210000005075 mammary gland Anatomy 0.000 description 1
- 238000005259 measurement Methods 0.000 description 1
- 230000007246 mechanism Effects 0.000 description 1
- 201000001441 melanoma Diseases 0.000 description 1
- 238000002844 melting Methods 0.000 description 1
- 230000008018 melting Effects 0.000 description 1
- 210000004779 membrane envelope Anatomy 0.000 description 1
- 230000002503 metabolic effect Effects 0.000 description 1
- 229910052751 metal Inorganic materials 0.000 description 1
- 239000002184 metal Substances 0.000 description 1
- 150000002739 metals Chemical class 0.000 description 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 description 1
- 230000000813 microbial effect Effects 0.000 description 1
- 239000008108 microcrystalline cellulose Substances 0.000 description 1
- 229940016286 microcrystalline cellulose Drugs 0.000 description 1
- 235000019813 microcrystalline cellulose Nutrition 0.000 description 1
- 239000004005 microsphere Substances 0.000 description 1
- 235000013336 milk Nutrition 0.000 description 1
- 239000008267 milk Substances 0.000 description 1
- 210000004080 milk Anatomy 0.000 description 1
- 235000010755 mineral Nutrition 0.000 description 1
- 239000011707 mineral Substances 0.000 description 1
- 230000009149 molecular binding Effects 0.000 description 1
- 230000009456 molecular mechanism Effects 0.000 description 1
- 210000003205 muscle Anatomy 0.000 description 1
- 231100000243 mutagenic effect Toxicity 0.000 description 1
- 230000003505 mutagenic effect Effects 0.000 description 1
- 210000004898 n-terminal fragment Anatomy 0.000 description 1
- 201000006478 necrosis of pituitary Diseases 0.000 description 1
- 210000004126 nerve fiber Anatomy 0.000 description 1
- 230000007935 neutral effect Effects 0.000 description 1
- 229920001220 nitrocellulos Polymers 0.000 description 1
- 229910052757 nitrogen Inorganic materials 0.000 description 1
- 125000004433 nitrogen atom Chemical group N* 0.000 description 1
- 125000000018 nitroso group Chemical group N(=O)* 0.000 description 1
- 230000037434 nonsense mutation Effects 0.000 description 1
- 235000015097 nutrients Nutrition 0.000 description 1
- 150000007524 organic acids Chemical class 0.000 description 1
- 235000005985 organic acids Nutrition 0.000 description 1
- 230000008520 organization Effects 0.000 description 1
- 210000000963 osteoblast Anatomy 0.000 description 1
- 230000002018 overexpression Effects 0.000 description 1
- 239000006179 pH buffering agent Substances 0.000 description 1
- 210000000496 pancreas Anatomy 0.000 description 1
- 230000001717 pathogenic effect Effects 0.000 description 1
- 230000037361 pathway Effects 0.000 description 1
- 239000000312 peanut oil Substances 0.000 description 1
- 239000001814 pectin Substances 0.000 description 1
- 235000010987 pectin Nutrition 0.000 description 1
- 229920001277 pectin Polymers 0.000 description 1
- 230000035515 penetration Effects 0.000 description 1
- 230000007030 peptide scission Effects 0.000 description 1
- 238000010647 peptide synthesis reaction Methods 0.000 description 1
- 239000000816 peptidomimetic Substances 0.000 description 1
- 102000013415 peroxidase activity proteins Human genes 0.000 description 1
- 108040007629 peroxidase activity proteins Proteins 0.000 description 1
- 239000003208 petroleum Substances 0.000 description 1
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Natural products OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 description 1
- 229940067107 phenylethyl alcohol Drugs 0.000 description 1
- 235000021317 phosphate Nutrition 0.000 description 1
- 239000002953 phosphate buffered saline Substances 0.000 description 1
- 150000003904 phospholipids Chemical class 0.000 description 1
- 150000003013 phosphoric acid derivatives Chemical class 0.000 description 1
- 102000020233 phosphotransferase Human genes 0.000 description 1
- 238000003976 plant breeding Methods 0.000 description 1
- 210000001778 pluripotent stem cell Anatomy 0.000 description 1
- 229960000502 poloxamer Drugs 0.000 description 1
- 229920001983 poloxamer Polymers 0.000 description 1
- 230000008488 polyadenylation Effects 0.000 description 1
- 238000006116 polymerization reaction Methods 0.000 description 1
- 239000000244 polyoxyethylene sorbitan monooleate Substances 0.000 description 1
- 235000010482 polyoxyethylene sorbitan monooleate Nutrition 0.000 description 1
- 125000004424 polypyridyl Polymers 0.000 description 1
- 210000002729 polyribosome Anatomy 0.000 description 1
- 229940068968 polysorbate 80 Drugs 0.000 description 1
- 229920000053 polysorbate 80 Polymers 0.000 description 1
- 235000010241 potassium sorbate Nutrition 0.000 description 1
- 239000004302 potassium sorbate Substances 0.000 description 1
- 229940069338 potassium sorbate Drugs 0.000 description 1
- 244000144977 poultry Species 0.000 description 1
- 238000002360 preparation method Methods 0.000 description 1
- 239000003755 preservative agent Substances 0.000 description 1
- 238000003825 pressing Methods 0.000 description 1
- 238000007639 printing Methods 0.000 description 1
- XJMOSONTPMZWPB-UHFFFAOYSA-M propidium iodide Chemical compound [I-].[I-].C12=CC(N)=CC=C2C2=CC=C(N)C=C2[N+](CCC[N+](C)(CC)CC)=C1C1=CC=CC=C1 XJMOSONTPMZWPB-UHFFFAOYSA-M 0.000 description 1
- 230000004845 protein aggregation Effects 0.000 description 1
- 108060006633 protein kinase Proteins 0.000 description 1
- 238000001273 protein sequence alignment Methods 0.000 description 1
- 230000012743 protein tagging Effects 0.000 description 1
- 230000006337 proteolytic cleavage Effects 0.000 description 1
- 238000013138 pruning Methods 0.000 description 1
- ZJFJVRPLNAMIKH-UHFFFAOYSA-N pseudo-u Chemical compound O=C1NC(=O)C(C)=CN1C1OC(COP(O)(=S)OC2C(OC(C2)N2C(N=C(N)C=C2)=O)COP(O)(=S)OC2C(OC(C2)N2C(N=C(N)C=C2)=O)COP(O)(=S)OC2C(OC(C2)N2C(N=C(N)C=C2)=O)COP(O)(=S)OC2C(OC(C2)N2C(NC(=O)C(C)=C2)=O)COP(O)(=S)OC2C(OC(C2)N2C3=C(C(NC(N)=N3)=O)N=C2)COP(O)(=S)OC2C(OC(C2)N2C3=NC=NC(N)=C3N=C2)COP(O)(=S)OC2C(OC(C2)N2C3=NC=NC(N)=C3N=C2)COP(O)(=S)OC2C(OC(C2)N2C(N=C(N)C=C2)=O)COP(O)(=S)OC2C(OC(C2)N2C(NC(=O)C(C)=C2)=O)COP(O)(=S)OC2C(OC(C2)N2C(NC(=O)C(C)=C2)=O)COP(O)(=S)OC2C(OC(C2)N2C3=C(C(NC(N)=N3)=O)N=C2)COP(O)(=S)OC2C(OC(C2)N2C3=C(C(NC(N)=N3)=O)N=C2)COP(O)(=S)OC2C(OC(C2)N2C3=C(C(NC(N)=N3)=O)N=C2)COP(O)(=S)OC2C(OC(C2)N2C3=NC=NC(N)=C3N=C2)CO)C(O)C1 ZJFJVRPLNAMIKH-UHFFFAOYSA-N 0.000 description 1
- 230000002685 pulmonary effect Effects 0.000 description 1
- 230000000541 pulsatile effect Effects 0.000 description 1
- 238000004445 quantitative analysis Methods 0.000 description 1
- 239000013645 rAAV1 vector Substances 0.000 description 1
- 239000013646 rAAV2 vector Substances 0.000 description 1
- 108700022487 rRNA Genes Proteins 0.000 description 1
- 238000003127 radioimmunoassay Methods 0.000 description 1
- 238000003753 real-time PCR Methods 0.000 description 1
- 102000005962 receptors Human genes 0.000 description 1
- 108020003175 receptors Proteins 0.000 description 1
- 238000010188 recombinant method Methods 0.000 description 1
- 108010054624 red fluorescent protein Proteins 0.000 description 1
- 238000007670 refining Methods 0.000 description 1
- 101150066583 rep gene Proteins 0.000 description 1
- 230000003252 repetitive effect Effects 0.000 description 1
- 238000002271 resection Methods 0.000 description 1
- 239000011347 resin Substances 0.000 description 1
- 229920005989 resin Polymers 0.000 description 1
- 230000029058 respiratory gaseous exchange Effects 0.000 description 1
- 230000004044 response Effects 0.000 description 1
- 230000016914 response to endoplasmic reticulum stress Effects 0.000 description 1
- 230000000717 retained effect Effects 0.000 description 1
- 238000010839 reverse transcription Methods 0.000 description 1
- 238000012552 review Methods 0.000 description 1
- 239000002336 ribonucleotide Substances 0.000 description 1
- 125000002652 ribonucleotide group Chemical group 0.000 description 1
- 108020004418 ribosomal RNA Proteins 0.000 description 1
- 108091092562 ribozyme Proteins 0.000 description 1
- 229910052707 ruthenium Inorganic materials 0.000 description 1
- 238000007480 sanger sequencing Methods 0.000 description 1
- 230000035945 sensitivity Effects 0.000 description 1
- 230000001953 sensory effect Effects 0.000 description 1
- 238000000926 separation method Methods 0.000 description 1
- 239000008159 sesame oil Substances 0.000 description 1
- 235000011803 sesame oil Nutrition 0.000 description 1
- 230000014639 sexual reproduction Effects 0.000 description 1
- 230000019491 signal transduction Effects 0.000 description 1
- 230000003584 silencer Effects 0.000 description 1
- 239000010703 silicon Substances 0.000 description 1
- 229910052710 silicon Inorganic materials 0.000 description 1
- 210000003491 skin Anatomy 0.000 description 1
- 150000003384 small molecules Chemical class 0.000 description 1
- 235000017557 sodium bicarbonate Nutrition 0.000 description 1
- 229910000030 sodium bicarbonate Inorganic materials 0.000 description 1
- 229910000029 sodium carbonate Inorganic materials 0.000 description 1
- 239000011780 sodium chloride Substances 0.000 description 1
- 238000002415 sodium dodecyl sulfate polyacrylamide gel electrophoresis Methods 0.000 description 1
- 239000007787 solid Substances 0.000 description 1
- 235000010199 sorbic acid Nutrition 0.000 description 1
- 229940075582 sorbic acid Drugs 0.000 description 1
- 239000004334 sorbic acid Substances 0.000 description 1
- 238000011895 specific detection Methods 0.000 description 1
- 230000002269 spontaneous effect Effects 0.000 description 1
- 239000003381 stabilizer Substances 0.000 description 1
- 102000005969 steroid hormone receptors Human genes 0.000 description 1
- 108020003113 steroid hormone receptors Proteins 0.000 description 1
- 238000003860 storage Methods 0.000 description 1
- 229940031000 streptococcus pneumoniae Drugs 0.000 description 1
- 239000005720 sucrose Substances 0.000 description 1
- 229940044609 sulfur dioxide Drugs 0.000 description 1
- 150000003467 sulfuric acid derivatives Chemical class 0.000 description 1
- 235000010269 sulphur dioxide Nutrition 0.000 description 1
- 230000001629 suppression Effects 0.000 description 1
- 239000002344 surface layer Substances 0.000 description 1
- 239000004094 surface-active agent Substances 0.000 description 1
- 230000004083 survival effect Effects 0.000 description 1
- 239000000725 suspension Substances 0.000 description 1
- 208000011580 syndromic disease Diseases 0.000 description 1
- 238000012385 systemic delivery Methods 0.000 description 1
- 230000009885 systemic effect Effects 0.000 description 1
- 238000010809 targeting technique Methods 0.000 description 1
- 229960002180 tetracycline Drugs 0.000 description 1
- 229930101283 tetracycline Natural products 0.000 description 1
- 235000019364 tetracycline Nutrition 0.000 description 1
- 150000003522 tetracyclines Chemical class 0.000 description 1
- 229940124597 therapeutic agent Drugs 0.000 description 1
- 229940094937 thioredoxin Drugs 0.000 description 1
- 229960004072 thrombin Drugs 0.000 description 1
- 230000036962 time dependent Effects 0.000 description 1
- ZCIHMQAPACOQHT-ZGMPDRQDSA-N trans-isorenieratene Natural products CC(=C/C=C/C=C(C)/C=C/C=C(C)/C=C/c1c(C)ccc(C)c1C)C=CC=C(/C)C=Cc2c(C)ccc(C)c2C ZCIHMQAPACOQHT-ZGMPDRQDSA-N 0.000 description 1
- 230000005026 transcription initiation Effects 0.000 description 1
- 230000005030 transcription termination Effects 0.000 description 1
- 230000037426 transcriptional repression Effects 0.000 description 1
- 108091006107 transcriptional repressors Proteins 0.000 description 1
- 238000003151 transfection method Methods 0.000 description 1
- 239000012096 transfection reagent Substances 0.000 description 1
- 238000011426 transformation method Methods 0.000 description 1
- 238000003146 transient transfection Methods 0.000 description 1
- 230000014621 translational initiation Effects 0.000 description 1
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 description 1
- 108091005990 tyrosine-phosphorylated proteins Proteins 0.000 description 1
- 125000001493 tyrosinyl group Chemical group [H]OC1=C([H])C([H])=C(C([H])=C1[H])C([H])([H])C([H])(N([H])[H])C(*)=O 0.000 description 1
- 238000005199 ultracentrifugation Methods 0.000 description 1
- 235000015112 vegetable and seed oil Nutrition 0.000 description 1
- 239000008158 vegetable oil Substances 0.000 description 1
- 210000003501 vero cell Anatomy 0.000 description 1
- 230000009385 viral infection Effects 0.000 description 1
- 239000000277 virosome Substances 0.000 description 1
- 229940088594 vitamin Drugs 0.000 description 1
- 239000011782 vitamin Substances 0.000 description 1
- 235000013343 vitamin Nutrition 0.000 description 1
- 229930003231 vitamin Natural products 0.000 description 1
- 150000003722 vitamin derivatives Chemical class 0.000 description 1
- 239000007762 w/o emulsion Substances 0.000 description 1
- 238000005303 weighing Methods 0.000 description 1
- 238000009736 wetting Methods 0.000 description 1
- 210000005253 yeast cell Anatomy 0.000 description 1
- 235000013618 yogurt Nutrition 0.000 description 1
- 229910052725 zinc Inorganic materials 0.000 description 1
- 239000011701 zinc Substances 0.000 description 1
Images
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
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- 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
- C12N15/1034—Isolating an individual clone by screening libraries
- C12N15/1082—Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, viral vectors
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
-
- 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 [RNase]; Deoxyribonucleases [DNase]
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Crystallography & Structural Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Mycology (AREA)
- Epidemiology (AREA)
- Cell Biology (AREA)
- Virology (AREA)
- Bioinformatics & Computational Biology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Apparatus Associated With Microorganisms And Enzymes (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (13)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361836123P | 2013-06-17 | 2013-06-17 | |
| US61/836,123 | 2013-06-17 | ||
| US201361847537P | 2013-07-17 | 2013-07-17 | |
| US61/847,537 | 2013-07-17 | ||
| US201361862355P | 2013-08-05 | 2013-08-05 | |
| US61/862,355 | 2013-08-05 | ||
| US201361871301P | 2013-08-28 | 2013-08-28 | |
| US61/871,301 | 2013-08-28 | ||
| US201361915383P | 2013-12-12 | 2013-12-12 | |
| PCT/US2013/074667 WO2014093622A2 (en) | 2012-12-12 | 2013-12-12 | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| USPCT/US2013/074667 | 2013-12-12 | ||
| US61/915,383 | 2013-12-12 | ||
| PCT/US2014/041803 WO2014204725A1 (en) | 2013-06-17 | 2014-06-10 | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20160034901A true KR20160034901A (ko) | 2016-03-30 |
Family
ID=52105120
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020167001293A Ceased KR20160034901A (ko) | 2013-06-17 | 2014-06-10 | 서열 조작에 최적화된 crispr-cas 이중 닉카아제 시스템, 방법 및 조성물 |
Country Status (8)
| Country | Link |
|---|---|
| US (3) | US10711285B2 (enExample) |
| EP (2) | EP3011030B1 (enExample) |
| JP (4) | JP6665088B2 (enExample) |
| KR (1) | KR20160034901A (enExample) |
| CN (1) | CN105492611A (enExample) |
| AU (1) | AU2014281027A1 (enExample) |
| CA (1) | CA2915837A1 (enExample) |
| WO (1) | WO2014204725A1 (enExample) |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019088496A3 (ko) * | 2017-10-31 | 2019-06-27 | 주식회사 에이치유비바이오텍 | 내인성 폴리펩타이드 생산을 위한 재조합 세포 및 방법 |
| WO2022169235A1 (ko) * | 2021-02-04 | 2022-08-11 | 연세대학교 산학협력단 | 교정 효율이 개선된 프라임에디팅용 조성물 |
Families Citing this family (345)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| JP2014531908A (ja) | 2011-10-14 | 2014-12-04 | 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 | 構造アッセンブリによる配列決定 |
| ES2991004T3 (es) | 2011-12-22 | 2024-12-02 | Harvard College | Métodos para la detección de analitos |
| FI3597749T3 (fi) | 2012-05-25 | 2023-10-09 | Univ California | Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten |
| KR101656236B1 (ko) * | 2012-10-23 | 2016-09-12 | 주식회사 툴젠 | 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도 |
| PT3363902T (pt) | 2012-12-06 | 2019-12-19 | Sigma Aldrich Co Llc | Modificação e regulação de genoma baseadas em crispr |
| EP4234696A3 (en) | 2012-12-12 | 2023-09-06 | The Broad Institute Inc. | Crispr-cas component systems, methods and compositions for sequence manipulation |
| 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 |
| EP3434776A1 (en) | 2012-12-12 | 2019-01-30 | 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 |
| KR20150105956A (ko) | 2012-12-12 | 2015-09-18 | 더 브로드 인스티튜트, 인코퍼레이티드 | 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화 |
| EP4282970A3 (en) | 2012-12-17 | 2024-01-17 | President and Fellows of Harvard College | Rna-guided human genome engineering |
| US10138509B2 (en) | 2013-03-12 | 2018-11-27 | President And Fellows Of Harvard College | Method for generating a three-dimensional nucleic acid containing matrix |
| AU2014235794A1 (en) * | 2013-03-14 | 2015-10-22 | Caribou Biosciences, Inc. | Compositions and methods of nucleic acid-targeting nucleic acids |
| EP3456831B1 (en) | 2013-04-16 | 2021-07-14 | Regeneron Pharmaceuticals, Inc. | Targeted modification of rat genome |
| US20140356956A1 (en) * | 2013-06-04 | 2014-12-04 | President And Fellows Of Harvard College | RNA-Guided Transcriptional Regulation |
| RU2690935C2 (ru) | 2013-06-04 | 2019-06-06 | Президент Энд Фэллоуз Оф Харвард Коллидж | Направляемая рнк регуляция транскрипции |
| EP3674411A1 (en) | 2013-06-17 | 2020-07-01 | The Broad Institute, Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| CN107995927B (zh) | 2013-06-17 | 2021-07-30 | 布罗德研究所有限公司 | 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途 |
| EP3725885A1 (en) | 2013-06-17 | 2020-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
| CA2915795C (en) | 2013-06-17 | 2021-07-13 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components |
| CN105492611A (zh) * | 2013-06-17 | 2016-04-13 | 布罗德研究所有限公司 | 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物 |
| US11306328B2 (en) | 2013-07-26 | 2022-04-19 | President And Fellows Of Harvard College | Genome engineering |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| 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 |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| EP3988649B1 (en) | 2013-09-18 | 2024-11-27 | Kymab Limited | Methods, cells and organisms |
| WO2015065964A1 (en) | 2013-10-28 | 2015-05-07 | The Broad Institute Inc. | Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof |
| KR20230054509A (ko) | 2013-11-07 | 2023-04-24 | 에디타스 메디신, 인코포레이티드 | 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물 |
| MX388127B (es) | 2013-12-11 | 2025-03-19 | Regeneron Pharma | Metodos y composiciones para la modificacion dirigida de un genoma. |
| EP3080259B1 (en) | 2013-12-12 | 2023-02-01 | The Broad Institute, Inc. | Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation |
| SG10201804973TA (en) | 2013-12-12 | 2018-07-30 | Broad Inst Inc | Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders |
| BR112016013201B1 (pt) * | 2013-12-12 | 2023-01-31 | The Broad Institute, Inc. | Uso de uma composição compreendendo um sistema crispr-cas no tratamento de uma doença genética ocular |
| EP3540051B1 (en) | 2013-12-12 | 2022-08-17 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hsv and viral diseases and disorders. |
| WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| 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 |
| AU2015222944A1 (en) | 2014-02-27 | 2016-09-08 | Massachusetts Institute Of Technology | T cell balance gene expression, compositions of matters and methods of use thereof |
| WO2015134812A1 (en) | 2014-03-05 | 2015-09-11 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
| 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 |
| EP3553176A1 (en) | 2014-03-10 | 2019-10-16 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| EP3122880B1 (en) | 2014-03-26 | 2021-05-05 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
| EP3540061A1 (en) | 2014-04-02 | 2019-09-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| WO2016007839A1 (en) | 2014-07-11 | 2016-01-14 | President And Fellows Of Harvard College | Methods for high-throughput labelling and detection of biological features in situ using microscopy |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| EP3180426B1 (en) | 2014-08-17 | 2019-12-25 | The Broad Institute, Inc. | Genome editing using cas9 nickases |
| 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 |
| 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 |
| WO2016049258A2 (en) | 2014-09-25 | 2016-03-31 | The Broad Institute Inc. | Functional screening with optimized functional crispr-cas systems |
| EP3204496A1 (en) | 2014-10-10 | 2017-08-16 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| CA2963315A1 (en) | 2014-10-15 | 2016-04-21 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for generating or maintaining pluripotent cells |
| EP3212788A2 (en) | 2014-10-27 | 2017-09-06 | The Broad Institute, Inc. | Compositions, methods and use of synthetic lethal screening |
| AU2015342749B2 (en) | 2014-11-07 | 2022-01-27 | Editas Medicine, Inc. | Methods for improving CRISPR/Cas-mediated genome-editing |
| CA3176380A1 (en) | 2014-11-21 | 2016-05-26 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modification using paired guide rnas |
| EP3626832B1 (en) | 2014-11-25 | 2024-06-12 | The Brigham and Women's Hospital, Inc. | Method of identifying a person having a predisposition to or afflicted with a cardiometabolic disease |
| 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 |
| AU2015360502A1 (en) | 2014-12-10 | 2017-06-29 | Regents Of The University Of Minnesota | Genetically modified cells, tissues, and organs for treating disease |
| EP3889260A1 (en) | 2014-12-12 | 2021-10-06 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
| 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) |
| WO2016094872A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Dead guides for crispr transcription factors |
| WO2016094874A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Escorted and functionalized guides for crispr-cas systems |
| EP3234192B1 (en) | 2014-12-19 | 2021-07-14 | The Broad Institute, Inc. | Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing |
| 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 |
| 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 |
| WO2016112351A1 (en) * | 2015-01-09 | 2016-07-14 | Bio-Rad Laboratories, Inc. | Detection of genome editing |
| EP3262193A2 (en) | 2015-02-26 | 2018-01-03 | The Broad Institute Inc. | T cell balance gene expression, compositions of matters and methods of use thereof |
| EP3265560B1 (en) * | 2015-03-02 | 2021-12-08 | Sinai Health System | Homologous recombination factors |
| KR102598856B1 (ko) | 2015-03-03 | 2023-11-07 | 더 제너럴 하스피탈 코포레이션 | 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제 |
| 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 |
| ES2884838T3 (es) | 2015-04-06 | 2021-12-13 | Univ Leland Stanford Junior | ARN guía químicamente modificados para la regulación génica mediada por CRISPR/CAS |
| KR20170135966A (ko) | 2015-04-13 | 2017-12-08 | 맥스시티 인코포레이티드 | 게놈 dna를 변형시키기 위한 방법 및 조성물 |
| JP2018522249A (ja) | 2015-04-24 | 2018-08-09 | エディタス・メディシン、インコーポレイテッド | Cas9分子/ガイドrna分子複合体の評価 |
| DE112016002056T5 (de) | 2015-05-06 | 2018-02-08 | Snipr Technologies Limited | Verändern mikrobieller Populationen und Modifizieren von Mikrobiomen |
| 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 |
| EP3294896A1 (en) | 2015-05-11 | 2018-03-21 | Editas Medicine, Inc. | Optimized crispr/cas9 systems and methods for gene editing in stem cells |
| CA3000155A1 (en) * | 2015-05-29 | 2016-12-08 | Agenovir Corporation | Compositions and methods for cell targeted hpv treatment |
| WO2016196499A1 (en) * | 2015-05-29 | 2016-12-08 | Clark Atlanta University | Human cell lines mutant for zic2 |
| KR102553518B1 (ko) | 2015-06-01 | 2023-07-07 | 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 | Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물 |
| WO2016201047A1 (en) | 2015-06-09 | 2016-12-15 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for improving transplantation |
| WO2016205728A1 (en) | 2015-06-17 | 2016-12-22 | Massachusetts Institute Of Technology | Crispr mediated recording of cellular events |
| AU2016279062A1 (en) | 2015-06-18 | 2019-03-28 | Omar O. Abudayyeh | 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 |
| 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 |
| CA3012631A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Novel crispr enzymes and systems |
| WO2016205745A2 (en) * | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Cell sorting |
| US9790490B2 (en) | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| CA2990699A1 (en) | 2015-06-29 | 2017-01-05 | Ionis Pharmaceuticals, Inc. | Modified crispr rna and modified single crispr rna and uses thereof |
| EP3322804B1 (en) | 2015-07-15 | 2021-09-01 | Rutgers, The State University of New Jersey | Nuclease-independent targeted gene editing platform and uses thereof |
| GB2557123B (en) | 2015-07-31 | 2021-11-03 | Univ Minnesota | Modified cells and methods of therapy |
| WO2017027810A2 (en) | 2015-08-12 | 2017-02-16 | The General Hospital Corporation | Compositions and methods that promote hypoxia or the hypoxia response for treatment and prevention of mitochondrial dysfunction and oxidative stress disorders |
| US11214800B2 (en) | 2015-08-18 | 2022-01-04 | The Broad Institute, Inc. | Methods and compositions for altering function and structure of chromatin loops and/or domains |
| US20170072025A1 (en) | 2015-09-10 | 2017-03-16 | Youhealth Biotech, Limited | Methods and compositions for the treatment of glaucoma |
| CA2999500A1 (en) | 2015-09-24 | 2017-03-30 | Editas Medicine, Inc. | Use of exonucleases to improve crispr/cas-mediated genome editing |
| WO2017069958A2 (en) | 2015-10-09 | 2017-04-27 | The Brigham And Women's Hospital, Inc. | Modulation of novel immune checkpoint targets |
| KR102761827B1 (ko) | 2015-10-22 | 2025-02-03 | 더 브로드 인스티튜트, 인코퍼레이티드 | 타입 vi-b crispr 효소 및 시스템 |
| US12043852B2 (en) | 2015-10-23 | 2024-07-23 | President And Fellows Of Harvard College | Evolved Cas9 proteins for gene editing |
| EP3368687B1 (en) | 2015-10-27 | 2021-09-29 | The Broad Institute, Inc. | Compositions and methods for targeting cancer-specific sequence variations |
| WO2017075451A1 (en) | 2015-10-28 | 2017-05-04 | The Broad Institute Inc. | Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1 |
| US11092607B2 (en) | 2015-10-28 | 2021-08-17 | The Board Institute, Inc. | Multiplex analysis of single cell constituents |
| WO2017075294A1 (en) | 2015-10-28 | 2017-05-04 | The Board Institute Inc. | Assays for massively combinatorial perturbation profiling and cellular circuit reconstruction |
| EP3368689B1 (en) | 2015-10-28 | 2020-06-17 | The Broad Institute, Inc. | Composition for modulating immune responses by use of immune cell gene signature |
| WO2017075465A1 (en) | 2015-10-28 | 2017-05-04 | The Broad Institute Inc. | Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3 |
| JP6882282B2 (ja) | 2015-11-03 | 2021-06-02 | 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 | 三次元核酸含有マトリックスの立体撮像のための方法と装置 |
| EP3374501B1 (en) * | 2015-11-11 | 2023-07-12 | Lonza Ltd | Crispr-associated (cas) proteins with reduced immunogenicity |
| KR102877920B1 (ko) | 2015-11-16 | 2025-10-30 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법 |
| US11001622B2 (en) | 2015-11-19 | 2021-05-11 | The Brigham And Women's Hospital, Inc. | Method of treating autoimmune disease with lymphocyte antigen CD5-like (CD5L) protein |
| WO2017106569A1 (en) | 2015-12-18 | 2017-06-22 | The Regents Of The University Of California | Modified site-directed modifying polypeptides and methods of use thereof |
| CN109072218B (zh) * | 2015-12-18 | 2023-04-18 | 国立研究开发法人科学技术振兴机构 | 基因修饰非人生物、卵细胞、受精卵以及目的基因的修饰方法 |
| EA201891532A1 (ru) * | 2015-12-28 | 2019-01-31 | Новартис Аг | Композиции и способы лечения гемоглобинопатий |
| JP2019500394A (ja) | 2015-12-30 | 2019-01-10 | ノバルティス アーゲー | 有効性が増強された免疫エフェクター細胞治療 |
| WO2017127807A1 (en) * | 2016-01-22 | 2017-07-27 | The Broad Institute Inc. | Crystal structure of crispr cpf1 |
| EP3420102B1 (en) | 2016-02-22 | 2024-04-03 | Massachusetts Institute of Technology | Methods for identifying and modulating immune phenotypes |
| 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 |
| US11427861B2 (en) | 2016-03-17 | 2022-08-30 | Massachusetts Institute Of Technology | Methods for identifying and modulating co-occurant cellular phenotypes |
| EP3219799A1 (en) | 2016-03-17 | 2017-09-20 | IMBA-Institut für Molekulare Biotechnologie GmbH | Conditional crispr sgrna expression |
| EP3433363A1 (en) | 2016-03-25 | 2019-01-30 | Editas Medicine, Inc. | Genome editing systems comprising repair-modulating enzyme molecules and methods of their use |
| EP3433364A1 (en) | 2016-03-25 | 2019-01-30 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| EP4047092B1 (en) | 2016-04-13 | 2025-07-30 | Editas Medicine, Inc. | Cas9 fusion molecules, gene editing systems, and methods of use thereof |
| EP4613756A3 (en) | 2016-04-25 | 2025-11-12 | President And Fellows Of Harvard College | Hybridization chain reaction methods for in situ molecular detection |
| WO2017201476A1 (en) | 2016-05-20 | 2017-11-23 | Regeneron Pharmaceuticals, Inc. | Methods for breaking immunological tolerance using multiple guide rnas |
| CA3018294A1 (en) * | 2016-06-01 | 2017-12-07 | Excision Biotherapeutics, Inc. | Compositions and methods of treatment for lytic and lysogenic viruses |
| GB201609811D0 (en) | 2016-06-05 | 2016-07-20 | Snipr Technologies Ltd | Methods, cells, systems, arrays, RNA and kits |
| US10767175B2 (en) | 2016-06-08 | 2020-09-08 | Agilent Technologies, Inc. | High specificity genome editing using chemically modified guide RNAs |
| 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 |
| WO2017217768A1 (ko) * | 2016-06-15 | 2017-12-21 | 주식회사 툴젠 | 온타겟 및 오프타겟의 다중 타겟 시스템을 이용하는, 표적 특이적 유전자 가위 스크리닝 방법 및 이의 용도 |
| CA3028074A1 (en) * | 2016-06-16 | 2018-12-20 | Oslo Universitetssykehus Hf | Improved gene editing |
| CN107513538A (zh) * | 2016-06-17 | 2017-12-26 | 北京大学 | 基因敲除方法 |
| JP7267013B2 (ja) | 2016-06-17 | 2023-05-01 | ザ・ブロード・インスティテュート・インコーポレイテッド | Vi型crisprオルソログ及び系 |
| WO2017216771A2 (en) * | 2016-06-17 | 2017-12-21 | Genesis Technologies Limited | Crispr-cas system, materials and methods |
| 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 |
| DK3474669T3 (da) * | 2016-06-24 | 2022-06-27 | Univ Colorado Regents | Fremgangsmåde til generering af stregkodede kombinatoriske biblioteker |
| CN105907777A (zh) * | 2016-06-28 | 2016-08-31 | 中国科学院海洋研究所 | 一种Cas9核酸内切酶分泌型表达载体及其构建方法和应用 |
| US11359234B2 (en) | 2016-07-01 | 2022-06-14 | Microsoft Technology Licensing, Llc | Barcoding sequences for identification of gene expression |
| US10669558B2 (en) | 2016-07-01 | 2020-06-02 | Microsoft Technology Licensing, Llc | Storage through iterative DNA editing |
| US20180004537A1 (en) * | 2016-07-01 | 2018-01-04 | Microsoft Technology Licensing, Llc | Molecular State Machines |
| CA3030783A1 (en) | 2016-07-13 | 2018-01-18 | Vertex Pharmaceuticals Incorporated | Methods, compositions and kits for increasing genome editing efficiency |
| EP3491131B1 (en) * | 2016-07-29 | 2022-05-25 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | Targeted in situ protein diversification by site directed dna cleavage and repair |
| WO2018026976A1 (en) | 2016-08-02 | 2018-02-08 | Editas Medicine, Inc. | Compositions and methods for treating cep290 associated disease |
| KR20250103795A (ko) | 2016-08-03 | 2025-07-07 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| CN109804066A (zh) | 2016-08-09 | 2019-05-24 | 哈佛大学的校长及成员们 | 可编程cas9-重组酶融合蛋白及其用途 |
| EP3498834A4 (en) * | 2016-08-10 | 2020-06-17 | Genahead Bio, Inc. | METHOD FOR MODIFYING A TARGET SITE IN THE GENOME OF A EUKARYOTE CELL, AND METHOD FOR DETECTING THE PRESENCE OR ABSENCE OF A NUCLEIC ACID SEQUENCE TO BE DETECTED AT A TARGET SITE |
| US12431216B2 (en) | 2016-08-17 | 2025-09-30 | Broad Institute, Inc. | Methods for identifying class 2 crispr-cas systems |
| US11352647B2 (en) | 2016-08-17 | 2022-06-07 | The Broad Institute, Inc. | Crispr enzymes and systems |
| WO2018035364A1 (en) | 2016-08-17 | 2018-02-22 | The Broad Institute Inc. | Product and methods useful for modulating and evaluating immune responses |
| WO2018035387A1 (en) | 2016-08-17 | 2018-02-22 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| WO2018049025A2 (en) | 2016-09-07 | 2018-03-15 | The Broad Institute Inc. | Compositions and methods for evaluating and modulating immune responses |
| US12188059B2 (en) | 2016-09-13 | 2025-01-07 | The Broad Institute, Inc. | Proximity-dependent biotinylation and uses thereof |
| EP3516056B1 (en) * | 2016-09-23 | 2024-11-27 | DSM IP Assets B.V. | A guide-rna expression system for a host cell |
| US20190225974A1 (en) | 2016-09-23 | 2019-07-25 | BASF Agricultural Solutions Seed US LLC | Targeted genome optimization in plants |
| US12447213B2 (en) | 2016-10-07 | 2025-10-21 | The Broad Institute, Inc. | Modulation of novel immune checkpoint targets |
| AU2017342543B2 (en) | 2016-10-14 | 2024-06-27 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| 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 |
| WO2018089386A1 (en) | 2016-11-11 | 2018-05-17 | The Broad Institute, Inc. | Modulation of intestinal epithelial cell differentiation, maintenance and/or function through t cell action |
| WO2018107103A1 (en) | 2016-12-09 | 2018-06-14 | The Broad Institute, Inc. | Crispr-systems for modifying a trait of interest in a plant |
| US12286727B2 (en) | 2016-12-19 | 2025-04-29 | Editas Medicine, Inc. | Assessing nuclease cleavage |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| JP7219972B2 (ja) | 2017-01-05 | 2023-02-09 | ラトガース,ザ ステート ユニバーシティ オブ ニュー ジャージー | Dna二本鎖切断に非依存的な標的化遺伝子編集プラットフォームおよびその用途 |
| EP3565907B1 (en) | 2017-01-06 | 2022-05-04 | Editas Medicine, Inc. | Methods of assessing nuclease cleavage |
| KR102515727B1 (ko) * | 2017-01-11 | 2023-03-30 | 주식회사 툴젠 | 중첩된 가이드핵산을 이용한 표적 핵산에 특정 핵산 서열을 삽입하기 위한 조성물 및 방법 |
| US20180258418A1 (en) * | 2017-01-17 | 2018-09-13 | Institute For Basic Science | Method of identifying genome-wide off-target sites of base editors by detecting single strand breaks in genomic dna |
| 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 |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Cancer vaccine |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| WO2018170184A1 (en) | 2017-03-14 | 2018-09-20 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| EP4361261A3 (en) | 2017-03-15 | 2024-07-10 | The Broad Institute Inc. | Novel cas13b orthologues crispr enzymes and systems |
| WO2018170515A1 (en) | 2017-03-17 | 2018-09-20 | The Broad Institute, Inc. | Methods for identifying and modulating co-occurant cellular phenotypes |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| WO2018175924A1 (en) | 2017-03-24 | 2018-09-27 | The Broad Institute, Inc. | Methods and compositions for regulating innate lymphoid cell inflammatory responses |
| KR102758434B1 (ko) | 2017-03-30 | 2025-01-21 | 고쿠리츠 다이가쿠 호진 교토 다이가쿠 | 게놈 편집에 의한 엑손 스키핑 유도 방법 |
| WO2018183908A1 (en) | 2017-03-31 | 2018-10-04 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for treating ovarian tumors |
| US11913075B2 (en) | 2017-04-01 | 2024-02-27 | The Broad Institute, Inc. | Methods and compositions for detecting and modulating an immunotherapy resistance gene signature in cancer |
| US20200071773A1 (en) | 2017-04-12 | 2020-03-05 | Massachusetts Eye And Ear Infirmary | Tumor signature for metastasis, compositions of matter methods of use thereof |
| US12331319B2 (en) | 2017-04-12 | 2025-06-17 | The Broad Institute, Inc. | Respiratory and sweat gland ionocytes |
| CN110799645B (zh) | 2017-04-12 | 2024-08-02 | 博德研究所 | 新型vi型crispr直系同源物和系统 |
| WO2018191750A2 (en) | 2017-04-14 | 2018-10-18 | The Broad Institute Inc. | Novel delivery of large payloads |
| WO2018195019A1 (en) | 2017-04-18 | 2018-10-25 | The Broad Institute Inc. | Compositions for detecting secretion and methods of use |
| US12058986B2 (en) | 2017-04-20 | 2024-08-13 | Egenesis, Inc. | Method for generating a genetically modified pig with inactivated porcine endogenous retrovirus (PERV) elements |
| US12208140B2 (en) | 2017-04-21 | 2025-01-28 | The Broad Institute, Inc. | Targeted delivery to beta cells |
| EP3615672A1 (en) | 2017-04-28 | 2020-03-04 | Editas Medicine, Inc. | Methods and systems for analyzing guide rna molecules |
| WO2018204777A2 (en) | 2017-05-05 | 2018-11-08 | The Broad Institute, Inc. | Methods for identification and modification of lncrna associated with target genotypes and phenotypes |
| WO2018209158A2 (en) | 2017-05-10 | 2018-11-15 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| US12226479B2 (en) | 2017-05-11 | 2025-02-18 | The General Hospital Corporation | Methods and compositions of use of CD8+ tumor infiltrating lymphocyte subtypes and gene signatures thereof |
| 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 |
| US12297436B2 (en) | 2017-05-18 | 2025-05-13 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| CN111417727A (zh) | 2017-05-18 | 2020-07-14 | 博德研究所 | 用于靶向核酸编辑的系统、方法和组合物 |
| WO2018218188A2 (en) | 2017-05-25 | 2018-11-29 | The General Hospital Corporation | Base editors with improved precision and specificity |
| WO2018226762A1 (en) | 2017-06-05 | 2018-12-13 | Fred Hutchinson Cancer Research Center | Genomic safe harbors for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies |
| CN110997908A (zh) | 2017-06-09 | 2020-04-10 | 爱迪塔斯医药公司 | 工程化的cas9核酸酶 |
| EP3638218A4 (en) | 2017-06-14 | 2021-06-09 | The Broad Institute, Inc. | COMPOSITIONS AND METHOD OF TARGETING COMPLEMENTING COMPONENT 3 FOR INHIBITION OF TUMOR GROWTH |
| JP7454494B2 (ja) | 2017-06-26 | 2024-03-22 | ザ・ブロード・インスティテュート・インコーポレイテッド | 標的化された核酸編集のためのcrispr/cas-アデニンデアミナーゼ系の組成物、系及び方法 |
| US11541072B2 (en) | 2017-06-27 | 2023-01-03 | Massachusetts Eye And Ear Infirmary | AAV-CRISPR/Cas9 genome editing of VEGFR2 for treating ocular diseases |
| WO2019006418A2 (en) | 2017-06-30 | 2019-01-03 | Intima Bioscience, Inc. | ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY |
| US20200140896A1 (en) | 2017-06-30 | 2020-05-07 | Novartis Ag | Methods for the treatment of disease with gene editing systems |
| EP3652312A1 (en) | 2017-07-14 | 2020-05-20 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| US12049643B2 (en) | 2017-07-14 | 2024-07-30 | The Broad Institute, Inc. | Methods and compositions for modulating cytotoxic lymphocyte activity |
| WO2019018440A1 (en) | 2017-07-17 | 2019-01-24 | The Broad Institute, Inc. | HUMAN COLON CELL ATLAS IN GOOD HEALTH AND WITH HEMORRHAGIC RECTO-COLITIS |
| US12239646B2 (en) | 2017-07-19 | 2025-03-04 | Dana-Farber Cancer Institute, Inc. | Cancer diagnostic and treatment |
| CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| EP3672612A4 (en) | 2017-08-23 | 2021-09-29 | The General Hospital Corporation | GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES WITH MODIFIED PAM SPECIFICITY |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| KR102338449B1 (ko) | 2017-09-21 | 2021-12-10 | 더 브로드 인스티튜트, 인코퍼레이티드 | 표적화된 핵산 편집을 위한 시스템, 방법, 및 조성물 |
| SG11202002562QA (en) | 2017-09-29 | 2020-04-29 | Intellia Therapeutics Inc | Polynucleotides, compositions, and methods for genome editing |
| WO2019070755A1 (en) | 2017-10-02 | 2019-04-11 | The Broad Institute, Inc. | METHODS AND COMPOSITIONS FOR DETECTING AND MODULATING A GENETIC SIGNATURE OF IMMUNOTHERAPY RESISTANCE IN CANCER |
| WO2019071054A1 (en) | 2017-10-04 | 2019-04-11 | The Broad Institute, Inc. | METHODS AND COMPOSITIONS FOR MODIFYING THE FUNCTION AND STRUCTURE OF BUCKLES AND / OR CHROMATIN DOMAINS |
| US11680296B2 (en) | 2017-10-16 | 2023-06-20 | Massachusetts Institute Of Technology | Mycobacterium tuberculosis host-pathogen interaction |
| CA3082251A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | Uses of adenosine base editors |
| KR102723182B1 (ko) | 2017-10-20 | 2024-10-31 | 프레드 허친슨 캔서 센터 | 선택된 항체를 발현하도록 유전자 변형된 b 세포를 생산하기 위한 시스템 및 방법 |
| WO2019135816A2 (en) | 2017-10-23 | 2019-07-11 | The Broad Institute, Inc. | Novel nucleic acid modifiers |
| WO2019089803A1 (en) | 2017-10-31 | 2019-05-09 | The Broad Institute, Inc. | Methods and compositions for studying cell evolution |
| US20210180053A1 (en) | 2017-11-01 | 2021-06-17 | Novartis Ag | Synthetic rnas and methods of use |
| WO2019094984A1 (en) | 2017-11-13 | 2019-05-16 | The Broad Institute, Inc. | Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells |
| WO2019094955A1 (en) | 2017-11-13 | 2019-05-16 | The Broad Institute, Inc. | Methods and compositions for targeting developmental and oncogenic programs in h3k27m gliomas |
| CN120310773A (zh) * | 2017-11-16 | 2025-07-15 | 阿斯利康(瑞典)有限公司 | 用于改善基于Cas9的敲入策略的有效性的组合物和方法 |
| US11332736B2 (en) | 2017-12-07 | 2022-05-17 | The Broad Institute, Inc. | Methods and compositions for multiplexing single cell and single nuclei sequencing |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| US11994512B2 (en) | 2018-01-04 | 2024-05-28 | Massachusetts Institute Of Technology | Single-cell genomic methods to generate ex vivo cell systems that recapitulate in vivo biology with improved fidelity |
| MX2020007466A (es) | 2018-01-12 | 2020-11-12 | Basf Se | Gen subyacente al qtl de la cantidad de espiguillas por espiga en trigo en el cromosoma 7a. |
| CN111741955B (zh) | 2018-01-17 | 2024-02-23 | 沃泰克斯药物股份有限公司 | Dna-pk抑制剂 |
| EP3740480B1 (en) | 2018-01-17 | 2024-03-06 | Vertex Pharmaceuticals Incorporated | Dna-pk inhibitors |
| JP7483612B2 (ja) | 2018-01-17 | 2024-05-15 | バーテックス ファーマシューティカルズ インコーポレイテッド | ゲノム編集効率を増加するためのキノキサリノン化合物、組成物、方法、およびキット |
| SG11202006722RA (en) | 2018-01-31 | 2020-08-28 | Res Inst Nationwide Childrens Hospital | Gene therapy for limb-girdle muscular dystrophy type 2c |
| SG11202008956XA (en) | 2018-03-14 | 2020-10-29 | Editas Medicine Inc | Systems and methods for the treatment of hemoglobinopathies |
| US10760075B2 (en) | 2018-04-30 | 2020-09-01 | Snipr Biome Aps | Treating and preventing microbial infections |
| US12338486B2 (en) | 2018-04-19 | 2025-06-24 | Massachusetts Institute Of Technology | Single-stranded break detection in double-stranded DNA |
| SI3560330T1 (sl) | 2018-04-24 | 2022-08-31 | KWS SAAT SE & Co. KGaA | Rastline z izboljšano prebavljivostjo in markerskimi haplotipi |
| US11957695B2 (en) | 2018-04-26 | 2024-04-16 | The Broad Institute, Inc. | Methods and compositions targeting glucocorticoid signaling for modulating immune responses |
| WO2019210268A2 (en) | 2018-04-27 | 2019-10-31 | The Broad Institute, Inc. | Sequencing-based proteomics |
| WO2019210491A1 (zh) * | 2018-05-03 | 2019-11-07 | 深圳市博奥康生物科技有限公司 | CRISPR/Cas9靶向敲除人CVAP基因及其特异性gRNA |
| US20210386829A1 (en) | 2018-05-04 | 2021-12-16 | The Broad Institute, Inc. | Compositions and methods for modulating cgrp signaling to regulate innate lymphoid cell inflammatory responses |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| US20210371932A1 (en) | 2018-06-01 | 2021-12-02 | Massachusetts Institute Of Technology | Methods and compositions for detecting and modulating microenvironment gene signatures from the csf of metastasis patients |
| EP3578658A1 (en) * | 2018-06-08 | 2019-12-11 | Johann Wolfgang Goethe-Universität Frankfurt | Method for generating a gene editing vector with fixed guide rna pairs |
| US12036240B2 (en) | 2018-06-14 | 2024-07-16 | The Broad Institute, Inc. | Compositions and methods targeting complement component 3 for inhibiting tumor growth |
| SG11202012785VA (en) | 2018-06-26 | 2021-01-28 | Broad Inst Inc | Crispr double nickase based amplification compositions, systems, and methods |
| CN112543812A (zh) | 2018-06-26 | 2021-03-23 | 麻省理工学院 | 基于crispr效应系统的扩增方法、系统和诊断 |
| KR20210024009A (ko) | 2018-06-26 | 2021-03-04 | 더 브로드 인스티튜트, 인코퍼레이티드 | Crispr/cas 및 트랜스포사제 기반 증폭 조성물, 시스템 및 방법 |
| CA3104856A1 (en) | 2018-06-29 | 2020-01-02 | Editas Medicine, Inc. | Synthetic guide molecules, compositions and methods relating thereto |
| KR20210028162A (ko) | 2018-06-29 | 2021-03-11 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법 |
| CA3106162A1 (en) * | 2018-07-13 | 2020-01-16 | Allele Biotechnology And Pharmaceuticals, Inc. | Methods of achieving high specificity of genome editing |
| CA3111432A1 (en) | 2018-07-31 | 2020-02-06 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
| CN108998406B (zh) * | 2018-08-03 | 2022-05-10 | 福州大学 | 一种人类原代培养细胞基因组编辑、定点基因敲入方法 |
| CN108949830B (zh) * | 2018-08-03 | 2021-11-26 | 福州大学 | 一种在鱼类中实现基因组编辑、精确定点基因敲入的方法 |
| EP3833761A1 (en) | 2018-08-07 | 2021-06-16 | The Broad Institute, Inc. | Novel cas12b enzymes and systems |
| WO2020041380A1 (en) | 2018-08-20 | 2020-02-27 | The Broad Institute, Inc. | Methods and compositions for optochemical control of crispr-cas9 |
| US20210324357A1 (en) | 2018-08-20 | 2021-10-21 | The Brigham And Women's Hospital, Inc. | Degradation domain modifications for spatio-temporal control of rna-guided nucleases |
| EP3847650A1 (en) | 2018-09-06 | 2021-07-14 | The Broad Institute, Inc. | Nucleic acid assemblies for use in targeted delivery |
| US12398402B2 (en) | 2018-09-12 | 2025-08-26 | Fred Hutchinson Cancer Center | Reducing CD33 expression to selectively protect therapeutic cells |
| JP7344300B2 (ja) | 2018-09-18 | 2023-09-13 | ブイエヌブイ ニューコ インク. | Arcベースのカプシドおよびその使用 |
| WO2020076976A1 (en) | 2018-10-10 | 2020-04-16 | Readcoor, Inc. | Three-dimensional spatial molecular indexing |
| US20220411783A1 (en) | 2018-10-12 | 2022-12-29 | The Broad Institute, Inc. | Method for extracting nuclei or whole cells from formalin-fixed paraffin-embedded tissues |
| US20210379057A1 (en) | 2018-10-16 | 2021-12-09 | Massachusetts Institute Of Technology | Nutlin-3a for use in treating a mycobacterium tuberculosis infection |
| BR112021007301A2 (pt) | 2018-10-18 | 2021-07-27 | Intellia Therapeutics, Inc. | composições e métodos para expressar fator ix |
| EP3867381A2 (en) * | 2018-10-18 | 2021-08-25 | Intellia Therapeutics, Inc. | Compositions and methods for transgene expression from an albumin locus |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| CN109411022A (zh) * | 2018-11-07 | 2019-03-01 | 宁夏农林科学院农业生物技术研究中心(宁夏农业生物技术重点实验室) | 一种基于字符切片技术的含PAM结构gRNA靶向序列筛选方法及应用 |
| US12165743B2 (en) | 2018-11-09 | 2024-12-10 | The Broad Institute, Inc. | Compressed sensing for screening and tissue imaging |
| US12402610B2 (en) | 2018-11-09 | 2025-09-02 | The Broad Institute, Inc. | Methods and compositions for modulating innate lymphoid cell pathogenic effectors |
| WO2020131862A1 (en) | 2018-12-17 | 2020-06-25 | The Broad Institute, Inc. | Crispr-associated transposase systems and methods of use thereof |
| US20220062394A1 (en) | 2018-12-17 | 2022-03-03 | The Broad Institute, Inc. | Methods for identifying neoantigens |
| CN109679953B (zh) * | 2018-12-28 | 2022-08-16 | 广州赛业百沐生物科技有限公司 | 利用CRISPR-Cas9系统制得基因点突变动物模型胚胎的靶序列组、载体和方法 |
| US11739156B2 (en) | 2019-01-06 | 2023-08-29 | The Broad Institute, Inc. Massachusetts Institute of Technology | Methods and compositions for overcoming immunosuppression |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| WO2020163396A1 (en) | 2019-02-04 | 2020-08-13 | The General Hospital Corporation | Adenine dna base editor variants with reduced off-target rna editing |
| CA3131390A1 (en) | 2019-02-26 | 2020-09-03 | Research Institute Of Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy |
| WO2020186101A1 (en) | 2019-03-12 | 2020-09-17 | The Broad Institute, Inc. | Detection means, compositions and methods for modulating synovial sarcoma cells |
| US12419915B2 (en) | 2019-03-13 | 2025-09-23 | The Broad Institute, Inc. | Microglial progenitors for regeneration of functional microglia in the central nervous system and therapeutics uses thereof |
| EP3937969A1 (en) | 2019-03-14 | 2022-01-19 | The Broad Institute, Inc. | Compositions and methods for modulating cgrp signaling to regulate intestinal innate lymphoid cells |
| WO2020191069A1 (en) | 2019-03-18 | 2020-09-24 | The Broad Institute, Inc. | Modulation of type 2 immunity by targeting clec-2 signaling |
| EP3942023A1 (en) | 2019-03-18 | 2022-01-26 | The Broad Institute, Inc. | Compositions and methods for modulating metabolic regulators of t cell pathogenicity |
| DE112020001306T5 (de) | 2019-03-19 | 2022-01-27 | Massachusetts Institute Of Technology | Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen |
| EP3947687A1 (en) | 2019-03-29 | 2022-02-09 | Massachusetts Institute of Technology | Constructs for continuous monitoring of live cells |
| US20220162649A1 (en) | 2019-04-01 | 2022-05-26 | The Broad Institute, Inc. | Novel nucleic acid modifiers |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| EP3969607A1 (en) | 2019-05-13 | 2022-03-23 | KWS SAAT SE & Co. KGaA | Drought tolerance in corn |
| US20220249701A1 (en) | 2019-05-14 | 2022-08-11 | The Broad Institute, Inc. | Compositions and methods for targeting multinucleated cells |
| US20220235340A1 (en) | 2019-05-20 | 2022-07-28 | The Broad Institute, Inc. | Novel crispr-cas systems and uses thereof |
| WO2020236972A2 (en) | 2019-05-20 | 2020-11-26 | The Broad Institute, Inc. | Non-class i multi-component nucleic acid targeting systems |
| AR118995A1 (es) | 2019-05-25 | 2021-11-17 | Kws Saat Se & Co Kgaa | Mejorador de la inducción de haploides |
| WO2020243371A1 (en) | 2019-05-28 | 2020-12-03 | Massachusetts Institute Of Technology | Methods and compositions for modulating immune responses |
| WO2020243661A1 (en) | 2019-05-31 | 2020-12-03 | The Broad Institute, Inc. | Methods for treating metabolic disorders by targeting adcy5 |
| US20220257796A1 (en) | 2019-07-02 | 2022-08-18 | Fred Hutchinson Cancer Research Center | Recombinant ad35 vectors and related gene therapy improvements |
| EP3772542A1 (en) | 2019-08-07 | 2021-02-10 | KWS SAAT SE & Co. KGaA | Modifying genetic variation in crops by modulating the pachytene checkpoint protein 2 |
| WO2021030627A1 (en) | 2019-08-13 | 2021-02-18 | The General Hospital Corporation | Methods for predicting outcomes of checkpoint inhibition and treatment thereof |
| US12421557B2 (en) | 2019-08-16 | 2025-09-23 | The Broad Institute, Inc. | Methods for predicting outcomes and treating colorectal cancer using a cell atlas |
| HRP20240505T1 (hr) | 2019-08-21 | 2024-07-05 | Research Institute At Nationwide Children's Hospital | Dostava alfa-sarkoglikana vektorom povezanim s adenovirusom i liječenje mišićne distrofije |
| WO2021041922A1 (en) | 2019-08-30 | 2021-03-04 | The Broad Institute, Inc. | Crispr-associated mu transposase systems |
| AU2020348879A1 (en) | 2019-09-20 | 2022-04-14 | Massachusetts Institute Of Technology | Novel type VI CRISPR enzymes and systems |
| US11981922B2 (en) | 2019-10-03 | 2024-05-14 | Dana-Farber Cancer Institute, Inc. | Methods and compositions for the modulation of cell interactions and signaling in the tumor microenvironment |
| US12195725B2 (en) | 2019-10-03 | 2025-01-14 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for modulating and detecting tissue specific TH17 cell pathogenicity |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| EP4045522A1 (en) | 2019-10-17 | 2022-08-24 | KWS SAAT SE & Co. KGaA | Enhanced disease resistance of crops by downregulation of repressor genes |
| CN112725348B (zh) * | 2019-10-28 | 2022-04-01 | 安徽省农业科学院水稻研究所 | 一种提高水稻单碱基编辑效率的基因、方法及应用 |
| US11844800B2 (en) | 2019-10-30 | 2023-12-19 | Massachusetts Institute Of Technology | Methods and compositions for predicting and preventing relapse of acute lymphoblastic leukemia |
| US12195723B2 (en) | 2019-11-08 | 2025-01-14 | The Broad Institute, Inc. | Engineered antigen presenting cells and uses thereof |
| WO2021146641A1 (en) | 2020-01-17 | 2021-07-22 | The Broad Institute, Inc. | Small type ii-d cas proteins and methods of use thereof |
| US12165747B2 (en) | 2020-01-23 | 2024-12-10 | The Broad Institute, Inc. | Molecular spatial mapping of metastatic tumor microenvironment |
| US12264341B2 (en) | 2020-01-24 | 2025-04-01 | The General Hospital Corporation | CRISPR-Cas enzymes with enhanced on-target activity |
| EP4093864A4 (en) | 2020-01-24 | 2024-04-10 | The General Hospital Corporation | Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants |
| AU2021249119A1 (en) * | 2020-03-31 | 2022-10-20 | Arbor Biotechnologies, Inc. | Compositions comprising a Cas12i2 variant polypeptide and uses thereof |
| JP2023525304A (ja) | 2020-05-08 | 2023-06-15 | ザ ブロード インスティテュート,インコーポレーテッド | 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物 |
| WO2021239986A1 (en) | 2020-05-29 | 2021-12-02 | KWS SAAT SE & Co. KGaA | Plant haploid induction |
| AU2021321586A1 (en) | 2020-08-07 | 2023-03-16 | Dana-Farber Cancer Institute, Inc. | Therapeutic targeting of phosphate dysregulation in cancer via the XPR1:KIDINS220 protein complex |
| US20220049303A1 (en) | 2020-08-17 | 2022-02-17 | Readcoor, Llc | Methods and systems for spatial mapping of genetic variants |
| EP4047095A1 (en) * | 2021-02-22 | 2022-08-24 | Eberhard Karls Universität Tübingen Medizinische Fakultät | Method and composition for a targeted gene knockout |
| CN113403309B (zh) * | 2021-05-24 | 2022-08-16 | 珠海舒桐医疗科技有限公司 | 非同源双链寡聚核苷酸片段在基因敲除系统中的应用 |
| US20240102007A1 (en) | 2021-06-01 | 2024-03-28 | Arbor Biotechnologies, Inc. | Gene editing systems comprising a crispr nuclease and uses thereof |
| US20250179591A1 (en) | 2021-07-30 | 2025-06-05 | KWS SAAT SE & Co. KGaA | Plants with improved digestibility and marker haplotypes |
| EP4392557A1 (en) * | 2021-08-23 | 2024-07-03 | Max-Delbrück-Centrum für Molekulare Medizin in der Helmholtz-Gemeinschaft | Method for cas9 nickase-mediated gene editing |
| US11884915B2 (en) | 2021-09-10 | 2024-01-30 | Agilent Technologies, Inc. | Guide RNAs with chemical modification for prime editing |
| EP4219726A1 (en) | 2021-10-15 | 2023-08-02 | Research Institute at Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| EP4426828A1 (en) | 2021-11-01 | 2024-09-11 | Tome Biosciences, Inc. | Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo |
| EP4437092A1 (en) | 2021-11-26 | 2024-10-02 | Epigenic Therapeutics Inc. | Method of modulating pcsk9 and uses thereof |
| CA3244180A1 (en) | 2021-12-22 | 2023-06-29 | Tome Biosciences Inc | CO-ADMINISTRATION OF A GENE EDITOR CONSTRUCTION AND A PATTERN DONOR |
| EP4503923A1 (en) | 2022-04-04 | 2025-02-12 | The Regents of the University of California | Genetic complementation compositions and methods |
| WO2023205744A1 (en) | 2022-04-20 | 2023-10-26 | Tome Biosciences, Inc. | Programmable gene insertion compositions |
| WO2023215831A1 (en) | 2022-05-04 | 2023-11-09 | Tome Biosciences, Inc. | Guide rna compositions for programmable gene insertion |
| WO2023225670A2 (en) | 2022-05-20 | 2023-11-23 | Tome Biosciences, Inc. | Ex vivo programmable gene insertion |
| GB202209518D0 (en) | 2022-06-29 | 2022-08-10 | Snipr Biome Aps | Treating & preventing E coli infections |
| WO2024020587A2 (en) | 2022-07-22 | 2024-01-25 | Tome Biosciences, Inc. | Pleiopluripotent stem cell programmable gene insertion |
| WO2024042199A1 (en) | 2022-08-26 | 2024-02-29 | KWS SAAT SE & Co. KGaA | Use of paired genes in hybrid breeding |
| CN116218905B (zh) * | 2022-12-06 | 2024-01-26 | 复旦大学附属华山医院 | 一种点突变大鼠癫痫模型的构建与应用 |
| WO2024138194A1 (en) | 2022-12-22 | 2024-06-27 | Tome Biosciences, Inc. | Platforms, compositions, and methods for in vivo programmable gene insertion |
| AU2024220323A1 (en) | 2023-02-15 | 2025-08-14 | Arbor Biotechnologies, Inc. | Gene editing method for inhibiting aberrant splicing in stathmin 2 (stmn2) transcript |
| WO2024192141A1 (en) | 2023-03-13 | 2024-09-19 | Dana-Farber Cancer Institute, Inc. | Treatment of cancers having a drug-resistant mesenchymal cell state |
| WO2024197185A1 (en) | 2023-03-21 | 2024-09-26 | The Broad Institute, Inc. | Methods and compositions for dissecting organelle physiology |
| TW202449159A (zh) | 2023-04-19 | 2024-12-16 | 美商旗艦先鋒創新有限責任(Vii)公司 | 用於產生文庫之組合物及方法 |
| WO2024234006A1 (en) | 2023-05-11 | 2024-11-14 | Tome Biosciences, Inc. | Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs) |
| WO2024236547A1 (en) | 2023-05-18 | 2024-11-21 | Inceptor Bio, Llc | Modified phagocytic cells expressing chimeric antigen receptors comprising a herpes virus entry mediator (hvem) co-stimulatory domain and uses thereof |
| TW202509220A (zh) | 2023-06-26 | 2025-03-01 | 美商旗艦先鋒創新有限責任(Vii)公司 | 經工程化之瘧原蟲及相關方法 |
| WO2025046513A1 (en) | 2023-08-29 | 2025-03-06 | Inceptor Bio, Llc | Methods of manufacturing myeloid-derived cells from hematopoietic stem cells and compositions and uses thereof |
| WO2025049959A2 (en) | 2023-09-01 | 2025-03-06 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| WO2025050069A1 (en) | 2023-09-01 | 2025-03-06 | Tome Biosciences, Inc. | Programmable gene insertion using engineered integration enzymes |
| WO2025059533A1 (en) | 2023-09-13 | 2025-03-20 | The Broad Institute, Inc. | Crispr enzymes and systems |
| KR20250113347A (ko) | 2024-01-17 | 2025-07-25 | 중앙대학교 산학협력단 | CRISPR-Cas nickase 시스템 기반 정교한 유전체 편집 방법 |
| WO2025171210A1 (en) | 2024-02-09 | 2025-08-14 | Arbor Biotechnologies, Inc. | Compositions and methods for gene editing via homology-mediated end joining |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025224107A1 (en) | 2024-04-22 | 2025-10-30 | Basecamp Research Ltd | Method and compositions for detecting off-target editing |
| WO2025224182A2 (en) | 2024-04-23 | 2025-10-30 | Basecamp Research Ltd | Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo |
| WO2025250808A1 (en) | 2024-05-29 | 2025-12-04 | The Brigham And Women’S Hospital, Inc. | Anti-crispr delivery compositions and methods |
Family Cites Families (171)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4217344A (en) | 1976-06-23 | 1980-08-12 | L'oreal | Compositions containing aqueous dispersions of lipid spheres |
| US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
| US4186183A (en) | 1978-03-29 | 1980-01-29 | The United States Of America As Represented By The Secretary Of The Army | Liposome carriers in chemotherapy of leishmaniasis |
| US4261975A (en) | 1979-09-19 | 1981-04-14 | Merck & Co., Inc. | Viral liposome particle |
| US4485054A (en) | 1982-10-04 | 1984-11-27 | Lipoderm Pharmaceuticals Limited | Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV) |
| US4501728A (en) | 1983-01-06 | 1985-02-26 | Technology Unlimited, Inc. | Masking of liposomes from RES recognition |
| US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4946787A (en) | 1985-01-07 | 1990-08-07 | Syntex (U.S.A.) Inc. | N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US5049386A (en) | 1985-01-07 | 1991-09-17 | Syntex (U.S.A.) Inc. | N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US4774085A (en) | 1985-07-09 | 1988-09-27 | 501 Board of Regents, Univ. of Texas | Pharmaceutical administration systems containing a mixture of immunomodulators |
| JP2874751B2 (ja) | 1986-04-09 | 1999-03-24 | ジェンザイム・コーポレーション | 希望する蛋白質をミルク中へ分泌する遺伝子移植動物 |
| US4837028A (en) | 1986-12-24 | 1989-06-06 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
| US4873316A (en) | 1987-06-23 | 1989-10-10 | Biogen, Inc. | Isolation of exogenous recombinant proteins from the milk of transgenic mammals |
| US5143854A (en) | 1989-06-07 | 1992-09-01 | Affymax Technologies N.V. | Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof |
| US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
| WO1991017424A1 (en) | 1990-05-03 | 1991-11-14 | Vical, Inc. | Intracellular delivery of biologically active substances by means of self-assembling lipid complexes |
| US5210015A (en) | 1990-08-06 | 1993-05-11 | Hoffman-La Roche Inc. | Homogeneous assay system using the nuclease activity of a nucleic acid polymerase |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US7150982B2 (en) | 1991-09-09 | 2006-12-19 | Third Wave Technologies, Inc. | RNA detection assays |
| US5587308A (en) | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| US7745416B2 (en) | 1995-04-11 | 2010-06-29 | The Regents Of The University Of California | Method for in vivo regulation of cardiac muscle contractility |
| CA2223103A1 (en) | 1995-06-06 | 1996-12-12 | Isis Pharmaceuticals Inc. | Oligonucleotides having phosphorothioate linkages of high chiral purity |
| US5985662A (en) | 1995-07-13 | 1999-11-16 | Isis Pharmaceuticals Inc. | Antisense inhibition of hepatitis B virus replication |
| US5622856A (en) | 1995-08-03 | 1997-04-22 | Avigen | High efficiency helper system for AAV vector production |
| US5846946A (en) | 1996-06-14 | 1998-12-08 | Pasteur Merieux Serums Et Vaccins | Compositions and methods for administering Borrelia DNA |
| US6251677B1 (en) | 1997-08-25 | 2001-06-26 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| IL135776A0 (en) | 1997-10-24 | 2001-05-20 | Life Technologies Inc | Recombinational cloning using nucleic acids having recombination sites |
| US6750059B1 (en) | 1998-07-16 | 2004-06-15 | Whatman, Inc. | Archiving of vectors |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US7868149B2 (en) | 1999-07-20 | 2011-01-11 | Monsanto Technology Llc | Plant genome sequence and uses thereof |
| US6603061B1 (en) | 1999-07-29 | 2003-08-05 | Monsanto Company | Agrobacterium-mediated plant transformation method |
| JP3454818B1 (ja) | 2001-03-16 | 2003-10-06 | 直哉 小林 | 肝臓細胞の増殖方法、該方法により得られる肝臓細胞、およびその用途 |
| ES2404689T3 (es) | 2001-04-05 | 2013-05-28 | The Johns Hopkins University | Vacunas quiméricas que comprenden el dominio lumenal de LAMP-1 o LAMP-2 |
| ES2566561T3 (es) | 2001-07-12 | 2016-04-13 | University Of Massachusetts | Producción in vivo de ARN pequeños de interferencia que median el silenciamiento génico |
| AU2002327430A1 (en) | 2001-08-08 | 2003-02-24 | Genzyme Corporation | Methods for treating diabetes and other blood sugar disorders |
| WO2003016338A1 (en) | 2001-08-15 | 2003-02-27 | Parker Hughes Institute | Crystal structure of the btk kinase domain |
| AU2002336760A1 (en) | 2001-09-26 | 2003-06-10 | Mayo Foundation For Medical Education And Research | Mutable vaccines |
| US20090100536A1 (en) | 2001-12-04 | 2009-04-16 | Monsanto Company | Transgenic plants with enhanced agronomic traits |
| WO2003087993A2 (en) | 2002-04-09 | 2003-10-23 | Beattie Kenneth L | Oligonucleotide probes for genosensor chips |
| WO2003104414A2 (en) | 2002-06-11 | 2003-12-18 | The Scripps Research Institute | Artificial transcription factors |
| US20080226553A1 (en) | 2002-09-27 | 2008-09-18 | Cold Spring Harbor Laboratory | Cell-Based Rna Interference and Related Methods and Compositions |
| US9534224B2 (en) | 2002-11-15 | 2017-01-03 | Trustees Of Boston University | Cis/trans riboregulators |
| US20060178297A1 (en) | 2003-01-28 | 2006-08-10 | Troy Carol M | Systems and methods for silencing expression of a gene in a cell and uses thereof |
| AU2004257191B2 (en) | 2003-07-03 | 2009-05-07 | The Regents Of The University Of California | Genome mapping of functional DNA elements and cellular proteins |
| EP3222715A1 (en) | 2003-08-08 | 2017-09-27 | Sangamo BioSciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| FR2862659B1 (fr) | 2003-11-21 | 2006-02-10 | Pasteur Institut | Genome de legionella pneumophila souche paris- applications diagnostiques et epidemiologiques |
| WO2005070948A1 (en) | 2004-01-23 | 2005-08-04 | Intronn, Inc. | Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated rna trans splicing |
| WO2005074511A2 (en) | 2004-01-27 | 2005-08-18 | The Board Of Trustees Of The Leland Stanford Junior University | Methods and compositions for homozygous gene inactivation using collections of pre-defined nucleotide sequences complementary to chromosomal transcripts |
| US20050220796A1 (en) | 2004-03-31 | 2005-10-06 | Dynan William S | Compositions and methods for modulating DNA repair |
| FR2872170B1 (fr) | 2004-06-25 | 2006-11-10 | Centre Nat Rech Scient Cnrse | Lentivirus non interactif et non replicatif, preparation et utilisations |
| AU2005274948B2 (en) | 2004-07-16 | 2011-09-22 | Genvec, Inc. | Vaccines against aids comprising CMV/R-nucleic acid constructs |
| WO2006116756A1 (en) | 2005-04-28 | 2006-11-02 | Benitec, Limited. | Multiple-rnai expression cassettes for simultaneous delivery of rnai agents related to heterozygotic expression patterns |
| US7892224B2 (en) | 2005-06-01 | 2011-02-22 | Brainlab Ag | Inverse catheter planning |
| WO2007014275A2 (en) | 2005-07-26 | 2007-02-01 | Sangamo Biosciences, Inc. | Targeted integration and expression of exogenous nucleic acid sequences |
| EP2325332B1 (en) | 2005-08-26 | 2012-10-31 | DuPont Nutrition Biosciences ApS | Use of CRISPR associated genes (CAS) |
| US8362229B2 (en) | 2006-02-08 | 2013-01-29 | Quark Pharmaceuticals, Inc. | Tandem siRNAS |
| EP1994182B1 (en) | 2006-03-15 | 2019-05-29 | Siemens Healthcare Diagnostics Inc. | Degenerate nucleobase analogs |
| SG171676A1 (en) | 2006-05-11 | 2011-06-29 | Alnylam Pharmaceuticals Inc | Compositions and methods for inhibiting expression of the pcsk9 gene |
| US8748567B2 (en) | 2006-05-22 | 2014-06-10 | Children's Medical Center Corporation | Method for delivery across the blood brain barrier |
| WO2008093152A1 (en) | 2007-02-01 | 2008-08-07 | Cellectis | Obligate heterodimer meganucleases and uses thereof |
| NZ579002A (en) | 2007-03-02 | 2012-03-30 | Danisco | Cultures with improved phage resistance |
| PE20090064A1 (es) | 2007-03-26 | 2009-03-02 | Novartis Ag | Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende |
| CA2711179A1 (en) | 2007-12-31 | 2009-07-16 | Nanocor Therapeutics, Inc. | Rna interference for the treatment of heart failure |
| US20100081707A1 (en) | 2008-02-21 | 2010-04-01 | Ali Robin R | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| WO2010011961A2 (en) | 2008-07-25 | 2010-01-28 | University Of Georgia Research Foundation, Inc. | Prokaryotic rnai-like system and methods of use |
| US20100076057A1 (en) | 2008-09-23 | 2010-03-25 | Northwestern University | TARGET DNA INTERFERENCE WITH crRNA |
| WO2010054108A2 (en) | 2008-11-06 | 2010-05-14 | University Of Georgia Research Foundation, Inc. | Cas6 polypeptides and methods of use |
| MX353900B (es) | 2008-11-07 | 2018-02-01 | Massachusetts Inst Technology | Lipidoides de aminoalcohol y usos de los mismos. |
| CN102625655B (zh) | 2008-12-04 | 2016-07-06 | 桑格摩生物科学股份有限公司 | 使用锌指核酸酶在大鼠中进行基因组编辑 |
| US20110016540A1 (en) | 2008-12-04 | 2011-01-20 | Sigma-Aldrich Co. | Genome editing of genes associated with trinucleotide repeat expansion disorders in animals |
| CA2746514C (en) | 2008-12-10 | 2018-11-27 | Alnylam Pharmaceuticals, Inc. | Gnaq targeted dsrna compositions and methods for inhibiting expression |
| WO2010075424A2 (en) | 2008-12-22 | 2010-07-01 | The Regents Of University Of California | Compositions and methods for downregulating prokaryotic genes |
| US20110239315A1 (en) | 2009-01-12 | 2011-09-29 | Ulla Bonas | Modular dna-binding domains and methods of use |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| TWI576432B (zh) * | 2009-04-07 | 2017-04-01 | 陶氏農業科學公司 | 序列特異性核酸酶之奈米粒子媒介式遞送技術 |
| WO2011036510A1 (en) | 2009-09-24 | 2011-03-31 | Cellectis | Meganuclease variants cleaving the genome of the herpes simplex virus and uses thereof |
| US20120149115A1 (en) * | 2009-06-11 | 2012-06-14 | Snu R&Db Foundation | Targeted genomic rearrangements using site-specific nucleases |
| JP2013500018A (ja) | 2009-07-24 | 2013-01-07 | シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニー | ゲノム編集のための方法 |
| CA2769262C (en) | 2009-07-28 | 2019-04-30 | Sangamo Biosciences, Inc. | Methods and compositions for treating trinucleotide repeat disorders |
| WO2011028929A2 (en) | 2009-09-03 | 2011-03-10 | The Regents Of The University Of California | Nitrate-responsive promoter |
| US8889394B2 (en) | 2009-09-07 | 2014-11-18 | Empire Technology Development Llc | Multiple domain proteins |
| AU2010325549B2 (en) | 2009-11-27 | 2017-04-20 | Basf Plant Science Company Gmbh | Optimized endonucleases and uses thereof |
| CA2783351C (en) * | 2009-12-10 | 2021-09-07 | Regents Of The University Of Minnesota | Tal effector-mediated dna modification |
| SG181823A1 (en) | 2009-12-23 | 2012-07-30 | Max Planck Gesellschaft | Influenza targets |
| WO2011100058A1 (en) | 2010-02-09 | 2011-08-18 | Sangamo Biosciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| US10087431B2 (en) | 2010-03-10 | 2018-10-02 | The Regents Of The University Of California | Methods of generating nucleic acid fragments |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| EA024121B9 (ru) | 2010-05-10 | 2017-01-30 | Дзе Реджентс Ов Дзе Юниверсити Ов Калифорния | Композиции эндорибонуклеаз и способы их использования |
| CA2798988C (en) | 2010-05-17 | 2020-03-10 | Sangamo Biosciences, Inc. | Tal-effector (tale) dna-binding polypeptides and uses thereof |
| EP3489359A1 (en) * | 2010-07-23 | 2019-05-29 | Sigma Aldrich Co. LLC | Genome editing using targeting endonucleases and single-stranded nucleic acids |
| EP2601611B1 (en) | 2010-08-02 | 2020-12-09 | Integrated Dna Technologies, Inc. | Methods for predicting stability and melting temperatures of nucleic acid duplexes |
| WO2012031205A2 (en) | 2010-09-03 | 2012-03-08 | The Brigham And Women's Hospital, Inc. | Lipid-polymer hybrid particles |
| WO2012051343A1 (en) | 2010-10-12 | 2012-04-19 | The Children's Hospital Of Philadelphia | Methods and compositions for treating hemophilia b |
| US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
| RS66184B1 (sr) | 2011-04-22 | 2024-12-31 | Univ California | Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu |
| PT2702160T (pt) | 2011-04-27 | 2020-07-30 | Amyris Inc | Métodos para modificação genómica |
| WO2012164565A1 (en) | 2011-06-01 | 2012-12-06 | Yeda Research And Development Co. Ltd. | Compositions and methods for downregulating prokaryotic genes |
| CA3186126A1 (en) | 2011-09-21 | 2013-03-28 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
| CA2849920A1 (en) * | 2011-10-06 | 2013-04-11 | Sangamo Biosciences, Inc. | Methods and compositions for regulating hiv infection |
| US20130122096A1 (en) | 2011-11-14 | 2013-05-16 | Silenseed Ltd. | Compositions for drug delivery and methods of manufacturing and using same |
| EP2780376B1 (en) | 2011-11-18 | 2017-03-08 | Université Laval | Methods and products for increasing frataxin levels and uses thereof |
| JP6348064B2 (ja) | 2011-11-22 | 2018-06-27 | ザ チルドレンズ ホスピタル オブ フィラデルフィア | 効率の高いトランスジーン送達のためのウイルスベクター |
| US8450107B1 (en) | 2011-11-30 | 2013-05-28 | The Broad Institute Inc. | Nucleotide-specific recognition sequences for designer TAL effectors |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| BR112014021104B1 (pt) | 2012-02-29 | 2023-03-28 | Sangamo Biosciences, Inc | Proteína de fusão de ocorrência não natural compreendendo um domínio de ligação de dna de dedo de zinco manipulado que se liga a um gene htt, seu uso, método in vitro de modificação da expressão de um gene htt em uma célula, e método de geração de um sistema modelo para o estudo da doença de huntington |
| US9637739B2 (en) | 2012-03-20 | 2017-05-02 | Vilnius University | RNA-directed DNA cleavage by the Cas9-crRNA complex |
| WO2013141680A1 (en) | 2012-03-20 | 2013-09-26 | Vilnius University | RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX |
| AU2013204327B2 (en) | 2012-04-20 | 2016-09-01 | Aviagen | Cell transfection method |
| FI3597749T3 (fi) | 2012-05-25 | 2023-10-09 | Univ California | Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten |
| SG11201407970VA (en) | 2012-06-01 | 2014-12-30 | Univ Drexel | Modulation of hepatitis b virus cccdna transcription |
| ES2824024T3 (es) | 2012-10-10 | 2021-05-11 | Sangamo Therapeutics Inc | Compuestos modificadores de células T y usos de los mismos |
| KR101656236B1 (ko) | 2012-10-23 | 2016-09-12 | 주식회사 툴젠 | 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도 |
| PT3363902T (pt) * | 2012-12-06 | 2019-12-19 | Sigma Aldrich Co Llc | Modificação e regulação de genoma baseadas em crispr |
| WO2014093479A1 (en) | 2012-12-11 | 2014-06-19 | Montana State University | Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation |
| PT2784162E (pt) | 2012-12-12 | 2015-08-27 | Broad Inst Inc | Engenharia de sistemas, métodos e composições guia otimizadas para a manipulação de sequências |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| 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 |
| EP3434776A1 (en) | 2012-12-12 | 2019-01-30 | 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 |
| KR20150105956A (ko) | 2012-12-12 | 2015-09-18 | 더 브로드 인스티튜트, 인코퍼레이티드 | 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화 |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| EP2931898B1 (en) | 2012-12-12 | 2016-03-09 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| ES2576126T3 (es) | 2012-12-12 | 2016-07-05 | The Broad Institute, Inc. | Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias |
| EP2932421A1 (en) | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| EP4234696A3 (en) | 2012-12-12 | 2023-09-06 | The Broad Institute Inc. | Crispr-cas component systems, methods and compositions for sequence manipulation |
| EP4282970A3 (en) | 2012-12-17 | 2024-01-17 | President and Fellows of Harvard College | Rna-guided human genome engineering |
| EP3919505B1 (en) | 2013-01-16 | 2023-08-30 | Emory University | Uses of cas9-nucleic acid complexes |
| WO2014124226A1 (en) | 2013-02-07 | 2014-08-14 | The Rockefeller University | Sequence specific antimicrobials |
| US11135273B2 (en) | 2013-02-07 | 2021-10-05 | The Rockefeller University | Sequence specific antimicrobials |
| US9163837B2 (en) | 2013-02-27 | 2015-10-20 | Siemens Aktiengesellschaft | Flow conditioner in a combustor of a gas turbine engine |
| AU2014235794A1 (en) | 2013-03-14 | 2015-10-22 | Caribou Biosciences, Inc. | Compositions and methods of nucleic acid-targeting nucleic acids |
| KR102874079B1 (ko) | 2013-03-15 | 2025-10-22 | 더 제너럴 하스피탈 코포레이션 | Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용 |
| HUE034635T2 (en) | 2013-03-27 | 2018-02-28 | Wilco Ag | A method for checking and / or testing devices in a row and equipment for performing such a procedure |
| US11274305B2 (en) | 2013-04-04 | 2022-03-15 | Trustees Of Dartmouth College | Compositions and methods for in vivo excision of HIV-1 proviral DNA |
| AU2014265331B2 (en) | 2013-05-15 | 2019-12-05 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| EP3004349B1 (en) | 2013-05-29 | 2018-03-28 | Cellectis S.A. | A method for producing precise dna cleavage using cas9 nickase activity |
| RU2690935C2 (ru) * | 2013-06-04 | 2019-06-06 | Президент Энд Фэллоуз Оф Харвард Коллидж | Направляемая рнк регуляция транскрипции |
| US20140356956A1 (en) | 2013-06-04 | 2014-12-04 | President And Fellows Of Harvard College | RNA-Guided Transcriptional Regulation |
| JP7085716B2 (ja) | 2013-06-05 | 2022-06-17 | デューク ユニバーシティ | Rnaガイド遺伝子編集及び遺伝子調節 |
| CN105492611A (zh) * | 2013-06-17 | 2016-04-13 | 布罗德研究所有限公司 | 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物 |
| EP3725885A1 (en) | 2013-06-17 | 2020-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
| CA2915795C (en) | 2013-06-17 | 2021-07-13 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components |
| EP3674411A1 (en) | 2013-06-17 | 2020-07-01 | The Broad Institute, Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| CN107995927B (zh) | 2013-06-17 | 2021-07-30 | 布罗德研究所有限公司 | 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途 |
| JP6738729B2 (ja) | 2013-06-17 | 2020-08-12 | ザ・ブロード・インスティテュート・インコーポレイテッド | 分裂終了細胞の疾患および障害をターゲティングおよびモデリングするための系、方法および組成物の送達、エンジニアリングおよび最適化 |
| CN103343120B (zh) | 2013-07-04 | 2015-03-04 | 中国科学院遗传与发育生物学研究所 | 一种小麦基因组定点改造方法 |
| DK3019619T3 (da) | 2013-07-11 | 2021-10-11 | Modernatx Inc | Sammensætninger, der omfatter syntetiske polynukleotider, som koder for crispr-beslægtede proteiner, og syntetiske sgrna'er, og anvendelsesfremgangsmåder |
| 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 |
| AU2014312123A1 (en) | 2013-08-29 | 2016-03-17 | Temple University Of The Commonwealth System Of Higher Education | Methods and compositions for RNA-guided treatment of HIV infection |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US10822606B2 (en) | 2013-09-27 | 2020-11-03 | 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 |
| WO2015065964A1 (en) | 2013-10-28 | 2015-05-07 | The Broad Institute Inc. | Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof |
| KR20230054509A (ko) | 2013-11-07 | 2023-04-24 | 에디타스 메디신, 인코포레이티드 | 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물 |
| US20160298096A1 (en) | 2013-11-18 | 2016-10-13 | Crispr Therapeutics Ag | Crispr-cas system materials and methods |
| SG10201804973TA (en) | 2013-12-12 | 2018-07-30 | Broad Inst Inc | Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders |
| WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
| EP3079726B1 (en) | 2013-12-12 | 2018-12-05 | 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 |
| JP2017503485A (ja) | 2013-12-12 | 2017-02-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 遺伝子産物の発現、構造情報、及び誘導性モジュラーcas酵素を変更するためのcrispr−cas系並びに方法 |
| 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 |
| US10354746B2 (en) | 2014-01-27 | 2019-07-16 | Georgia Tech Research Corporation | Methods and systems for identifying CRISPR/Cas off-target sites |
| JP6323228B2 (ja) | 2014-07-18 | 2018-05-16 | 富士電機株式会社 | 電力変換装置 |
| US9932566B2 (en) | 2014-08-07 | 2018-04-03 | Agilent Technologies, Inc. | CIS-blocked guide RNA |
| EP3180426B1 (en) | 2014-08-17 | 2019-12-25 | The Broad Institute, Inc. | Genome editing using cas9 nickases |
| KR102598856B1 (ko) | 2015-03-03 | 2023-11-07 | 더 제너럴 하스피탈 코포레이션 | 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제 |
| DE112016002056T5 (de) | 2015-05-06 | 2018-02-08 | Snipr Technologies Limited | Verändern mikrobieller Populationen und Modifizieren von Mikrobiomen |
| 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 |
| US9512446B1 (en) | 2015-08-28 | 2016-12-06 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| JP6186470B2 (ja) | 2016-04-20 | 2017-08-23 | パイオニア株式会社 | 音響装置、音量制御方法、音量制御プログラム及び記録媒体 |
| CN107939288B (zh) | 2017-11-14 | 2019-04-02 | 中国科学院地质与地球物理研究所 | 一种非旋转套的防转装置以及旋转导向装置 |
-
2014
- 2014-06-10 CN CN201480045703.4A patent/CN105492611A/zh active Pending
- 2014-06-10 CA CA2915837A patent/CA2915837A1/en not_active Abandoned
- 2014-06-10 JP JP2016521450A patent/JP6665088B2/ja active Active
- 2014-06-10 WO PCT/US2014/041803 patent/WO2014204725A1/en not_active Ceased
- 2014-06-10 EP EP14734709.0A patent/EP3011030B1/en active Active
- 2014-06-10 AU AU2014281027A patent/AU2014281027A1/en not_active Abandoned
- 2014-06-10 EP EP23166208.1A patent/EP4245853A3/en active Pending
- 2014-06-10 KR KR1020167001293A patent/KR20160034901A/ko not_active Ceased
-
2015
- 2015-12-17 US US14/972,523 patent/US10711285B2/en active Active
-
2020
- 2020-02-19 JP JP2020025947A patent/JP7083364B2/ja active Active
- 2020-04-09 US US16/844,548 patent/US11597949B2/en active Active
-
2022
- 2022-03-02 JP JP2022031416A patent/JP7383062B2/ja active Active
-
2023
- 2023-02-06 US US18/106,039 patent/US20230374550A1/en active Pending
- 2023-11-07 JP JP2023189768A patent/JP7682975B2/ja active Active
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019088496A3 (ko) * | 2017-10-31 | 2019-06-27 | 주식회사 에이치유비바이오텍 | 내인성 폴리펩타이드 생산을 위한 재조합 세포 및 방법 |
| WO2022169235A1 (ko) * | 2021-02-04 | 2022-08-11 | 연세대학교 산학협력단 | 교정 효율이 개선된 프라임에디팅용 조성물 |
Also Published As
| Publication number | Publication date |
|---|---|
| US10711285B2 (en) | 2020-07-14 |
| EP4245853A2 (en) | 2023-09-20 |
| EP3011030A1 (en) | 2016-04-27 |
| JP2020110151A (ja) | 2020-07-27 |
| US11597949B2 (en) | 2023-03-07 |
| JP2022071089A (ja) | 2022-05-13 |
| JP7083364B2 (ja) | 2022-06-10 |
| EP4245853A3 (en) | 2023-10-18 |
| US20160153006A1 (en) | 2016-06-02 |
| JP6665088B2 (ja) | 2020-03-13 |
| CN105492611A (zh) | 2016-04-13 |
| JP2024023239A (ja) | 2024-02-21 |
| JP7682975B2 (ja) | 2025-05-26 |
| CA2915837A1 (en) | 2014-12-24 |
| HK1223645A1 (en) | 2017-08-04 |
| EP3011030B1 (en) | 2023-11-08 |
| JP2016521994A (ja) | 2016-07-28 |
| WO2014204725A8 (en) | 2015-05-28 |
| AU2014281027A1 (en) | 2016-01-28 |
| US20200377912A1 (en) | 2020-12-03 |
| US20230374550A1 (en) | 2023-11-23 |
| WO2014204725A1 (en) | 2014-12-24 |
| JP7383062B2 (ja) | 2023-11-17 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7682975B2 (ja) | 配列操作のための最適化されたCRISPR-Cas二重ニッカーゼ系、方法および組成物 | |
| JP2024023194A (ja) | 肝臓ターゲティングおよび治療のためのCRISPR-Cas系、ベクターおよび組成物の送達および使用 | |
| RU2721275C2 (ru) | Доставка, конструирование и оптимизация систем, способов и композиций для манипуляции с последовательностями и применения в терапии | |
| JP6625971B2 (ja) | 配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化 | |
| RU2716421C2 (ru) | Доставка, применение и применения в терапии систем crispr-cas и композиций для целенаправленного воздействия на нарушения и заболевания с использованием вирусных компонентов | |
| JP6896786B2 (ja) | 配列操作のためのCRISPR−Cas成分系、方法および組成物 | |
| RU2725502C2 (ru) | Доставка, конструирование и оптимизация систем, способы и композиции для целенаправленного воздействия и моделирования заболеваний и нарушений постмитотических клеток | |
| JP2024163122A (ja) | 配列操作のための改善された系、方法および酵素組成物のエンジニアリングおよび最適化 | |
| KR102613296B1 (ko) | 신규한 crispr 효소 및 시스템 | |
| JP2024170440A (ja) | 配列操作のための系、方法および最適化ガイド組成物のエンジニアリング | |
| DK2784162T3 (en) | Design of systems, methods and optimized control manipulations for sequence manipulation | |
| KR20150107739A (ko) | 유전자 산물의 발현의 변경을 위한 crispr―cas 시스템 및 방법 | |
| WO2014204723A9 (en) | Oncogenic models based on delivery and use of the crispr-cas systems, vectors and compositions | |
| JP2025181841A (ja) | 配列操作および治療適用のための系、方法および組成物の送達、エンジニアリングおよび最適化 | |
| KR102539173B1 (ko) | 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PA0105 | International application |
Patent event date: 20160115 Patent event code: PA01051R01D Comment text: International Patent Application |
|
| PG1501 | Laying open of application | ||
| A201 | Request for examination | ||
| PA0201 | Request for examination |
Patent event code: PA02012R01D Patent event date: 20190523 Comment text: Request for Examination of Application |
|
| PE0902 | Notice of grounds for rejection |
Comment text: Notification of reason for refusal Patent event date: 20210730 Patent event code: PE09021S01D |
|
| E601 | Decision to refuse application | ||
| PE0601 | Decision on rejection of patent |
Patent event date: 20211112 Comment text: Decision to Refuse Application Patent event code: PE06012S01D Patent event date: 20210730 Comment text: Notification of reason for refusal Patent event code: PE06011S01I |