BR122021008308B1 - Sistemas de componente crispr-cas, métodos e composições para manipulação de sequência - Google Patents
Sistemas de componente crispr-cas, métodos e composições para manipulação de sequência Download PDFInfo
- Publication number
- BR122021008308B1 BR122021008308B1 BR122021008308-8A BR122021008308A BR122021008308B1 BR 122021008308 B1 BR122021008308 B1 BR 122021008308B1 BR 122021008308 A BR122021008308 A BR 122021008308A BR 122021008308 B1 BR122021008308 B1 BR 122021008308B1
- Authority
- BR
- Brazil
- Prior art keywords
- sequence
- cas9
- crispr
- target
- chimeric rna
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract description 125
- 239000000203 mixture Substances 0.000 title abstract description 11
- 108091033409 CRISPR Proteins 0.000 claims abstract description 320
- 239000013598 vector Substances 0.000 claims abstract description 161
- 230000035772 mutation Effects 0.000 claims abstract description 147
- 210000003527 eukaryotic cell Anatomy 0.000 claims abstract description 118
- 230000000694 effects Effects 0.000 claims abstract description 108
- 238000010354 CRISPR gene editing Methods 0.000 claims abstract 6
- 108020004414 DNA Proteins 0.000 claims description 131
- 102000040430 polynucleotide Human genes 0.000 claims description 117
- 108091033319 polynucleotide Proteins 0.000 claims description 117
- 239000002157 polynucleotide Substances 0.000 claims description 117
- 230000014509 gene expression Effects 0.000 claims description 107
- 230000008685 targeting Effects 0.000 claims description 107
- 125000003729 nucleotide group Chemical group 0.000 claims description 89
- 239000002773 nucleotide Substances 0.000 claims description 86
- 108091092236 Chimeric RNA Proteins 0.000 claims description 81
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 68
- 239000000700 radioactive tracer Substances 0.000 claims description 64
- 150000007523 nucleic acids Chemical class 0.000 claims description 61
- 108010077850 Nuclear Localization Signals Proteins 0.000 claims description 54
- 201000010099 disease Diseases 0.000 claims description 49
- 102000039446 nucleic acids Human genes 0.000 claims description 48
- 108020004707 nucleic acids Proteins 0.000 claims description 48
- 241000193996 Streptococcus pyogenes Species 0.000 claims description 39
- 238000013518 transcription Methods 0.000 claims description 30
- 230000035897 transcription Effects 0.000 claims description 30
- 230000004048 modification Effects 0.000 claims description 28
- 238000012986 modification Methods 0.000 claims description 28
- 108020004999 messenger RNA Proteins 0.000 claims description 20
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims description 19
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims description 19
- 210000004962 mammalian cell Anatomy 0.000 claims description 19
- 230000027455 binding Effects 0.000 claims description 18
- 241000194020 Streptococcus thermophilus Species 0.000 claims description 16
- 239000013603 viral vector Substances 0.000 claims description 15
- 230000003612 virological effect Effects 0.000 claims description 15
- 230000007018 DNA scission Effects 0.000 claims description 14
- 102000053602 DNA Human genes 0.000 claims description 13
- 238000004519 manufacturing process Methods 0.000 claims description 12
- 108020001580 protein domains Proteins 0.000 claims description 10
- 230000004913 activation Effects 0.000 claims description 8
- 239000003814 drug Substances 0.000 claims description 8
- 230000001177 retroviral effect Effects 0.000 claims description 8
- 208000026350 Inborn Genetic disease Diseases 0.000 claims description 6
- 230000003197 catalytic effect Effects 0.000 claims description 6
- 229940079593 drug Drugs 0.000 claims description 6
- 208000016361 genetic disease Diseases 0.000 claims description 6
- 208000009869 Neu-Laxova syndrome Diseases 0.000 claims description 5
- 241000700584 Simplexvirus Species 0.000 claims description 5
- 239000002502 liposome Substances 0.000 claims description 5
- 108020001778 catalytic domains Proteins 0.000 claims description 4
- 210000005260 human cell Anatomy 0.000 claims description 4
- 239000002245 particle Substances 0.000 claims description 4
- 108010033040 Histones Proteins 0.000 claims description 3
- 101000615488 Homo sapiens Methyl-CpG-binding domain protein 2 Proteins 0.000 claims description 3
- 102100021299 Methyl-CpG-binding domain protein 2 Human genes 0.000 claims description 3
- 230000007022 RNA scission Effects 0.000 claims description 3
- 239000003488 releasing hormone Substances 0.000 claims description 3
- 210000002845 virion Anatomy 0.000 claims description 3
- 239000000277 virosome Substances 0.000 claims description 3
- 102220605874 Cytosolic arginine sensor for mTORC1 subunit 2_D10A_mutation Human genes 0.000 claims 4
- 101150029664 PELO gene Proteins 0.000 claims 1
- 210000004027 cell Anatomy 0.000 abstract description 289
- 230000009918 complex formation Effects 0.000 abstract description 5
- 238000010453 CRISPR/Cas method Methods 0.000 abstract description 3
- 108090000623 proteins and genes Proteins 0.000 description 203
- 102000004190 Enzymes Human genes 0.000 description 183
- 108090000790 Enzymes Proteins 0.000 description 183
- 229940088598 enzyme Drugs 0.000 description 183
- 238000003776 cleavage reaction Methods 0.000 description 123
- 230000007017 scission Effects 0.000 description 122
- 239000013612 plasmid Substances 0.000 description 92
- 230000001105 regulatory effect Effects 0.000 description 73
- 239000000047 product Substances 0.000 description 69
- 102000004169 proteins and genes Human genes 0.000 description 64
- 125000006850 spacer group Chemical group 0.000 description 64
- 235000018102 proteins Nutrition 0.000 description 59
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 55
- 238000002744 homologous recombination Methods 0.000 description 53
- 230000006801 homologous recombination Effects 0.000 description 53
- 238000003556 assay Methods 0.000 description 52
- 241000282414 Homo sapiens Species 0.000 description 46
- 108020005004 Guide RNA Proteins 0.000 description 44
- 238000012217 deletion Methods 0.000 description 40
- 230000037430 deletion Effects 0.000 description 40
- 238000010362 genome editing Methods 0.000 description 39
- 230000037431 insertion Effects 0.000 description 39
- 238000003780 insertion Methods 0.000 description 39
- 238000005516 engineering process Methods 0.000 description 38
- 102100023823 Homeobox protein EMX1 Human genes 0.000 description 36
- 101001048956 Homo sapiens Homeobox protein EMX1 Proteins 0.000 description 36
- 108091034117 Oligonucleotide Proteins 0.000 description 36
- 229930027917 kanamycin Natural products 0.000 description 36
- 229960000318 kanamycin Drugs 0.000 description 36
- SBUJHOSQTJFQJX-NOAMYHISSA-N kanamycin Chemical compound O[C@@H]1[C@@H](O)[C@H](O)[C@@H](CN)O[C@@H]1O[C@H]1[C@H](O)[C@@H](O[C@@H]2[C@@H]([C@@H](N)[C@H](O)[C@@H](CO)O2)O)[C@H](N)C[C@@H]1N SBUJHOSQTJFQJX-NOAMYHISSA-N 0.000 description 36
- 229930182823 kanamycin A Natural products 0.000 description 36
- 230000001404 mediated effect Effects 0.000 description 36
- 101710163270 Nuclease Proteins 0.000 description 34
- 241000588724 Escherichia coli Species 0.000 description 33
- 230000009466 transformation Effects 0.000 description 33
- UCSJYZPVAKXKNQ-HZYVHMACSA-N streptomycin Chemical compound CN[C@H]1[C@H](O)[C@@H](O)[C@H](CO)O[C@H]1O[C@@H]1[C@](C=O)(O)[C@H](C)O[C@H]1O[C@@H]1[C@@H](NC(N)=N)[C@H](O)[C@@H](NC(N)=N)[C@H](O)[C@H]1O UCSJYZPVAKXKNQ-HZYVHMACSA-N 0.000 description 32
- 239000000499 gel Substances 0.000 description 31
- 150000001413 amino acids Chemical class 0.000 description 30
- ULGZDMOVFRHVEP-RWJQBGPGSA-N Erythromycin Chemical compound O([C@@H]1[C@@H](C)C(=O)O[C@@H]([C@@]([C@H](O)[C@@H](C)C(=O)[C@H](C)C[C@@](C)(O)[C@H](O[C@H]2[C@@H]([C@H](C[C@@H](C)O2)N(C)C)O)[C@H]1C)(C)O)CC)[C@H]1C[C@@](C)(OC)[C@@H](O)[C@H](C)O1 ULGZDMOVFRHVEP-RWJQBGPGSA-N 0.000 description 28
- 239000013604 expression vector Substances 0.000 description 28
- 230000006798 recombination Effects 0.000 description 28
- 238000005215 recombination Methods 0.000 description 28
- 239000000523 sample Substances 0.000 description 28
- 241000320123 Streptococcus pyogenes M1 GAS Species 0.000 description 25
- 238000004458 analytical method Methods 0.000 description 25
- 238000011144 upstream manufacturing Methods 0.000 description 25
- 229940024606 amino acid Drugs 0.000 description 24
- 235000001014 amino acid Nutrition 0.000 description 24
- 230000030648 nucleus localization Effects 0.000 description 24
- 238000013461 design Methods 0.000 description 23
- 210000004940 nucleus Anatomy 0.000 description 23
- 108091028043 Nucleic acid sequence Proteins 0.000 description 22
- 238000012163 sequencing technique Methods 0.000 description 22
- 101150087718 srtA gene Proteins 0.000 description 22
- 230000000295 complement effect Effects 0.000 description 21
- 238000002474 experimental method Methods 0.000 description 21
- 238000001890 transfection Methods 0.000 description 21
- 108091093088 Amplicon Proteins 0.000 description 20
- 241000196324 Embryophyta Species 0.000 description 20
- 238000004422 calculation algorithm Methods 0.000 description 20
- 238000012360 testing method Methods 0.000 description 20
- 108091026890 Coding region Proteins 0.000 description 19
- WIIZWVCIJKGZOK-RKDXNWHRSA-N chloramphenicol Chemical compound ClC(Cl)C(=O)N[C@H](CO)[C@H](O)C1=CC=C([N+]([O-])=O)C=C1 WIIZWVCIJKGZOK-RKDXNWHRSA-N 0.000 description 19
- 208000035475 disorder Diseases 0.000 description 19
- 101150098466 rpsL gene Proteins 0.000 description 19
- 101100478373 Bacillus subtilis (strain 168) srtD gene Proteins 0.000 description 18
- 241000699666 Mus <mouse, genus> Species 0.000 description 18
- 229960005091 chloramphenicol Drugs 0.000 description 18
- 230000009870 specific binding Effects 0.000 description 18
- 101150087081 bgaA gene Proteins 0.000 description 17
- 108010025678 empty spiracles homeobox proteins Proteins 0.000 description 17
- 210000001519 tissue Anatomy 0.000 description 17
- 229920002401 polyacrylamide Polymers 0.000 description 16
- 230000008569 process Effects 0.000 description 16
- 229960005322 streptomycin Drugs 0.000 description 16
- 241001633172 Streptococcus thermophilus LMD-9 Species 0.000 description 15
- 230000002068 genetic effect Effects 0.000 description 15
- 238000000338 in vitro Methods 0.000 description 15
- 108020004705 Codon Proteins 0.000 description 14
- 108010005774 beta-Galactosidase Proteins 0.000 description 14
- 239000000872 buffer Substances 0.000 description 14
- 229960003276 erythromycin Drugs 0.000 description 14
- 230000002441 reversible effect Effects 0.000 description 14
- 102100026189 Beta-galactosidase Human genes 0.000 description 13
- 238000000636 Northern blotting Methods 0.000 description 13
- 230000001580 bacterial effect Effects 0.000 description 13
- 230000005782 double-strand break Effects 0.000 description 13
- 101000910046 Streptococcus thermophilus (strain ATCC BAA-491 / LMD-9) CRISPR-associated endonuclease Cas9 1 Proteins 0.000 description 12
- 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 12
- 238000009825 accumulation Methods 0.000 description 12
- 230000015572 biosynthetic process Effects 0.000 description 12
- 238000001514 detection method Methods 0.000 description 12
- 239000012634 fragment Substances 0.000 description 12
- 230000004927 fusion Effects 0.000 description 12
- 230000001965 increasing effect Effects 0.000 description 12
- 230000006780 non-homologous end joining Effects 0.000 description 12
- 241000894006 Bacteria Species 0.000 description 11
- 101150038500 cas9 gene Proteins 0.000 description 11
- 230000006870 function Effects 0.000 description 11
- 230000036961 partial effect Effects 0.000 description 11
- 108090000765 processed proteins & peptides Proteins 0.000 description 11
- 230000008439 repair process Effects 0.000 description 11
- 238000006467 substitution reaction Methods 0.000 description 11
- 101000706121 Homo sapiens Parvalbumin alpha Proteins 0.000 description 10
- 238000010367 cloning Methods 0.000 description 10
- 102000037865 fusion proteins Human genes 0.000 description 10
- 108020001507 fusion proteins Proteins 0.000 description 10
- 239000002609 medium Substances 0.000 description 10
- 238000005457 optimization Methods 0.000 description 10
- 230000037361 pathway Effects 0.000 description 10
- 102000004196 processed proteins & peptides Human genes 0.000 description 10
- 108010035563 Chloramphenicol O-acetyltransferase Proteins 0.000 description 9
- 238000007400 DNA extraction Methods 0.000 description 9
- 241000702421 Dependoparvovirus Species 0.000 description 9
- 241001465754 Metazoa Species 0.000 description 9
- 108091027544 Subgenomic mRNA Proteins 0.000 description 9
- 241000700605 Viruses Species 0.000 description 9
- 239000003623 enhancer Substances 0.000 description 9
- 238000009396 hybridization Methods 0.000 description 9
- 238000011002 quantification Methods 0.000 description 9
- 238000011160 research Methods 0.000 description 9
- 241000701161 unidentified adenovirus Species 0.000 description 9
- 108700028369 Alleles Proteins 0.000 description 8
- 101100118093 Drosophila melanogaster eEF1alpha2 gene Proteins 0.000 description 8
- 238000013459 approach Methods 0.000 description 8
- 230000008901 benefit Effects 0.000 description 8
- 230000002759 chromosomal effect Effects 0.000 description 8
- 238000009826 distribution Methods 0.000 description 8
- 238000001415 gene therapy Methods 0.000 description 8
- 230000007246 mechanism Effects 0.000 description 8
- 238000004806 packaging method and process Methods 0.000 description 8
- 210000001236 prokaryotic cell Anatomy 0.000 description 8
- 230000002103 transcriptional effect Effects 0.000 description 8
- 238000012546 transfer Methods 0.000 description 8
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 7
- -1 Cas2 Proteins 0.000 description 7
- 108091033380 Coding strand Proteins 0.000 description 7
- 239000006144 Dulbecco’s modified Eagle's medium Substances 0.000 description 7
- 102100030856 Myoglobin Human genes 0.000 description 7
- 238000010222 PCR analysis Methods 0.000 description 7
- 108010008281 Recombinant Fusion Proteins Proteins 0.000 description 7
- 102000007056 Recombinant Fusion Proteins Human genes 0.000 description 7
- 108091081062 Repeated sequence (DNA) Proteins 0.000 description 7
- 101150055766 cat gene Proteins 0.000 description 7
- 230000030833 cell death Effects 0.000 description 7
- 238000010276 construction Methods 0.000 description 7
- 230000003247 decreasing effect Effects 0.000 description 7
- 238000012350 deep sequencing Methods 0.000 description 7
- 230000001939 inductive effect Effects 0.000 description 7
- 239000012528 membrane Substances 0.000 description 7
- 238000003199 nucleic acid amplification method Methods 0.000 description 7
- 244000052769 pathogen Species 0.000 description 7
- 230000009467 reduction Effects 0.000 description 7
- 239000000243 solution Substances 0.000 description 7
- 208000024891 symptom Diseases 0.000 description 7
- VUFNLQXQSDUXKB-DOFZRALJSA-N 2-[4-[4-[bis(2-chloroethyl)amino]phenyl]butanoyloxy]ethyl (5z,8z,11z,14z)-icosa-5,8,11,14-tetraenoate Chemical compound CCCCC\C=C/C\C=C/C\C=C/C\C=C/CCCC(=O)OCCOC(=O)CCCC1=CC=C(N(CCCl)CCCl)C=C1 VUFNLQXQSDUXKB-DOFZRALJSA-N 0.000 description 6
- PEDCQBHIVMGVHV-UHFFFAOYSA-N Glycerine Chemical compound OCC(O)CO PEDCQBHIVMGVHV-UHFFFAOYSA-N 0.000 description 6
- 108010043121 Green Fluorescent Proteins Proteins 0.000 description 6
- 102000004144 Green Fluorescent Proteins Human genes 0.000 description 6
- 241000124008 Mammalia Species 0.000 description 6
- 241000347336 Melichthys niger Species 0.000 description 6
- 238000010459 TALEN Methods 0.000 description 6
- 108010043645 Transcription Activator-Like Effector Nucleases Proteins 0.000 description 6
- 230000003321 amplification Effects 0.000 description 6
- 230000000692 anti-sense effect Effects 0.000 description 6
- 210000004899 c-terminal region Anatomy 0.000 description 6
- 230000005754 cellular signaling Effects 0.000 description 6
- 239000003153 chemical reaction reagent Substances 0.000 description 6
- 210000000349 chromosome Anatomy 0.000 description 6
- 230000001419 dependent effect Effects 0.000 description 6
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 description 6
- 238000003205 genotyping method Methods 0.000 description 6
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 description 6
- 230000002779 inactivation Effects 0.000 description 6
- 230000006698 induction Effects 0.000 description 6
- 239000003550 marker Substances 0.000 description 6
- 229920001184 polypeptide Polymers 0.000 description 6
- 238000012545 processing Methods 0.000 description 6
- 241000894007 species Species 0.000 description 6
- 230000009261 transgenic effect Effects 0.000 description 6
- 238000012800 visualization Methods 0.000 description 6
- 101150069031 CSN2 gene Proteins 0.000 description 5
- 108700010070 Codon Usage Proteins 0.000 description 5
- 241000701022 Cytomegalovirus Species 0.000 description 5
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 description 5
- 108700020796 Oncogene Proteins 0.000 description 5
- 108091028113 Trans-activating crRNA Proteins 0.000 description 5
- 238000000137 annealing Methods 0.000 description 5
- 239000003795 chemical substances by application Substances 0.000 description 5
- 150000001875 compounds Chemical class 0.000 description 5
- 230000029087 digestion Effects 0.000 description 5
- 230000002255 enzymatic effect Effects 0.000 description 5
- 239000005090 green fluorescent protein Substances 0.000 description 5
- 229910052739 hydrogen Inorganic materials 0.000 description 5
- 239000001257 hydrogen Substances 0.000 description 5
- 238000001727 in vivo Methods 0.000 description 5
- 230000010354 integration Effects 0.000 description 5
- 230000029279 positive regulation of transcription, DNA-dependent Effects 0.000 description 5
- 239000002243 precursor Substances 0.000 description 5
- 238000000746 purification Methods 0.000 description 5
- 229960000268 spectinomycin Drugs 0.000 description 5
- UNFWWIHTNXNPBV-WXKVUWSESA-N spectinomycin Chemical compound O([C@@H]1[C@@H](NC)[C@@H](O)[C@H]([C@@H]([C@H]1O1)O)NC)[C@]2(O)[C@H]1O[C@H](C)CC2=O UNFWWIHTNXNPBV-WXKVUWSESA-N 0.000 description 5
- 241001430294 unidentified retrovirus Species 0.000 description 5
- HJCMDXDYPOUFDY-WHFBIAKZSA-N Ala-Gln Chemical compound C[C@H](N)C(=O)N[C@H](C(O)=O)CCC(N)=O HJCMDXDYPOUFDY-WHFBIAKZSA-N 0.000 description 4
- 102100033312 Alpha-2-macroglobulin Human genes 0.000 description 4
- 208000024827 Alzheimer disease Diseases 0.000 description 4
- LSNNMFCWUKXFEE-UHFFFAOYSA-M Bisulfite Chemical compound OS([O-])=O LSNNMFCWUKXFEE-UHFFFAOYSA-M 0.000 description 4
- 108091003079 Bovine Serum Albumin Proteins 0.000 description 4
- 238000001712 DNA sequencing Methods 0.000 description 4
- 108010070675 Glutathione transferase Proteins 0.000 description 4
- 102100029100 Hematopoietic prostaglandin D synthase Human genes 0.000 description 4
- 241000238631 Hexapoda Species 0.000 description 4
- 101000635854 Homo sapiens Myoglobin Proteins 0.000 description 4
- 101000891092 Homo sapiens TAR DNA-binding protein 43 Proteins 0.000 description 4
- 101000687905 Homo sapiens Transcription factor SOX-2 Proteins 0.000 description 4
- 239000012097 Lipofectamine 2000 Substances 0.000 description 4
- TWRXJAOTZQYOKJ-UHFFFAOYSA-L Magnesium chloride Chemical compound [Mg+2].[Cl-].[Cl-] TWRXJAOTZQYOKJ-UHFFFAOYSA-L 0.000 description 4
- 101100101250 Mus musculus Th gene Proteins 0.000 description 4
- 229930182555 Penicillin Natural products 0.000 description 4
- JGSARLDLIJGVTE-MBNYWOFBSA-N Penicillin G Chemical compound N([C@H]1[C@H]2SC([C@@H](N2C1=O)C(O)=O)(C)C)C(=O)CC1=CC=CC=C1 JGSARLDLIJGVTE-MBNYWOFBSA-N 0.000 description 4
- 102100033616 Phospholipid-transporting ATPase ABCA1 Human genes 0.000 description 4
- 108010015078 Pregnancy-Associated alpha 2-Macroglobulins Proteins 0.000 description 4
- 238000011529 RT qPCR Methods 0.000 description 4
- 102100030547 Serotonin N-acetyltransferase Human genes 0.000 description 4
- 241000694196 Streptococcus pneumoniae R6 Species 0.000 description 4
- 108010021188 Superoxide Dismutase-1 Proteins 0.000 description 4
- 102100038836 Superoxide dismutase [Cu-Zn] Human genes 0.000 description 4
- 102100040347 TAR DNA-binding protein 43 Human genes 0.000 description 4
- 108020004566 Transfer RNA Proteins 0.000 description 4
- 101710176279 Tubulin beta-2 chain Proteins 0.000 description 4
- 108010073925 Vascular Endothelial Growth Factor B Proteins 0.000 description 4
- 102000009520 Vascular Endothelial Growth Factor C Human genes 0.000 description 4
- 108010073923 Vascular Endothelial Growth Factor C Proteins 0.000 description 4
- 102100038217 Vascular endothelial growth factor B Human genes 0.000 description 4
- 239000013543 active substance Substances 0.000 description 4
- 201000008266 amyotrophic lateral sclerosis type 2 Diseases 0.000 description 4
- 208000029560 autism spectrum disease Diseases 0.000 description 4
- 230000009286 beneficial effect Effects 0.000 description 4
- 230000009287 biochemical signal transduction Effects 0.000 description 4
- 238000004113 cell culture Methods 0.000 description 4
- 238000006243 chemical reaction Methods 0.000 description 4
- 230000034994 death Effects 0.000 description 4
- 230000007547 defect Effects 0.000 description 4
- 238000006911 enzymatic reaction Methods 0.000 description 4
- 239000012091 fetal bovine serum Substances 0.000 description 4
- 230000036541 health Effects 0.000 description 4
- 102000049240 human PVALB Human genes 0.000 description 4
- 238000011534 incubation Methods 0.000 description 4
- 230000000977 initiatory effect Effects 0.000 description 4
- 230000003993 interaction Effects 0.000 description 4
- 208000032799 juvenile amyotrophic lateral sclerosis type 2 Diseases 0.000 description 4
- 210000003734 kidney Anatomy 0.000 description 4
- 238000001638 lipofection Methods 0.000 description 4
- 239000011159 matrix material Substances 0.000 description 4
- 239000013642 negative control Substances 0.000 description 4
- 229940049954 penicillin Drugs 0.000 description 4
- 229920000642 polymer Polymers 0.000 description 4
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 description 4
- 238000003259 recombinant expression Methods 0.000 description 4
- 230000010076 replication Effects 0.000 description 4
- QZAYGJVTTNCVMB-UHFFFAOYSA-N serotonin Chemical compound C1=C(O)C=C2C(CCN)=CNC2=C1 QZAYGJVTTNCVMB-UHFFFAOYSA-N 0.000 description 4
- 239000004055 small Interfering RNA Substances 0.000 description 4
- 230000001225 therapeutic effect Effects 0.000 description 4
- 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 3
- 108020005345 3' Untranslated Regions Proteins 0.000 description 3
- 102100035923 4-aminobutyrate aminotransferase, mitochondrial Human genes 0.000 description 3
- FVFVNNKYKYZTJU-UHFFFAOYSA-N 6-chloro-1,3,5-triazine-2,4-diamine Chemical compound NC1=NC(N)=NC(Cl)=N1 FVFVNNKYKYZTJU-UHFFFAOYSA-N 0.000 description 3
- 239000013607 AAV vector Substances 0.000 description 3
- 206010003594 Ataxia telangiectasia Diseases 0.000 description 3
- 238000010446 CRISPR interference Methods 0.000 description 3
- 102100025064 Cellular tumor antigen p53 Human genes 0.000 description 3
- 241000195597 Chlamydomonas reinhardtii 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
- 241000252212 Danio rerio Species 0.000 description 3
- 102000004533 Endonucleases Human genes 0.000 description 3
- 108010042407 Endonucleases Proteins 0.000 description 3
- 241000660147 Escherichia coli str. K-12 substr. MG1655 Species 0.000 description 3
- 108700024394 Exon Proteins 0.000 description 3
- 241000282326 Felis catus Species 0.000 description 3
- 101150106478 GPS1 gene Proteins 0.000 description 3
- 108700039691 Genetic Promoter Regions Proteins 0.000 description 3
- 229940123611 Genome editing Drugs 0.000 description 3
- 239000007995 HEPES buffer Substances 0.000 description 3
- 108091027305 Heteroduplex Proteins 0.000 description 3
- 101000721661 Homo sapiens Cellular tumor antigen p53 Proteins 0.000 description 3
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 description 3
- 241000713666 Lentivirus Species 0.000 description 3
- 101710175625 Maltose/maltodextrin-binding periplasmic protein Proteins 0.000 description 3
- 101100219625 Mus musculus Casd1 gene Proteins 0.000 description 3
- 206010028980 Neoplasm Diseases 0.000 description 3
- 108020004485 Nonsense Codon Proteins 0.000 description 3
- 108020004711 Nucleic Acid Probes Proteins 0.000 description 3
- 102000002488 Nucleoplasmin Human genes 0.000 description 3
- 241000283973 Oryctolagus cuniculus Species 0.000 description 3
- 241000700159 Rattus Species 0.000 description 3
- 206010039491 Sarcoma Diseases 0.000 description 3
- 241000193998 Streptococcus pneumoniae Species 0.000 description 3
- 238000000692 Student's t-test Methods 0.000 description 3
- 108010006785 Taq Polymerase Proteins 0.000 description 3
- 108091023040 Transcription factor Proteins 0.000 description 3
- 102000040945 Transcription factor Human genes 0.000 description 3
- 108010017070 Zinc Finger Nucleases Proteins 0.000 description 3
- 230000002159 abnormal effect Effects 0.000 description 3
- 208000021841 acute erythroid leukemia Diseases 0.000 description 3
- 230000006978 adaptation Effects 0.000 description 3
- 235000004279 alanine Nutrition 0.000 description 3
- 229960000723 ampicillin Drugs 0.000 description 3
- AVKUERGKIZMTKX-NJBDSQKTSA-N ampicillin Chemical compound C1([C@@H](N)C(=O)N[C@H]2[C@H]3SC([C@@H](N3C2=O)C(O)=O)(C)C)=CC=CC=C1 AVKUERGKIZMTKX-NJBDSQKTSA-N 0.000 description 3
- 230000008436 biogenesis Effects 0.000 description 3
- 210000004369 blood Anatomy 0.000 description 3
- 239000008280 blood Substances 0.000 description 3
- 230000008859 change Effects 0.000 description 3
- 230000001332 colony forming effect Effects 0.000 description 3
- 101150055601 cops2 gene Proteins 0.000 description 3
- 108010082025 cyan fluorescent protein Proteins 0.000 description 3
- 206010015037 epilepsy Diseases 0.000 description 3
- 238000011156 evaluation Methods 0.000 description 3
- 210000002950 fibroblast Anatomy 0.000 description 3
- 238000001914 filtration Methods 0.000 description 3
- 238000010363 gene targeting Methods 0.000 description 3
- 230000000415 inactivating effect Effects 0.000 description 3
- 230000000670 limiting effect Effects 0.000 description 3
- 150000002632 lipids Chemical class 0.000 description 3
- 210000004185 liver Anatomy 0.000 description 3
- 238000011068 loading method Methods 0.000 description 3
- 238000010369 molecular cloning Methods 0.000 description 3
- 231100000350 mutagenesis Toxicity 0.000 description 3
- 239000002853 nucleic acid probe Substances 0.000 description 3
- 108060005597 nucleoplasmin Proteins 0.000 description 3
- 210000000496 pancreas Anatomy 0.000 description 3
- 230000001717 pathogenic effect Effects 0.000 description 3
- 238000013081 phylogenetic analysis Methods 0.000 description 3
- 238000003752 polymerase chain reaction Methods 0.000 description 3
- 238000002360 preparation method Methods 0.000 description 3
- 102000005962 receptors Human genes 0.000 description 3
- 108020003175 receptors Proteins 0.000 description 3
- 230000002829 reductive effect Effects 0.000 description 3
- 238000007894 restriction fragment length polymorphism technique Methods 0.000 description 3
- 238000007480 sanger sequencing Methods 0.000 description 3
- 201000000980 schizophrenia Diseases 0.000 description 3
- 230000009897 systematic effect Effects 0.000 description 3
- 238000012353 t test Methods 0.000 description 3
- 230000005026 transcription initiation Effects 0.000 description 3
- 230000005030 transcription termination Effects 0.000 description 3
- 230000001131 transforming effect Effects 0.000 description 3
- 230000014616 translation Effects 0.000 description 3
- 238000013519 translation Methods 0.000 description 3
- 229910021642 ultra pure water Inorganic materials 0.000 description 3
- 239000012498 ultrapure water Substances 0.000 description 3
- 241000701447 unidentified baculovirus Species 0.000 description 3
- 241001515965 unidentified phage Species 0.000 description 3
- 238000010200 validation analysis Methods 0.000 description 3
- 101150092476 ABCA1 gene Proteins 0.000 description 2
- 125000003345 AMP group Chemical group 0.000 description 2
- 108700005241 ATP Binding Cassette Transporter 1 Proteins 0.000 description 2
- 102100036618 ATP-binding cassette sub-family A member 13 Human genes 0.000 description 2
- 102100033618 ATP-binding cassette sub-family A member 2 Human genes 0.000 description 2
- 102100033092 ATP-binding cassette sub-family G member 8 Human genes 0.000 description 2
- 229920001817 Agar Polymers 0.000 description 2
- 102100024401 Alpha-1D adrenergic receptor Human genes 0.000 description 2
- 102100022815 Alpha-2A adrenergic receptor Human genes 0.000 description 2
- 102100025983 Alpha-2C adrenergic receptor Human genes 0.000 description 2
- 108010043324 Amyloid Precursor Protein Secretases Proteins 0.000 description 2
- 102000002659 Amyloid Precursor Protein Secretases Human genes 0.000 description 2
- 102100032187 Androgen receptor Human genes 0.000 description 2
- 102100029470 Apolipoprotein E Human genes 0.000 description 2
- 101710095339 Apolipoprotein E Proteins 0.000 description 2
- 101001123982 Arabidopsis thaliana Protochlorophyllide reductase C, chloroplastic Proteins 0.000 description 2
- 241000203069 Archaea Species 0.000 description 2
- 108010074515 Arylalkylamine N-Acetyltransferase Proteins 0.000 description 2
- 102000007370 Ataxin2 Human genes 0.000 description 2
- 108010032951 Ataxin2 Proteins 0.000 description 2
- 108091032955 Bacterial small RNA Proteins 0.000 description 2
- 208000014644 Brain disease Diseases 0.000 description 2
- 101710155857 C-C motif chemokine 2 Proteins 0.000 description 2
- 108091079001 CRISPR RNA Proteins 0.000 description 2
- 101000909256 Caldicellulosiruptor bescii (strain ATCC BAA-1888 / DSM 6725 / Z-1320) DNA polymerase I Proteins 0.000 description 2
- 102000014914 Carrier Proteins Human genes 0.000 description 2
- 241000282693 Cercopithecidae Species 0.000 description 2
- 102000000018 Chemokine CCL2 Human genes 0.000 description 2
- 241000195585 Chlamydomonas Species 0.000 description 2
- 206010010904 Convulsion Diseases 0.000 description 2
- 239000004971 Cross linker Substances 0.000 description 2
- 230000007067 DNA methylation Effects 0.000 description 2
- 230000033616 DNA repair Effects 0.000 description 2
- 230000006820 DNA synthesis Effects 0.000 description 2
- 230000004568 DNA-binding Effects 0.000 description 2
- 102000001301 EGF receptor Human genes 0.000 description 2
- 108060006698 EGF receptor Proteins 0.000 description 2
- 101100275895 Emericella nidulans (strain FGSC A4 / ATCC 38163 / CBS 112.46 / NRRL 194 / M139) csnB gene Proteins 0.000 description 2
- 101100326871 Escherichia coli (strain K12) ygbF gene Proteins 0.000 description 2
- 241000206602 Eukaryota Species 0.000 description 2
- 108010032606 Fragile X Mental Retardation Protein Proteins 0.000 description 2
- 102000003869 Frataxin Human genes 0.000 description 2
- 108090000217 Frataxin Proteins 0.000 description 2
- 208000024412 Friedreich ataxia Diseases 0.000 description 2
- 241000233866 Fungi Species 0.000 description 2
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 description 2
- 102100040870 Glycine amidinotransferase, mitochondrial Human genes 0.000 description 2
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 description 2
- 206010053185 Glycogen storage disease type II Diseases 0.000 description 2
- 101710154606 Hemagglutinin Proteins 0.000 description 2
- 241000282412 Homo Species 0.000 description 2
- 101001000686 Homo sapiens 4-aminobutyrate aminotransferase, mitochondrial Proteins 0.000 description 2
- 101000929660 Homo sapiens ATP-binding cassette sub-family A member 13 Proteins 0.000 description 2
- 101000801645 Homo sapiens ATP-binding cassette sub-family A member 2 Proteins 0.000 description 2
- 101000756842 Homo sapiens Alpha-2A adrenergic receptor Proteins 0.000 description 2
- 101000720032 Homo sapiens Alpha-2C adrenergic receptor Proteins 0.000 description 2
- 101000893303 Homo sapiens Glycine amidinotransferase, mitochondrial Proteins 0.000 description 2
- 101000891579 Homo sapiens Microtubule-associated protein tau Proteins 0.000 description 2
- 101000801684 Homo sapiens Phospholipid-transporting ATPase ABCA1 Proteins 0.000 description 2
- 101000801640 Homo sapiens Phospholipid-transporting ATPase ABCA3 Proteins 0.000 description 2
- 101001135385 Homo sapiens Prostacyclin synthase Proteins 0.000 description 2
- 101000801643 Homo sapiens Retinal-specific phospholipid-transporting ATPase ABCA4 Proteins 0.000 description 2
- 101100257362 Homo sapiens SOX2 gene Proteins 0.000 description 2
- 101000652292 Homo sapiens Serotonin N-acetyltransferase Proteins 0.000 description 2
- 108010001336 Horseradish Peroxidase Proteins 0.000 description 2
- 241000725303 Human immunodeficiency virus Species 0.000 description 2
- 206010061218 Inflammation Diseases 0.000 description 2
- 102000004388 Interleukin-4 Human genes 0.000 description 2
- 108090000978 Interleukin-4 Proteins 0.000 description 2
- 108020004684 Internal Ribosome Entry Sites Proteins 0.000 description 2
- 108091092195 Intron Proteins 0.000 description 2
- XEEYBQQBJWHFJM-UHFFFAOYSA-N Iron Chemical compound [Fe] XEEYBQQBJWHFJM-UHFFFAOYSA-N 0.000 description 2
- 102100036600 Kynurenine/alpha-aminoadipate aminotransferase, mitochondrial Human genes 0.000 description 2
- CKLJMWTZIZZHCS-REOHCLBHSA-N L-aspartic acid Chemical compound OC(=O)[C@@H](N)CC(O)=O CKLJMWTZIZZHCS-REOHCLBHSA-N 0.000 description 2
- 208000005870 Lafora disease Diseases 0.000 description 2
- 208000014161 Lafora myoclonic epilepsy Diseases 0.000 description 2
- 101710128836 Large T antigen Proteins 0.000 description 2
- 235000007688 Lycopersicon esculentum Nutrition 0.000 description 2
- 102100025169 Max-binding protein MNT Human genes 0.000 description 2
- 241001599018 Melanogaster Species 0.000 description 2
- 102000018697 Membrane Proteins Human genes 0.000 description 2
- 108010052285 Membrane Proteins Proteins 0.000 description 2
- 208000036626 Mental retardation Diseases 0.000 description 2
- 102100040243 Microtubule-associated protein tau Human genes 0.000 description 2
- 241001529936 Murinae Species 0.000 description 2
- 241000714177 Murine leukemia virus Species 0.000 description 2
- 101100494762 Mus musculus Nedd9 gene Proteins 0.000 description 2
- 108010052185 Myotonin-Protein Kinase Proteins 0.000 description 2
- 102100022437 Myotonin-protein kinase Human genes 0.000 description 2
- 208000012902 Nervous system disease Diseases 0.000 description 2
- 208000025966 Neurological disease Diseases 0.000 description 2
- 102100022397 Nitric oxide synthase, brain 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
- 238000012408 PCR amplification Methods 0.000 description 2
- 238000002944 PCR assay Methods 0.000 description 2
- 208000018737 Parkinson disease Diseases 0.000 description 2
- 102100033623 Phospholipid-transporting ATPase ABCA3 Human genes 0.000 description 2
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 description 2
- 108010021757 Polynucleotide 5'-Hydroxyl-Kinase Proteins 0.000 description 2
- 102000008422 Polynucleotide 5'-hydroxyl-kinase Human genes 0.000 description 2
- 102100022033 Presenilin-1 Human genes 0.000 description 2
- 102100022036 Presenilin-2 Human genes 0.000 description 2
- 102000029797 Prion Human genes 0.000 description 2
- 108091000054 Prion Proteins 0.000 description 2
- 208000024777 Prion disease Diseases 0.000 description 2
- 102100033075 Prostacyclin synthase Human genes 0.000 description 2
- 101710176177 Protein A56 Proteins 0.000 description 2
- 108700020978 Proto-Oncogene Proteins 0.000 description 2
- 102000052575 Proto-Oncogene Human genes 0.000 description 2
- 101710130181 Protochlorophyllide reductase A, chloroplastic Proteins 0.000 description 2
- 101710138718 Protochlorophyllide reductase B, chloroplastic Proteins 0.000 description 2
- 101000902592 Pyrococcus furiosus (strain ATCC 43587 / DSM 3638 / JCM 8422 / Vc1) DNA polymerase Proteins 0.000 description 2
- 101100273635 Rattus norvegicus Ccn5 gene Proteins 0.000 description 2
- 108020004511 Recombinant DNA Proteins 0.000 description 2
- 108700008625 Reporter Genes Proteins 0.000 description 2
- 101100273253 Rhizopus niveus RNAP gene Proteins 0.000 description 2
- 102000003661 Ribonuclease III Human genes 0.000 description 2
- 108010057163 Ribonuclease III Proteins 0.000 description 2
- 240000004808 Saccharomyces cerevisiae Species 0.000 description 2
- 241000713311 Simian immunodeficiency virus Species 0.000 description 2
- 108091027967 Small hairpin RNA Proteins 0.000 description 2
- 108020004459 Small interfering 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
- 240000003768 Solanum lycopersicum Species 0.000 description 2
- 102000005157 Somatostatin Human genes 0.000 description 2
- 108010056088 Somatostatin Proteins 0.000 description 2
- 102100023532 Synaptic functional regulator FMR1 Human genes 0.000 description 2
- 102100036407 Thioredoxin Human genes 0.000 description 2
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 description 2
- 102100024270 Transcription factor SOX-2 Human genes 0.000 description 2
- 108700019146 Transgenes Proteins 0.000 description 2
- 238000010162 Tukey test Methods 0.000 description 2
- 108010073929 Vascular Endothelial Growth Factor A Proteins 0.000 description 2
- 102100039037 Vascular endothelial growth factor A Human genes 0.000 description 2
- 102100039066 Very low-density lipoprotein receptor Human genes 0.000 description 2
- 101710177612 Very low-density lipoprotein receptor Proteins 0.000 description 2
- 108020005202 Viral DNA Proteins 0.000 description 2
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 description 2
- 108010091383 Xanthine dehydrogenase Proteins 0.000 description 2
- 102000005773 Xanthine dehydrogenase Human genes 0.000 description 2
- 108010093894 Xanthine oxidase Proteins 0.000 description 2
- 201000004525 Zellweger Syndrome Diseases 0.000 description 2
- ZKHQWZAMYRWXGA-KNYAHOBESA-N [[(2r,3s,4r,5r)-5-(6-aminopurin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl] dihydroxyphosphoryl hydrogen phosphate Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](COP(O)(=O)OP(O)(=O)O[32P](O)(O)=O)[C@@H](O)[C@H]1O ZKHQWZAMYRWXGA-KNYAHOBESA-N 0.000 description 2
- 230000003213 activating effect Effects 0.000 description 2
- 239000008272 agar Substances 0.000 description 2
- 239000011543 agarose gel Substances 0.000 description 2
- 125000003295 alanine group Chemical group N[C@@H](C)C(=O)* 0.000 description 2
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 description 2
- 238000000540 analysis of variance Methods 0.000 description 2
- 108010080146 androgen receptors Proteins 0.000 description 2
- 210000004102 animal cell Anatomy 0.000 description 2
- 239000003242 anti bacterial agent Substances 0.000 description 2
- 230000003466 anti-cipated effect Effects 0.000 description 2
- 229940088710 antibiotic agent Drugs 0.000 description 2
- 101150059062 apln gene Proteins 0.000 description 2
- 229940009098 aspartate Drugs 0.000 description 2
- 230000002146 bilateral effect Effects 0.000 description 2
- 108091008324 binding proteins Proteins 0.000 description 2
- 230000003115 biocidal effect Effects 0.000 description 2
- 230000008827 biological function Effects 0.000 description 2
- 108091005948 blue fluorescent proteins Proteins 0.000 description 2
- 101150117416 cas2 gene Proteins 0.000 description 2
- 210000002421 cell wall Anatomy 0.000 description 2
- 208000003131 cerebrooculofacioskeletal syndrome 1 Diseases 0.000 description 2
- 101150085358 chd8 gene Proteins 0.000 description 2
- 239000003086 colorant Substances 0.000 description 2
- 239000002299 complementary DNA Substances 0.000 description 2
- 230000021615 conjugation Effects 0.000 description 2
- 238000005520 cutting process Methods 0.000 description 2
- 238000007405 data analysis Methods 0.000 description 2
- 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 2
- 238000004520 electroporation Methods 0.000 description 2
- 210000001671 embryonic stem cell Anatomy 0.000 description 2
- 230000002708 enhancing effect Effects 0.000 description 2
- 229920006227 ethylene-grafted-maleic anhydride Polymers 0.000 description 2
- 108091006047 fluorescent proteins Proteins 0.000 description 2
- 102000034287 fluorescent proteins Human genes 0.000 description 2
- 230000037433 frameshift Effects 0.000 description 2
- 238000003633 gene expression assay Methods 0.000 description 2
- 238000012252 genetic analysis Methods 0.000 description 2
- 238000010353 genetic engineering Methods 0.000 description 2
- 230000007614 genetic variation Effects 0.000 description 2
- 201000004502 glycogen storage disease II Diseases 0.000 description 2
- 239000000185 hemagglutinin Substances 0.000 description 2
- 102000047444 human SOX2 Human genes 0.000 description 2
- 230000036039 immunity Effects 0.000 description 2
- 230000001976 improved effect Effects 0.000 description 2
- 230000006872 improvement Effects 0.000 description 2
- 238000010348 incorporation Methods 0.000 description 2
- 230000004054 inflammatory process Effects 0.000 description 2
- 229940028885 interleukin-4 Drugs 0.000 description 2
- 238000009630 liquid culture Methods 0.000 description 2
- 208000002780 macular degeneration Diseases 0.000 description 2
- 229910001629 magnesium chloride Inorganic materials 0.000 description 2
- 230000035800 maturation Effects 0.000 description 2
- 230000011987 methylation Effects 0.000 description 2
- 238000007069 methylation reaction Methods 0.000 description 2
- 108091070501 miRNA Proteins 0.000 description 2
- 239000002679 microRNA Substances 0.000 description 2
- 238000002703 mutagenesis Methods 0.000 description 2
- 231100000219 mutagenic Toxicity 0.000 description 2
- 239000003471 mutagenic agent Substances 0.000 description 2
- 230000003505 mutagenic effect Effects 0.000 description 2
- 230000036438 mutation frequency Effects 0.000 description 2
- 230000009826 neoplastic cell growth Effects 0.000 description 2
- 230000004770 neurodegeneration Effects 0.000 description 2
- 210000002569 neuron Anatomy 0.000 description 2
- 229910052757 nitrogen Inorganic materials 0.000 description 2
- 108091027963 non-coding RNA Proteins 0.000 description 2
- 102000042567 non-coding RNA Human genes 0.000 description 2
- 208000001749 optic atrophy Diseases 0.000 description 2
- 230000001575 pathological effect Effects 0.000 description 2
- 239000002953 phosphate buffered saline Substances 0.000 description 2
- 108010079892 phosphoglycerol kinase Proteins 0.000 description 2
- 229910052698 phosphorus Inorganic materials 0.000 description 2
- 239000011574 phosphorus Substances 0.000 description 2
- 230000026731 phosphorylation Effects 0.000 description 2
- 238000006366 phosphorylation reaction Methods 0.000 description 2
- 238000002264 polyacrylamide gel electrophoresis Methods 0.000 description 2
- 239000013641 positive control Substances 0.000 description 2
- 230000000069 prophylactic effect Effects 0.000 description 2
- 230000003252 repetitive effect Effects 0.000 description 2
- 230000000717 retained effect Effects 0.000 description 2
- 210000003705 ribosome Anatomy 0.000 description 2
- 239000000344 soap Substances 0.000 description 2
- NHXLMOGPVYXJNR-ATOGVRKGSA-N somatostatin Chemical compound C([C@H]1C(=O)N[C@H](C(N[C@@H](CO)C(=O)N[C@@H](CSSC[C@@H](C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](CC=2C=CC=CC=2)C(=O)N[C@@H](CC=2C=CC=CC=2)C(=O)N[C@@H](CC=2C3=CC=CC=C3NC=2)C(=O)N[C@@H](CCCCN)C(=O)N[C@H](C(=O)N1)[C@@H](C)O)NC(=O)CNC(=O)[C@H](C)N)C(O)=O)=O)[C@H](O)C)C1=CC=CC=C1 NHXLMOGPVYXJNR-ATOGVRKGSA-N 0.000 description 2
- 229960000553 somatostatin Drugs 0.000 description 2
- 210000000130 stem cell Anatomy 0.000 description 2
- 229940031000 streptococcus pneumoniae Drugs 0.000 description 2
- 239000006228 supernatant Substances 0.000 description 2
- WWJZWCUNLNYYAU-UHFFFAOYSA-N temephos Chemical compound C1=CC(OP(=S)(OC)OC)=CC=C1SC1=CC=C(OP(=S)(OC)OC)C=C1 WWJZWCUNLNYYAU-UHFFFAOYSA-N 0.000 description 2
- 108060008226 thioredoxin Proteins 0.000 description 2
- 108091006107 transcriptional repressors Proteins 0.000 description 2
- 238000010361 transduction Methods 0.000 description 2
- 230000026683 transduction Effects 0.000 description 2
- 230000001052 transient effect Effects 0.000 description 2
- 239000006150 trypticase soy agar Substances 0.000 description 2
- 238000005406 washing Methods 0.000 description 2
- 108091005957 yellow fluorescent proteins Proteins 0.000 description 2
- BAAVRTJSLCSMNM-CMOCDZPBSA-N (2s)-2-[[(2s)-2-[[(2s)-2-[[(2s)-2-amino-3-(4-hydroxyphenyl)propanoyl]amino]-4-carboxybutanoyl]amino]-3-(4-hydroxyphenyl)propanoyl]amino]pentanedioic acid Chemical compound C([C@H](N)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CC=1C=CC(O)=CC=1)C(=O)N[C@@H](CCC(O)=O)C(O)=O)C1=CC=C(O)C=C1 BAAVRTJSLCSMNM-CMOCDZPBSA-N 0.000 description 1
- GUAHPAJOXVYFON-ZETCQYMHSA-N (8S)-8-amino-7-oxononanoic acid zwitterion Chemical compound C[C@H](N)C(=O)CCCCCC(O)=O GUAHPAJOXVYFON-ZETCQYMHSA-N 0.000 description 1
- FMKJUUQOYOHLTF-OWOJBTEDSA-N (e)-4-azaniumylbut-2-enoate Chemical compound NC\C=C\C(O)=O FMKJUUQOYOHLTF-OWOJBTEDSA-N 0.000 description 1
- GZCWLCBFPRFLKL-UHFFFAOYSA-N 1-prop-2-ynoxypropan-2-ol Chemical compound CC(O)COCC#C GZCWLCBFPRFLKL-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
- JTTIOYHBNXDJOD-UHFFFAOYSA-N 2,4,6-triaminopyrimidine Chemical compound NC1=CC(N)=NC(N)=N1 JTTIOYHBNXDJOD-UHFFFAOYSA-N 0.000 description 1
- 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 1
- 101710094518 4-aminobutyrate aminotransferase Proteins 0.000 description 1
- 102100037991 85/88 kDa calcium-independent phospholipase A2 Human genes 0.000 description 1
- 101150020052 AADAT gene Proteins 0.000 description 1
- 108091022885 ADAM Proteins 0.000 description 1
- 108091060719 AF4 family Proteins 0.000 description 1
- 102000041039 AF4 family Human genes 0.000 description 1
- 102100024378 AF4/FMR2 family member 2 Human genes 0.000 description 1
- 101150037123 APOE gene Proteins 0.000 description 1
- 102100039601 ARF GTPase-activating protein GIT1 Human genes 0.000 description 1
- 102100024643 ATP-binding cassette sub-family D member 1 Human genes 0.000 description 1
- 108010051457 Acid Phosphatase Proteins 0.000 description 1
- 102000013563 Acid Phosphatase Human genes 0.000 description 1
- 208000034012 Acid sphingomyelinase deficiency Diseases 0.000 description 1
- 241000589291 Acinetobacter Species 0.000 description 1
- 108090000104 Actin-related protein 3 Proteins 0.000 description 1
- 102000007469 Actins Human genes 0.000 description 1
- 108010085238 Actins Proteins 0.000 description 1
- 102000008161 Adenosine A3 Receptor Human genes 0.000 description 1
- 108010060261 Adenosine A3 Receptor Proteins 0.000 description 1
- 102100036006 Adenosine receptor A3 Human genes 0.000 description 1
- 201000011452 Adrenoleukodystrophy Diseases 0.000 description 1
- 241000567147 Aeropyrum Species 0.000 description 1
- 101150029691 Aff2 gene Proteins 0.000 description 1
- 241000589158 Agrobacterium Species 0.000 description 1
- 208000024341 Aicardi syndrome Diseases 0.000 description 1
- 208000033237 Aicardi-Goutières syndrome Diseases 0.000 description 1
- 102000009027 Albumins Human genes 0.000 description 1
- 108010088751 Albumins Proteins 0.000 description 1
- 208000011403 Alexander disease Diseases 0.000 description 1
- 208000006829 Allan-Herndon-Dudley syndrome Diseases 0.000 description 1
- 208000023434 Alpers-Huttenlocher syndrome Diseases 0.000 description 1
- 108050000241 Alpha 1D adrenoceptor Proteins 0.000 description 1
- 102100035028 Alpha-L-iduronidase Human genes 0.000 description 1
- 208000029602 Alpha-N-acetylgalactosaminidase deficiency Diseases 0.000 description 1
- 102100023635 Alpha-fetoprotein Human genes 0.000 description 1
- 206010068783 Alstroem syndrome Diseases 0.000 description 1
- 201000005932 Alstrom Syndrome Diseases 0.000 description 1
- 208000026833 Alzheimer disease 4 Diseases 0.000 description 1
- 208000031277 Amaurotic familial idiocy Diseases 0.000 description 1
- 108010079054 Amyloid beta-Protein Precursor Proteins 0.000 description 1
- 102000014303 Amyloid beta-Protein Precursor Human genes 0.000 description 1
- 241000192542 Anabaena Species 0.000 description 1
- 208000009575 Angelman syndrome Diseases 0.000 description 1
- 108010048154 Angiopoietin-1 Proteins 0.000 description 1
- 102000009088 Angiopoietin-1 Human genes 0.000 description 1
- 102100034594 Angiopoietin-1 Human genes 0.000 description 1
- 108020005544 Antisense RNA Proteins 0.000 description 1
- 241000207208 Aquifex Species 0.000 description 1
- 241000205046 Archaeoglobus Species 0.000 description 1
- 241000238421 Arthropoda Species 0.000 description 1
- 102000014461 Ataxins Human genes 0.000 description 1
- 108010078286 Ataxins Proteins 0.000 description 1
- 208000006096 Attention Deficit Disorder with Hyperactivity Diseases 0.000 description 1
- 208000036864 Attention deficit/hyperactivity disease Diseases 0.000 description 1
- 206010003805 Autism Diseases 0.000 description 1
- 208000020706 Autistic disease Diseases 0.000 description 1
- 241000193830 Bacillus <bacterium> Species 0.000 description 1
- 201000005943 Barth syndrome Diseases 0.000 description 1
- 206010061692 Benign muscle neoplasm Diseases 0.000 description 1
- BTBUEUYNUDRHOZ-UHFFFAOYSA-N Borate Chemical compound [O-]B([O-])[O-] BTBUEUYNUDRHOZ-UHFFFAOYSA-N 0.000 description 1
- 102000004219 Brain-derived neurotrophic factor Human genes 0.000 description 1
- 108090000715 Brain-derived neurotrophic factor Proteins 0.000 description 1
- 102100035875 C-C chemokine receptor type 5 Human genes 0.000 description 1
- 101710149870 C-C chemokine receptor type 5 Proteins 0.000 description 1
- 102100021943 C-C motif chemokine 2 Human genes 0.000 description 1
- 102000014814 CACNA1C Human genes 0.000 description 1
- 208000010482 CADASIL Diseases 0.000 description 1
- 101150052909 CCL2 gene Proteins 0.000 description 1
- 101150083327 CCR2 gene Proteins 0.000 description 1
- 101150017501 CCR5 gene Proteins 0.000 description 1
- 108060001826 COP1 Proteins 0.000 description 1
- 238000010356 CRISPR-Cas9 genome editing Methods 0.000 description 1
- 101150018129 CSF2 gene Proteins 0.000 description 1
- 101150023944 CXCR5 gene Proteins 0.000 description 1
- 102100027557 Calcipressin-1 Human genes 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
- 208000022526 Canavan disease Diseases 0.000 description 1
- 102100025570 Cancer/testis antigen 1 Human genes 0.000 description 1
- 208000024172 Cardiovascular disease Diseases 0.000 description 1
- 102100025634 Caspase recruitment domain-containing protein 16 Human genes 0.000 description 1
- 108090000994 Catalytic RNA Proteins 0.000 description 1
- 102000053642 Catalytic RNA Human genes 0.000 description 1
- 108090000712 Cathepsin B Proteins 0.000 description 1
- 102000004225 Cathepsin B Human genes 0.000 description 1
- 102100021633 Cathepsin B Human genes 0.000 description 1
- 108090000625 Cathepsin K Proteins 0.000 description 1
- 102000004171 Cathepsin K Human genes 0.000 description 1
- 102100024940 Cathepsin K Human genes 0.000 description 1
- 208000033221 Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy Diseases 0.000 description 1
- 208000033935 Cerebral autosomal dominant arteriopathy-subcortical infarcts-leukoencephalopathy Diseases 0.000 description 1
- 108010012236 Chemokines Proteins 0.000 description 1
- 102000019034 Chemokines Human genes 0.000 description 1
- 241000191366 Chlorobium Species 0.000 description 1
- 102000017589 Chromo domains Human genes 0.000 description 1
- 108050005811 Chromo domains Proteins 0.000 description 1
- 241000588881 Chromobacterium Species 0.000 description 1
- 241000193403 Clostridium Species 0.000 description 1
- 101100007328 Cocos nucifera COS-1 gene Proteins 0.000 description 1
- 102000002734 Collagen Type VI Human genes 0.000 description 1
- 108010043741 Collagen Type VI Proteins 0.000 description 1
- 102100033601 Collagen alpha-1(I) chain Human genes 0.000 description 1
- 206010010099 Combined immunodeficiency Diseases 0.000 description 1
- 101710082261 Competence-stimulating peptide Proteins 0.000 description 1
- 102100027826 Complexin-1 Human genes 0.000 description 1
- 208000037141 Congenital muscular dystrophy, Fukuyama type Diseases 0.000 description 1
- 102000020856 Copper Transport Proteins Human genes 0.000 description 1
- 108091004554 Copper Transport Proteins Proteins 0.000 description 1
- 201000009343 Cornelia de Lange syndrome Diseases 0.000 description 1
- 241000186216 Corynebacterium Species 0.000 description 1
- 241000195493 Cryptophyta Species 0.000 description 1
- 101150074775 Csf1 gene Proteins 0.000 description 1
- 101150074155 DHFR gene Proteins 0.000 description 1
- 230000004544 DNA amplification Effects 0.000 description 1
- 230000005971 DNA damage repair Effects 0.000 description 1
- 108010063593 DNA modification methylase SssI Proteins 0.000 description 1
- 102100036951 DNA polymerase subunit gamma-1 Human genes 0.000 description 1
- 241000450599 DNA viruses Species 0.000 description 1
- 102000052510 DNA-Binding Proteins Human genes 0.000 description 1
- 101710096438 DNA-binding protein Proteins 0.000 description 1
- 108010014303 DNA-directed DNA polymerase Proteins 0.000 description 1
- 102000016928 DNA-directed DNA polymerase Human genes 0.000 description 1
- 208000003471 De Lange Syndrome Diseases 0.000 description 1
- 206010012289 Dementia Diseases 0.000 description 1
- 206010012335 Dependence Diseases 0.000 description 1
- 241000605716 Desulfovibrio Species 0.000 description 1
- 208000004986 Diffuse Cerebral Sclerosis of Schilder Diseases 0.000 description 1
- 101150088306 Disc1 gene Proteins 0.000 description 1
- 206010061818 Disease progression Diseases 0.000 description 1
- 102100022825 Disintegrin and metalloproteinase domain-containing protein 22 Human genes 0.000 description 1
- 201000007547 Dravet syndrome Diseases 0.000 description 1
- 102100028554 Dual specificity tyrosine-phosphorylation-regulated kinase 1A Human genes 0.000 description 1
- 108010040648 Dyrk kinase Proteins 0.000 description 1
- 102100038132 Endogenous retrovirus group K member 6 Pro protein Human genes 0.000 description 1
- 108010013369 Enteropeptidase Proteins 0.000 description 1
- 102100029727 Enteropeptidase Human genes 0.000 description 1
- 101710091045 Envelope protein Proteins 0.000 description 1
- YQYJSBFKSSDGFO-UHFFFAOYSA-N Epihygromycin Natural products OC1C(O)C(C(=O)C)OC1OC(C(=C1)O)=CC=C1C=C(C)C(=O)NC1C(O)C(O)C2OCOC2C1O YQYJSBFKSSDGFO-UHFFFAOYSA-N 0.000 description 1
- 241000588698 Erwinia Species 0.000 description 1
- 241000588722 Escherichia Species 0.000 description 1
- 108091029865 Exogenous DNA Proteins 0.000 description 1
- 101150107205 FCGR2 gene Proteins 0.000 description 1
- 101150061264 FXR1 gene Proteins 0.000 description 1
- 208000024720 Fabry Disease Diseases 0.000 description 1
- 108010074860 Factor Xa Proteins 0.000 description 1
- 241000192125 Firmicutes Species 0.000 description 1
- 102100036334 Fragile X mental retardation syndrome-related protein 1 Human genes 0.000 description 1
- 102100036336 Fragile X mental retardation syndrome-related protein 2 Human genes 0.000 description 1
- 208000001914 Fragile X syndrome Diseases 0.000 description 1
- 208000002234 Fryns syndrome Diseases 0.000 description 1
- 201000006813 Fukuyama congenital muscular dystrophy Diseases 0.000 description 1
- 241000223221 Fusarium oxysporum Species 0.000 description 1
- 241000611205 Fusarium oxysporum f. sp. lycopersici Species 0.000 description 1
- 241000605909 Fusobacterium Species 0.000 description 1
- 102000004300 GABA-A Receptors Human genes 0.000 description 1
- 108090000839 GABA-A Receptors Proteins 0.000 description 1
- 241001123946 Gaga Species 0.000 description 1
- 208000017462 Galactosialidosis Diseases 0.000 description 1
- 102100039556 Galectin-4 Human genes 0.000 description 1
- 102100033713 Gamma-secretase subunit APH-1B Human genes 0.000 description 1
- 102100028260 Gamma-secretase subunit PEN-2 Human genes 0.000 description 1
- 208000015872 Gaucher disease Diseases 0.000 description 1
- 208000031448 Genomic Instability Diseases 0.000 description 1
- 241001135750 Geobacter Species 0.000 description 1
- 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 description 1
- 102000003676 Glucocorticoid Receptors Human genes 0.000 description 1
- 108090000079 Glucocorticoid Receptors Proteins 0.000 description 1
- 108010060309 Glucuronidase Proteins 0.000 description 1
- 102000053187 Glucuronidase Human genes 0.000 description 1
- 102100031181 Glyceraldehyde-3-phosphate dehydrogenase Human genes 0.000 description 1
- 239000004471 Glycine Substances 0.000 description 1
- 108010051975 Glycogen Synthase Kinase 3 beta Proteins 0.000 description 1
- 102100022975 Glycogen synthase kinase-3 alpha Human genes 0.000 description 1
- 102100038104 Glycogen synthase kinase-3 beta Human genes 0.000 description 1
- 241000288105 Grus Species 0.000 description 1
- 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 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
- 208000036066 Hemophagocytic Lymphohistiocytosis Diseases 0.000 description 1
- 101001023784 Heteractis crispa GFP-like non-fluorescent chromoprotein Proteins 0.000 description 1
- 102000016871 Hexosaminidase A Human genes 0.000 description 1
- 108010053317 Hexosaminidase A Proteins 0.000 description 1
- 208000032672 Histiocytosis haematophagic Diseases 0.000 description 1
- 241001272567 Hominoidea Species 0.000 description 1
- 101000833172 Homo sapiens AF4/FMR2 family member 2 Proteins 0.000 description 1
- 101000888659 Homo sapiens ARF GTPase-activating protein GIT1 Proteins 0.000 description 1
- 101000800430 Homo sapiens ATP-binding cassette sub-family G member 8 Proteins 0.000 description 1
- 101000783645 Homo sapiens Adenosine receptor A3 Proteins 0.000 description 1
- 101000775498 Homo sapiens Adenylate cyclase type 10 Proteins 0.000 description 1
- 101001019502 Homo sapiens Alpha-L-iduronidase Proteins 0.000 description 1
- 101000924552 Homo sapiens Angiopoietin-1 Proteins 0.000 description 1
- 101000897480 Homo sapiens C-C motif chemokine 2 Proteins 0.000 description 1
- 101000856237 Homo sapiens Cancer/testis antigen 1 Proteins 0.000 description 1
- 101000898449 Homo sapiens Cathepsin B Proteins 0.000 description 1
- 101000761509 Homo sapiens Cathepsin K Proteins 0.000 description 1
- 101000859600 Homo sapiens Complexin-1 Proteins 0.000 description 1
- 101000804964 Homo sapiens DNA polymerase subunit gamma-1 Proteins 0.000 description 1
- 101000756722 Homo sapiens Disintegrin and metalloproteinase domain-containing protein 22 Proteins 0.000 description 1
- 101000619542 Homo sapiens E3 ubiquitin-protein ligase parkin Proteins 0.000 description 1
- 101000938351 Homo sapiens Ephrin type-A receptor 3 Proteins 0.000 description 1
- 101100335685 Homo sapiens FXR2 gene Proteins 0.000 description 1
- 101000930945 Homo sapiens Fragile X mental retardation syndrome-related protein 1 Proteins 0.000 description 1
- 101000930952 Homo sapiens Fragile X mental retardation syndrome-related protein 2 Proteins 0.000 description 1
- 101000608765 Homo sapiens Galectin-4 Proteins 0.000 description 1
- 101000733778 Homo sapiens Gamma-secretase subunit APH-1B Proteins 0.000 description 1
- 101000579663 Homo sapiens Gamma-secretase subunit PEN-2 Proteins 0.000 description 1
- 101000903717 Homo sapiens Glycogen synthase kinase-3 alpha Proteins 0.000 description 1
- 101000843809 Homo sapiens Hydroxycarboxylic acid receptor 2 Proteins 0.000 description 1
- 101000929733 Homo sapiens Kynurenine/alpha-aminoadipate aminotransferase, mitochondrial Proteins 0.000 description 1
- 101000917826 Homo sapiens Low affinity immunoglobulin gamma Fc region receptor II-a Proteins 0.000 description 1
- 101000917824 Homo sapiens Low affinity immunoglobulin gamma Fc region receptor II-b Proteins 0.000 description 1
- 101001057159 Homo sapiens Melanoma-associated antigen C3 Proteins 0.000 description 1
- 101000982010 Homo sapiens Myelin proteolipid protein Proteins 0.000 description 1
- 101000837517 Homo sapiens NEDD8-activating enzyme E1 catalytic subunit Proteins 0.000 description 1
- 101001108436 Homo sapiens Neurexin-1 Proteins 0.000 description 1
- 101001108433 Homo sapiens Neurexin-1-beta Proteins 0.000 description 1
- 101000724418 Homo sapiens Neutral amino acid transporter B(0) Proteins 0.000 description 1
- 101000974009 Homo sapiens Nitric oxide synthase, brain Proteins 0.000 description 1
- 101000595929 Homo sapiens POLG alternative reading frame Proteins 0.000 description 1
- 101001064282 Homo sapiens Platelet-activating factor acetylhydrolase IB subunit beta Proteins 0.000 description 1
- 101000617536 Homo sapiens Presenilin-1 Proteins 0.000 description 1
- 101000617546 Homo sapiens Presenilin-2 Proteins 0.000 description 1
- 101001001272 Homo sapiens Prostatic acid phosphatase Proteins 0.000 description 1
- 101000600434 Homo sapiens Putative uncharacterized protein encoded by MIR7-3HG Proteins 0.000 description 1
- 101100247605 Homo sapiens RCAN1 gene Proteins 0.000 description 1
- 101000665449 Homo sapiens RNA binding protein fox-1 homolog 1 Proteins 0.000 description 1
- 101001012157 Homo sapiens Receptor tyrosine-protein kinase erbB-2 Proteins 0.000 description 1
- 101000605835 Homo sapiens Serine/threonine-protein kinase PINK1, mitochondrial Proteins 0.000 description 1
- 101000600903 Homo sapiens Substance-P receptor Proteins 0.000 description 1
- 101000892398 Homo sapiens Tryptophan 2,3-dioxygenase Proteins 0.000 description 1
- 101000830742 Homo sapiens Tryptophan 5-hydroxylase 1 Proteins 0.000 description 1
- 101000851865 Homo sapiens Tryptophan 5-hydroxylase 2 Proteins 0.000 description 1
- 101000807306 Homo sapiens Ubiquitin-like modifier-activating enzyme 1 Proteins 0.000 description 1
- 101000867811 Homo sapiens Voltage-dependent L-type calcium channel subunit alpha-1C Proteins 0.000 description 1
- 101000983956 Homo sapiens Voltage-dependent L-type calcium channel subunit beta-2 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
- 208000025500 Hutchinson-Gilford progeria syndrome Diseases 0.000 description 1
- 102100030643 Hydroxycarboxylic acid receptor 2 Human genes 0.000 description 1
- 241000282620 Hylobates sp. Species 0.000 description 1
- 108010073807 IgG Receptors Proteins 0.000 description 1
- 102000009490 IgG Receptors Human genes 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
- 208000028547 Inborn Urea Cycle disease Diseases 0.000 description 1
- 102000000589 Interleukin-1 Human genes 0.000 description 1
- 108010002352 Interleukin-1 Proteins 0.000 description 1
- 208000021548 Jervell and Lange-Nielsen syndrome Diseases 0.000 description 1
- 201000003992 Jervell-Lange Nielsen Syndrome Diseases 0.000 description 1
- 208000028226 Krabbe disease Diseases 0.000 description 1
- 208000012383 LAMA2-related muscular dystrophy Diseases 0.000 description 1
- 208000031905 Laminin subunit alpha 2-related muscular dystrophy Diseases 0.000 description 1
- 102100022745 Laminin subunit alpha-2 Human genes 0.000 description 1
- 241000589248 Legionella Species 0.000 description 1
- 208000007764 Legionnaires' Disease Diseases 0.000 description 1
- 208000006136 Leigh Disease Diseases 0.000 description 1
- 208000017507 Leigh syndrome Diseases 0.000 description 1
- 208000009625 Lesch-Nyhan syndrome Diseases 0.000 description 1
- 108010020246 Leucine-Rich Repeat Serine-Threonine Protein Kinase-2 Proteins 0.000 description 1
- 102100032693 Leucine-rich repeat serine/threonine-protein kinase 2 Human genes 0.000 description 1
- 108090000364 Ligases Proteins 0.000 description 1
- 102000003960 Ligases Human genes 0.000 description 1
- 206010048911 Lissencephaly Diseases 0.000 description 1
- 241000186781 Listeria Species 0.000 description 1
- 206010057926 Long QT syndrome congenital Diseases 0.000 description 1
- 102100029204 Low affinity immunoglobulin gamma Fc region receptor II-a Human genes 0.000 description 1
- 108060001084 Luciferase Proteins 0.000 description 1
- 239000005089 Luciferase Substances 0.000 description 1
- 239000006142 Luria-Bertani Agar Substances 0.000 description 1
- 208000003221 Lysosomal acid lipase deficiency Diseases 0.000 description 1
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 description 1
- 208000024889 MECP2 duplication syndrome Diseases 0.000 description 1
- 101150083522 MECP2 gene Proteins 0.000 description 1
- 239000007993 MOPS buffer Substances 0.000 description 1
- 101001129124 Mannheimia haemolytica Outer membrane lipoprotein 1 Proteins 0.000 description 1
- 208000030162 Maple syrup disease Diseases 0.000 description 1
- 102000000440 Melanoma-associated antigen Human genes 0.000 description 1
- 108050008953 Melanoma-associated antigen Proteins 0.000 description 1
- 108010049137 Member 1 Subfamily D ATP Binding Cassette Transporter Proteins 0.000 description 1
- 108010090822 Member 8 Subfamily G ATP Binding Cassette Transporter Proteins 0.000 description 1
- 201000011442 Metachromatic leukodystrophy Diseases 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
- 102000006890 Methyl-CpG-Binding Protein 2 Human genes 0.000 description 1
- 108010072388 Methyl-CpG-Binding Protein 2 Proteins 0.000 description 1
- 102100039124 Methyl-CpG-binding protein 2 Human genes 0.000 description 1
- 241000589345 Methylococcus Species 0.000 description 1
- 108060004795 Methyltransferase Proteins 0.000 description 1
- 108020005196 Mitochondrial DNA Proteins 0.000 description 1
- 206010051403 Mitochondrial DNA deletion Diseases 0.000 description 1
- 206010058799 Mitochondrial encephalomyopathy Diseases 0.000 description 1
- 208000034819 Mobility Limitation Diseases 0.000 description 1
- 208000033180 Monosomy 22q13.3 Diseases 0.000 description 1
- 208000008955 Mucolipidoses Diseases 0.000 description 1
- 206010072927 Mucolipidosis type I Diseases 0.000 description 1
- 206010072928 Mucolipidosis type II Diseases 0.000 description 1
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 description 1
- 208000028781 Mucopolysaccharidosis type 1 Diseases 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
- 101100078999 Mus musculus Mx1 gene Proteins 0.000 description 1
- 241000699670 Mus sp. Species 0.000 description 1
- 208000007101 Muscle Cramp Diseases 0.000 description 1
- 102100038895 Myc proto-oncogene protein Human genes 0.000 description 1
- 101710135898 Myc proto-oncogene protein 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
- 102100026784 Myelin proteolipid protein Human genes 0.000 description 1
- 208000036572 Myoclonic epilepsy Diseases 0.000 description 1
- 108010062374 Myoglobin Proteins 0.000 description 1
- 201000004458 Myoma Diseases 0.000 description 1
- 241000863420 Myxococcus Species 0.000 description 1
- 108010005529 N-acetylglutaminylglutamine amide Proteins 0.000 description 1
- KLQXKYZBJWERSF-YUMQZZPRSA-N N2-Acetyl-L-glutaminyl-L-glutamamide Chemical compound NC(=O)CC[C@H](NC(=O)C)C(=O)N[C@@H](CCC(N)=O)C(N)=O KLQXKYZBJWERSF-YUMQZZPRSA-N 0.000 description 1
- 102100031911 NEDD8 Human genes 0.000 description 1
- 108700004934 NEDD8 Proteins 0.000 description 1
- 101150107958 NEDD8 gene Proteins 0.000 description 1
- 102100028719 NEDD8-activating enzyme E1 catalytic subunit Human genes 0.000 description 1
- 108010054200 NR2B NMDA receptor Proteins 0.000 description 1
- 101150026563 NR4A2 gene Proteins 0.000 description 1
- 108091061960 Naked DNA Proteins 0.000 description 1
- 241000588653 Neisseria Species 0.000 description 1
- 108700019961 Neoplasm Genes Proteins 0.000 description 1
- 102000048850 Neoplasm Genes Human genes 0.000 description 1
- 102000048238 Neuregulin-1 Human genes 0.000 description 1
- 108090000556 Neuregulin-1 Proteins 0.000 description 1
- 102100021582 Neurexin-1-beta Human genes 0.000 description 1
- 108010088373 Neurofilament Proteins Proteins 0.000 description 1
- 102000008763 Neurofilament Proteins Human genes 0.000 description 1
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 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
- 102100028267 Neutral amino acid transporter B(0) Human genes 0.000 description 1
- 208000010577 Niemann-Pick disease type C Diseases 0.000 description 1
- 108010008858 Nitric Oxide Synthase Type I Proteins 0.000 description 1
- 241000605122 Nitrosomonas Species 0.000 description 1
- 108091092724 Noncoding DNA Proteins 0.000 description 1
- 208000035544 Nonketotic hyperglycinaemia Diseases 0.000 description 1
- 108091005461 Nucleic proteins Proteins 0.000 description 1
- 101000761187 Odontomachus monticola U-poneritoxin(01)-Om1a Proteins 0.000 description 1
- 108700026244 Open Reading Frames Proteins 0.000 description 1
- 101100532088 Oryza sativa subsp. japonica RUB2 gene Proteins 0.000 description 1
- 101100532090 Oryza sativa subsp. japonica RUB3 gene Proteins 0.000 description 1
- 206010031243 Osteogenesis imperfecta Diseases 0.000 description 1
- 101710125553 PLA2G6 Proteins 0.000 description 1
- 108010011536 PTEN Phosphohydrolase Proteins 0.000 description 1
- 241000606860 Pasteurella Species 0.000 description 1
- 241001494479 Pecora Species 0.000 description 1
- 241000009328 Perro Species 0.000 description 1
- 201000004316 Perry syndrome Diseases 0.000 description 1
- 201000006880 Phelan-McDermid syndrome Diseases 0.000 description 1
- 102100032543 Phosphatidylinositol 3,4,5-trisphosphate 3-phosphatase and dual-specificity protein phosphatase PTEN Human genes 0.000 description 1
- 241000607568 Photobacterium Species 0.000 description 1
- ZYFVNVRFVHJEIU-UHFFFAOYSA-N PicoGreen Chemical compound CN(C)CCCN(CCCN(C)C)C1=CC(=CC2=[N+](C3=CC=CC=C3S2)C)C2=CC=CC=C2N1C1=CC=CC=C1 ZYFVNVRFVHJEIU-UHFFFAOYSA-N 0.000 description 1
- 241000204826 Picrophilus Species 0.000 description 1
- 102100030655 Platelet-activating factor acetylhydrolase IB subunit beta Human genes 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
- 108010036933 Presenilin-1 Proteins 0.000 description 1
- 108010036908 Presenilin-2 Proteins 0.000 description 1
- 108010050254 Presenilins Proteins 0.000 description 1
- 102000015499 Presenilins Human genes 0.000 description 1
- 208000007932 Progeria Diseases 0.000 description 1
- 208000033063 Progressive myoclonic epilepsy Diseases 0.000 description 1
- 102100035703 Prostatic acid phosphatase Human genes 0.000 description 1
- 229940124158 Protease/peptidase inhibitor Drugs 0.000 description 1
- 102000007659 Protein Deglycase DJ-1 Human genes 0.000 description 1
- 108010032428 Protein Deglycase DJ-1 Proteins 0.000 description 1
- 108010029485 Protein Isoforms Proteins 0.000 description 1
- 102000001708 Protein Isoforms Human genes 0.000 description 1
- 102100029812 Protein S100-A12 Human genes 0.000 description 1
- 101710110949 Protein S100-A12 Proteins 0.000 description 1
- 101710188315 Protein X Proteins 0.000 description 1
- 208000033876 Proximal Xq28 duplication syndrome Diseases 0.000 description 1
- 241000567197 Puccinia graminis f. sp. tritici 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
- 102000009572 RNA Polymerase II Human genes 0.000 description 1
- 108010009460 RNA Polymerase II Proteins 0.000 description 1
- 102000014450 RNA Polymerase III Human genes 0.000 description 1
- 108010078067 RNA Polymerase III Proteins 0.000 description 1
- 102100038188 RNA binding protein fox-1 homolog 1 Human genes 0.000 description 1
- 238000010240 RT-PCR analysis Methods 0.000 description 1
- 101100240886 Rattus norvegicus Nptx2 gene Proteins 0.000 description 1
- 101100047461 Rattus norvegicus Trpm8 gene Proteins 0.000 description 1
- 102100030086 Receptor tyrosine-protein kinase erbB-2 Human genes 0.000 description 1
- 101710100969 Receptor tyrosine-protein kinase erbB-3 Proteins 0.000 description 1
- 102100029986 Receptor tyrosine-protein kinase erbB-3 Human genes 0.000 description 1
- 102100029981 Receptor tyrosine-protein kinase erbB-4 Human genes 0.000 description 1
- 101710100963 Receptor tyrosine-protein kinase erbB-4 Proteins 0.000 description 1
- 102100033617 Retinal-specific phospholipid-transporting ATPase ABCA4 Human genes 0.000 description 1
- 201000000582 Retinoblastoma Diseases 0.000 description 1
- 201000008539 Rhizomelic chondrodysplasia punctata type 1 Diseases 0.000 description 1
- 108091028664 Ribonucleotide Proteins 0.000 description 1
- 201000001718 Roberts syndrome Diseases 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
- 208000021811 Sandhoff disease Diseases 0.000 description 1
- 101100023124 Schizosaccharomyces pombe (strain 972 / ATCC 24843) mfr2 gene Proteins 0.000 description 1
- 101150068315 Scn2a gene Proteins 0.000 description 1
- 108091081021 Sense strand Proteins 0.000 description 1
- 102100038376 Serine/threonine-protein kinase PINK1, mitochondrial Human genes 0.000 description 1
- 102220520989 Serine/threonine-protein phosphatase 2A regulatory subunit B'' subunit alpha_K42T_mutation Human genes 0.000 description 1
- 206010073677 Severe myoclonic epilepsy of infancy Diseases 0.000 description 1
- 201000007410 Smith-Lemli-Opitz syndrome Diseases 0.000 description 1
- 208000005392 Spasm Diseases 0.000 description 1
- 241000191940 Staphylococcus Species 0.000 description 1
- 108091081024 Start codon Proteins 0.000 description 1
- 102100036325 Sterol 26-hydroxylase, mitochondrial Human genes 0.000 description 1
- 241000187747 Streptomyces Species 0.000 description 1
- 102100037346 Substance-P receptor Human genes 0.000 description 1
- 241000205101 Sulfolobus Species 0.000 description 1
- 108700005078 Synthetic Genes Proteins 0.000 description 1
- 108091008874 T cell receptors Proteins 0.000 description 1
- 102000016266 T-Cell Antigen Receptors Human genes 0.000 description 1
- 102100036049 T-complex protein 1 subunit gamma Human genes 0.000 description 1
- 239000012163 TRI reagent Substances 0.000 description 1
- 101710149430 Tachykinin-1 Proteins 0.000 description 1
- 241000255588 Tephritidae Species 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
- 229910052776 Thorium Inorganic materials 0.000 description 1
- 108010022394 Threonine synthase Proteins 0.000 description 1
- 108090000190 Thrombin Proteins 0.000 description 1
- 208000035317 Total hypoxanthine-guanine phosphoribosyl transferase deficiency Diseases 0.000 description 1
- 241000283907 Tragelaphus oryx Species 0.000 description 1
- 108700009124 Transcription Initiation Site Proteins 0.000 description 1
- 101710150448 Transcriptional regulator Myc Proteins 0.000 description 1
- 241000589886 Treponema Species 0.000 description 1
- 239000007983 Tris buffer Substances 0.000 description 1
- 241000209140 Triticum Species 0.000 description 1
- 235000021307 Triticum Nutrition 0.000 description 1
- 229920004890 Triton X-100 Polymers 0.000 description 1
- 239000013504 Triton X-100 Substances 0.000 description 1
- 102100040653 Tryptophan 2,3-dioxygenase Human genes 0.000 description 1
- 102100024971 Tryptophan 5-hydroxylase 1 Human genes 0.000 description 1
- 208000007930 Type C Niemann-Pick Disease Diseases 0.000 description 1
- 108091026822 U6 spliceosomal RNA Proteins 0.000 description 1
- 101150044377 UBA1 gene Proteins 0.000 description 1
- 101150035006 UBA3 gene Proteins 0.000 description 1
- 108090000848 Ubiquitin Proteins 0.000 description 1
- 102000044159 Ubiquitin Human genes 0.000 description 1
- 108010005656 Ubiquitin Thiolesterase Proteins 0.000 description 1
- 102000005918 Ubiquitin Thiolesterase Human genes 0.000 description 1
- 102100037160 Ubiquitin-like modifier-activating enzyme 1 Human genes 0.000 description 1
- 208000014769 Usher Syndromes Diseases 0.000 description 1
- 241000251539 Vertebrata <Metazoa> Species 0.000 description 1
- 108700005077 Viral Genes Proteins 0.000 description 1
- 101150115477 Vldlr gene Proteins 0.000 description 1
- 102100025807 Voltage-dependent L-type calcium channel subunit beta-2 Human genes 0.000 description 1
- 201000006793 Walker-Warburg syndrome Diseases 0.000 description 1
- 239000005862 Whey Substances 0.000 description 1
- 102000007544 Whey Proteins Human genes 0.000 description 1
- 108010046377 Whey Proteins Proteins 0.000 description 1
- 208000006254 Wolf-Hirschhorn Syndrome Diseases 0.000 description 1
- 241000605941 Wolinella Species 0.000 description 1
- 241001492404 Woodchuck hepatitis virus Species 0.000 description 1
- 241000589634 Xanthomonas Species 0.000 description 1
- 201000006083 Xeroderma Pigmentosum Diseases 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
- 230000005856 abnormality Effects 0.000 description 1
- 230000021736 acetylation Effects 0.000 description 1
- 238000006640 acetylation reaction Methods 0.000 description 1
- 239000012190 activator Substances 0.000 description 1
- 230000003044 adaptive effect Effects 0.000 description 1
- 210000005006 adaptive immune system Anatomy 0.000 description 1
- 201000009628 adenosine deaminase deficiency Diseases 0.000 description 1
- 230000002411 adverse Effects 0.000 description 1
- 238000001261 affinity purification Methods 0.000 description 1
- 206010064930 age-related macular degeneration Diseases 0.000 description 1
- 108010029483 alpha 1 Chain Collagen Type I Proteins 0.000 description 1
- 102000009899 alpha Karyopherins Human genes 0.000 description 1
- 108010077099 alpha Karyopherins Proteins 0.000 description 1
- 108020004102 alpha-1 Adrenergic Receptor Proteins 0.000 description 1
- 108010026331 alpha-Fetoproteins Proteins 0.000 description 1
- 201000008333 alpha-mannosidosis Diseases 0.000 description 1
- 125000000539 amino acid group Chemical group 0.000 description 1
- 230000019552 anatomical structure morphogenesis Effects 0.000 description 1
- 230000003042 antagnostic effect Effects 0.000 description 1
- 239000000427 antigen Substances 0.000 description 1
- 108091007433 antigens Proteins 0.000 description 1
- 102000036639 antigens Human genes 0.000 description 1
- 239000003293 antisense oligodeoxyribonucleotide Substances 0.000 description 1
- 230000006907 apoptotic process Effects 0.000 description 1
- 235000009697 arginine Nutrition 0.000 description 1
- 150000001484 arginines Chemical class 0.000 description 1
- 238000003149 assay kit Methods 0.000 description 1
- 230000005784 autoimmunity Effects 0.000 description 1
- 208000036556 autosomal recessive T cell-negative B cell-negative NK cell-negative due to adenosine deaminase deficiency severe combined immunodeficiency Diseases 0.000 description 1
- 210000003578 bacterial chromosome Anatomy 0.000 description 1
- 230000037429 base substitution Effects 0.000 description 1
- 208000005980 beta thalassemia Diseases 0.000 description 1
- 230000008236 biological pathway Effects 0.000 description 1
- 238000001369 bisulfite sequencing Methods 0.000 description 1
- 230000000903 blocking effect Effects 0.000 description 1
- 238000006664 bond formation reaction Methods 0.000 description 1
- 210000000988 bone and bone Anatomy 0.000 description 1
- 210000004556 brain Anatomy 0.000 description 1
- 201000011510 cancer Diseases 0.000 description 1
- 229940041514 candida albicans extract Drugs 0.000 description 1
- 229910002091 carbon monoxide Inorganic materials 0.000 description 1
- 125000003178 carboxy group Chemical group [H]OC(*)=O 0.000 description 1
- 230000015556 catabolic process Effects 0.000 description 1
- 125000002091 cationic group Chemical group 0.000 description 1
- 230000001364 causal effect Effects 0.000 description 1
- 101150062912 cct3 gene Proteins 0.000 description 1
- 230000022131 cell cycle Effects 0.000 description 1
- 230000032823 cell division Effects 0.000 description 1
- 239000013592 cell lysate Substances 0.000 description 1
- 210000003855 cell nucleus Anatomy 0.000 description 1
- 108091092328 cellular RNA Proteins 0.000 description 1
- 230000001413 cellular effect Effects 0.000 description 1
- 208000025434 cerebellar degeneration Diseases 0.000 description 1
- 208000016886 cerebral arteriopathy with subcortical infarcts and leukoencephalopathy Diseases 0.000 description 1
- 210000003710 cerebral cortex Anatomy 0.000 description 1
- 208000001088 cerebrotendinous xanthomatosis Diseases 0.000 description 1
- WOWHHFRSBJGXCM-UHFFFAOYSA-M cetyltrimethylammonium chloride Chemical compound [Cl-].CCCCCCCCCCCCCCCC[N+](C)(C)C WOWHHFRSBJGXCM-UHFFFAOYSA-M 0.000 description 1
- 238000012512 characterization method Methods 0.000 description 1
- 210000003763 chloroplast Anatomy 0.000 description 1
- 230000004186 co-expression Effects 0.000 description 1
- 238000002742 combinatorial mutagenesis Methods 0.000 description 1
- 239000003184 complementary RNA Substances 0.000 description 1
- 201000006815 congenital muscular dystrophy Diseases 0.000 description 1
- 238000011109 contamination Methods 0.000 description 1
- 238000007796 conventional method Methods 0.000 description 1
- 210000000877 corpus callosum Anatomy 0.000 description 1
- 238000012937 correction Methods 0.000 description 1
- 239000013078 crystal Substances 0.000 description 1
- 101150064416 csp1 gene Proteins 0.000 description 1
- 210000000805 cytoplasm Anatomy 0.000 description 1
- 230000007123 defense Effects 0.000 description 1
- 230000007812 deficiency Effects 0.000 description 1
- 230000002950 deficient Effects 0.000 description 1
- 238000006731 degradation reaction Methods 0.000 description 1
- 238000002716 delivery method Methods 0.000 description 1
- 239000005547 deoxyribonucleotide Substances 0.000 description 1
- 125000002637 deoxyribonucleotide group Chemical group 0.000 description 1
- 238000011161 development Methods 0.000 description 1
- 230000018109 developmental process Effects 0.000 description 1
- 238000003745 diagnosis Methods 0.000 description 1
- 102000004419 dihydrofolate reductase Human genes 0.000 description 1
- 230000005750 disease progression Effects 0.000 description 1
- 206010013663 drug dependence Diseases 0.000 description 1
- 238000007877 drug screening Methods 0.000 description 1
- 241001493065 dsRNA viruses Species 0.000 description 1
- 230000008190 early vertebrate development Effects 0.000 description 1
- 239000012636 effector Substances 0.000 description 1
- 238000001962 electrophoresis Methods 0.000 description 1
- 230000008030 elimination Effects 0.000 description 1
- 238000003379 elimination reaction Methods 0.000 description 1
- 230000013020 embryo development Effects 0.000 description 1
- 238000001952 enzyme assay Methods 0.000 description 1
- 210000000981 epithelium Anatomy 0.000 description 1
- 238000004880 explosion Methods 0.000 description 1
- 239000013613 expression plasmid Substances 0.000 description 1
- 230000001605 fetal effect Effects 0.000 description 1
- 238000002795 fluorescence method Methods 0.000 description 1
- 108010021843 fluorescent protein 583 Proteins 0.000 description 1
- 231100000221 frame shift mutation induction Toxicity 0.000 description 1
- 201000008049 fucosidosis Diseases 0.000 description 1
- 101150045500 galK gene Proteins 0.000 description 1
- 238000003209 gene knockout Methods 0.000 description 1
- 238000007429 general method Methods 0.000 description 1
- 108020004445 glyceraldehyde-3-phosphate dehydrogenase Proteins 0.000 description 1
- 201000011205 glycine encephalopathy Diseases 0.000 description 1
- 230000013595 glycosylation Effects 0.000 description 1
- 238000006206 glycosylation reaction Methods 0.000 description 1
- PCHJSUWPFVWCPO-UHFFFAOYSA-N gold Chemical compound [Au] PCHJSUWPFVWCPO-UHFFFAOYSA-N 0.000 description 1
- 230000012010 growth Effects 0.000 description 1
- 230000009931 harmful effect Effects 0.000 description 1
- 238000010438 heat treatment Methods 0.000 description 1
- 208000014752 hemophagocytic syndrome Diseases 0.000 description 1
- 208000006454 hepatitis Diseases 0.000 description 1
- 231100000283 hepatitis Toxicity 0.000 description 1
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 description 1
- 102000057382 human EPHA3 Human genes 0.000 description 1
- 210000005119 human aortic smooth muscle cell Anatomy 0.000 description 1
- 239000005457 ice water Substances 0.000 description 1
- 238000003384 imaging method Methods 0.000 description 1
- 230000008076 immune mechanism Effects 0.000 description 1
- 230000007813 immunodeficiency Effects 0.000 description 1
- 102000018358 immunoglobulin Human genes 0.000 description 1
- 229940072221 immunoglobulins Drugs 0.000 description 1
- 238000003364 immunohistochemistry Methods 0.000 description 1
- 230000008676 import Effects 0.000 description 1
- 238000000126 in silico method Methods 0.000 description 1
- 208000010194 infantile onset spinocerebellar ataxia Diseases 0.000 description 1
- 206010022000 influenza Diseases 0.000 description 1
- 108700032552 influenza virus INS1 Proteins 0.000 description 1
- 230000005764 inhibitory process Effects 0.000 description 1
- 239000003999 initiator Substances 0.000 description 1
- 238000002347 injection Methods 0.000 description 1
- 239000007924 injection Substances 0.000 description 1
- 230000009545 invasion Effects 0.000 description 1
- 229910052742 iron Inorganic materials 0.000 description 1
- 238000005304 joining Methods 0.000 description 1
- 208000008106 junctional epidermolysis bullosa Diseases 0.000 description 1
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 description 1
- 101150044508 key gene Proteins 0.000 description 1
- 230000002147 killing effect Effects 0.000 description 1
- 238000011813 knockout mouse model Methods 0.000 description 1
- 238000011005 laboratory method Methods 0.000 description 1
- 238000012177 large-scale sequencing Methods 0.000 description 1
- 208000032839 leukemia Diseases 0.000 description 1
- IXHBTMCLRNMKHZ-LBPRGKRZSA-N levobunolol Chemical compound O=C1CCCC2=C1C=CC=C2OC[C@@H](O)CNC(C)(C)C IXHBTMCLRNMKHZ-LBPRGKRZSA-N 0.000 description 1
- 239000003446 ligand Substances 0.000 description 1
- 230000029226 lipidation Effects 0.000 description 1
- 208000014817 lissencephaly spectrum disease Diseases 0.000 description 1
- 239000012160 loading buffer Substances 0.000 description 1
- 230000004807 localization Effects 0.000 description 1
- 230000007774 longterm Effects 0.000 description 1
- 210000004698 lymphocyte Anatomy 0.000 description 1
- 235000018977 lysine Nutrition 0.000 description 1
- 150000002669 lysines Chemical class 0.000 description 1
- 239000012139 lysis buffer Substances 0.000 description 1
- 210000000415 mammalian chromosome Anatomy 0.000 description 1
- 210000001161 mammalian embryo Anatomy 0.000 description 1
- 210000005075 mammary gland Anatomy 0.000 description 1
- 208000024393 maple syrup urine disease Diseases 0.000 description 1
- 239000000463 material Substances 0.000 description 1
- 238000005259 measurement Methods 0.000 description 1
- 230000010534 mechanism of action Effects 0.000 description 1
- 238000012737 microarray-based gene expression Methods 0.000 description 1
- 230000000813 microbial effect Effects 0.000 description 1
- 244000005700 microbiome Species 0.000 description 1
- 238000000520 microinjection Methods 0.000 description 1
- 230000003278 mimic effect Effects 0.000 description 1
- 210000003470 mitochondria Anatomy 0.000 description 1
- 201000011540 mitochondrial DNA depletion syndrome 4a Diseases 0.000 description 1
- 201000011561 mitochondrial DNA depletion syndrome 7 Diseases 0.000 description 1
- 238000003032 molecular docking Methods 0.000 description 1
- 230000004001 molecular interaction Effects 0.000 description 1
- 230000009456 molecular mechanism Effects 0.000 description 1
- 208000020460 mucolipidosis II alpha/beta Diseases 0.000 description 1
- 201000002273 mucopolysaccharidosis II Diseases 0.000 description 1
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 description 1
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 description 1
- 238000012243 multiplex automated genomic engineering Methods 0.000 description 1
- 101150034514 murC gene Proteins 0.000 description 1
- 210000003205 muscle Anatomy 0.000 description 1
- 208000025855 muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A, 4 Diseases 0.000 description 1
- 231100000707 mutagenic chemical Toxicity 0.000 description 1
- 230000001537 neural effect Effects 0.000 description 1
- 230000000626 neurodegenerative effect Effects 0.000 description 1
- 230000007472 neurodevelopment Effects 0.000 description 1
- 210000005044 neurofilament Anatomy 0.000 description 1
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 description 1
- 230000007935 neutral effect Effects 0.000 description 1
- 201000006352 oculocerebrorenal syndrome Diseases 0.000 description 1
- 230000009437 off-target effect Effects 0.000 description 1
- 229940046166 oligodeoxynucleotide Drugs 0.000 description 1
- 230000003287 optical effect Effects 0.000 description 1
- 210000000056 organ Anatomy 0.000 description 1
- 210000003463 organelle Anatomy 0.000 description 1
- 201000008152 organic acidemia Diseases 0.000 description 1
- 230000008520 organization Effects 0.000 description 1
- 102000045222 parkin Human genes 0.000 description 1
- 238000005192 partition Methods 0.000 description 1
- 239000000137 peptide hydrolase inhibitor Substances 0.000 description 1
- 238000010647 peptide synthesis reaction Methods 0.000 description 1
- 239000000816 peptidomimetic Chemical class 0.000 description 1
- 230000002093 peripheral effect Effects 0.000 description 1
- 208000023269 peroxisome biogenesis disease Diseases 0.000 description 1
- 210000003800 pharynx Anatomy 0.000 description 1
- 238000003976 plant breeding Methods 0.000 description 1
- 230000008488 polyadenylation Effects 0.000 description 1
- 238000006116 polymerization reaction Methods 0.000 description 1
- 229920002451 polyvinyl alcohol Polymers 0.000 description 1
- 230000001124 posttranscriptional effect Effects 0.000 description 1
- 238000003825 pressing Methods 0.000 description 1
- 238000004393 prognosis Methods 0.000 description 1
- 230000000750 progressive effect Effects 0.000 description 1
- 201000001204 progressive myoclonus epilepsy Diseases 0.000 description 1
- 230000000644 propagated effect Effects 0.000 description 1
- 210000002307 prostate Anatomy 0.000 description 1
- 238000002331 protein detection Methods 0.000 description 1
- 239000012474 protein marker Substances 0.000 description 1
- 230000012743 protein tagging Effects 0.000 description 1
- 230000006337 proteolytic cleavage Effects 0.000 description 1
- 239000012264 purified product Substances 0.000 description 1
- 238000012797 qualification Methods 0.000 description 1
- 239000002096 quantum dot Substances 0.000 description 1
- 230000008263 repair mechanism Effects 0.000 description 1
- 230000029058 respiratory gaseous exchange Effects 0.000 description 1
- 108091008146 restriction endonucleases Proteins 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
- 108091092562 ribozyme Proteins 0.000 description 1
- 101150024074 rub1 gene Proteins 0.000 description 1
- 238000005070 sampling Methods 0.000 description 1
- 230000037390 scarring Effects 0.000 description 1
- 238000000926 separation method Methods 0.000 description 1
- 238000012772 sequence design Methods 0.000 description 1
- 238000013207 serial dilution Methods 0.000 description 1
- 229940076279 serotonin Drugs 0.000 description 1
- 230000035939 shock Effects 0.000 description 1
- 230000003007 single stranded DNA break Effects 0.000 description 1
- 230000005783 single-strand break Effects 0.000 description 1
- 238000002741 site-directed mutagenesis Methods 0.000 description 1
- 210000003491 skin Anatomy 0.000 description 1
- 239000002002 slurry Substances 0.000 description 1
- 210000002460 smooth muscle Anatomy 0.000 description 1
- 229910000030 sodium bicarbonate Inorganic materials 0.000 description 1
- 235000017557 sodium bicarbonate Nutrition 0.000 description 1
- 229910000029 sodium carbonate Inorganic materials 0.000 description 1
- 238000002415 sodium dodecyl sulfate polyacrylamide gel electrophoresis Methods 0.000 description 1
- 108090000250 sortase A Proteins 0.000 description 1
- 238000001228 spectrum Methods 0.000 description 1
- 208000002320 spinal muscular atrophy Diseases 0.000 description 1
- 230000006641 stabilisation Effects 0.000 description 1
- 238000011105 stabilization Methods 0.000 description 1
- 238000007619 statistical method Methods 0.000 description 1
- 238000013179 statistical model Methods 0.000 description 1
- 239000003270 steroid hormone Substances 0.000 description 1
- 238000003860 storage Methods 0.000 description 1
- 208000011117 substance-related disease Diseases 0.000 description 1
- 239000000758 substrate Substances 0.000 description 1
- 239000013589 supplement Substances 0.000 description 1
- 230000005062 synaptic transmission Effects 0.000 description 1
- 208000011580 syndromic disease Diseases 0.000 description 1
- 238000003786 synthesis reaction Methods 0.000 description 1
- 238000010809 targeting technique Methods 0.000 description 1
- 208000016094 thanatophoric dysplasia type 1 Diseases 0.000 description 1
- 229940124597 therapeutic agent Drugs 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
- 229940094937 thioredoxin Drugs 0.000 description 1
- 229960004072 thrombin Drugs 0.000 description 1
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical class CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 description 1
- 230000036962 time dependent Effects 0.000 description 1
- 231100000419 toxicity Toxicity 0.000 description 1
- 230000001988 toxicity Effects 0.000 description 1
- 230000002463 transducing effect Effects 0.000 description 1
- 238000003151 transfection method Methods 0.000 description 1
- 238000000844 transformation Methods 0.000 description 1
- 238000011426 transformation method Methods 0.000 description 1
- 230000010474 transient expression Effects 0.000 description 1
- 238000003146 transient transfection Methods 0.000 description 1
- 230000005945 translocation Effects 0.000 description 1
- 230000032258 transport Effects 0.000 description 1
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 description 1
- 230000010415 tropism Effects 0.000 description 1
- 230000005760 tumorsuppression Effects 0.000 description 1
- 108010032276 tyrosyl-glutamyl-tyrosyl-glutamic acid Proteins 0.000 description 1
- 208000030954 urea cycle disease Diseases 0.000 description 1
- 239000003981 vehicle Substances 0.000 description 1
- 210000003501 vero cell Anatomy 0.000 description 1
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 1
- 238000001262 western blot Methods 0.000 description 1
- 239000012130 whole-cell lysate Substances 0.000 description 1
- 210000005253 yeast cell Anatomy 0.000 description 1
- 239000012138 yeast extract Substances 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
- C12N15/74—Vectors or expression systems specially adapted for prokaryotic hosts other than E. coli, e.g. Lactobacillus, Micromonospora
- C12N15/746—Vectors or expression systems specially adapted for prokaryotic hosts other than E. coli, e.g. Lactobacillus, Micromonospora for lactic acid bacteria (Streptococcus; Lactococcus; Lactobacillus; Pediococcus; Enterococcus; Leuconostoc; Propionibacterium; Bifidobacterium; Sporolactobacillus)
-
- 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
- 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
- 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
- C12N15/63—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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/70—Vectors or expression systems specially adapted for E. coli
-
- 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
- C12N15/74—Vectors or expression systems specially adapted for prokaryotic hosts other than E. coli, e.g. Lactobacillus, Micromonospora
-
- 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
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
-
- 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
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
-
- 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
-
- G—PHYSICS
- G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
- G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
- G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
-
- G—PHYSICS
- G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
- G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
- G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
- G16B20/20—Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection
-
- G—PHYSICS
- G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
- G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
- G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
- G16B20/30—Detection of binding sites or motifs
-
- G—PHYSICS
- G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
- G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
- G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
- G16B20/50—Mutagenesis
-
- G—PHYSICS
- G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
- G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
- G16B30/00—ICT specially adapted for sequence analysis involving nucleotides or amino acids
- G16B30/10—Sequence alignment; Homology search
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
-
- 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
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2800/10—Plasmid DNA
- C12N2800/101—Plasmid DNA for bacteria
-
- G—PHYSICS
- G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
- G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
- G16B30/00—ICT specially adapted for sequence analysis involving nucleotides or amino acids
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Physics & Mathematics (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Bioinformatics & Computational Biology (AREA)
- Analytical Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Theoretical Computer Science (AREA)
- Evolutionary Biology (AREA)
- Medical Informatics (AREA)
- Spectroscopy & Molecular Physics (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Crystallography & Structural Chemistry (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
- Virology (AREA)
Applications Claiming Priority (12)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261736527P | 2012-12-12 | 2012-12-12 | |
| US61/736,527 | 2012-12-12 | ||
| US201361748427P | 2013-01-02 | 2013-01-02 | |
| US61/748,427 | 2013-01-02 | ||
| US201361757972P | 2013-01-29 | 2013-01-29 | |
| US201361768959P | 2013-02-25 | 2013-02-25 | |
| US61/768,959 | 2013-02-25 | ||
| US201361791409P | 2013-03-15 | 2013-03-15 | |
| US61/791,409 | 2013-03-15 | ||
| US201361835931P | 2013-06-17 | 2013-06-17 | |
| US61/835,931 | 2013-06-17 | ||
| PCT/US2013/074611 WO2014093595A1 (en) | 2012-12-12 | 2013-12-12 | Crispr-cas component systems, methods and compositions for sequence manipulation |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR122021008308B1 true BR122021008308B1 (pt) | 2022-12-27 |
Family
ID=49881125
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR122021008308-8A BR122021008308B1 (pt) | 2012-12-12 | 2013-12-12 | Sistemas de componente crispr-cas, métodos e composições para manipulação de sequência |
| BR112015013786-5A BR112015013786B1 (pt) | 2012-12-12 | 2013-12-12 | Sistemas de componente crispr-cas, métodos e composições para manipulação de sequência |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112015013786-5A BR112015013786B1 (pt) | 2012-12-12 | 2013-12-12 | Sistemas de componente crispr-cas, métodos e composições para manipulação de sequência |
Country Status (9)
| Country | Link |
|---|---|
| US (27) | US20140189896A1 (https=) |
| EP (9) | EP2840140B2 (https=) |
| JP (5) | JP2016505256A (https=) |
| CN (3) | CN110982844B (https=) |
| AU (4) | AU2013359262C1 (https=) |
| BR (2) | BR122021008308B1 (https=) |
| CA (1) | CA2894668A1 (https=) |
| DK (1) | DK3252160T3 (https=) |
| WO (1) | WO2014093595A1 (https=) |
Families Citing this family (943)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2449112A1 (en) | 2009-07-01 | 2012-05-09 | Transposagen Biopharmaceuticals, Inc. | Genetically modified rat models for severe combined immunodeficiency (scid) |
| US9719068B2 (en) | 2010-05-06 | 2017-08-01 | Children's Hospital Medical Center | Methods and systems for converting precursor cells into intestinal tissues through directed differentiation |
| US9528124B2 (en) | 2013-08-27 | 2016-12-27 | Recombinetics, Inc. | Efficient non-meiotic allele introgression |
| US10920242B2 (en) | 2011-02-25 | 2021-02-16 | Recombinetics, Inc. | Non-meiotic allele introgression |
| CA2831144A1 (en) | 2011-03-23 | 2012-09-27 | Pioneer Hi-Bred International, Inc. | Methods for producing a complex transgenic trait locus |
| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| EP3604555B1 (en) | 2011-10-14 | 2024-12-25 | President and Fellows of Harvard College | Sequencing by structure assembly |
| ES2991004T3 (es) | 2011-12-22 | 2024-12-02 | Harvard College | Métodos para la detección de analitos |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| WO2013119602A1 (en) | 2012-02-06 | 2013-08-15 | President And Fellows Of Harvard College | Arrdc1-mediated microvesicles (armms) and uses thereof |
| US9637739B2 (en) | 2012-03-20 | 2017-05-02 | Vilnius University | RNA-directed DNA cleavage by the Cas9-crRNA complex |
| SG11201406547YA (en) | 2012-04-25 | 2014-11-27 | Regeneron Pharma | Nuclease-mediated targeting with large targeting vectors |
| WO2013163628A2 (en) | 2012-04-27 | 2013-10-31 | Duke University | Genetic correction of mutated genes |
| AU2013266968B2 (en) | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| US20150225734A1 (en) | 2012-06-19 | 2015-08-13 | Regents Of The University Of Minnesota | Gene targeting in plants using dna viruses |
| US10648001B2 (en) | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| EP3196301B1 (en) | 2012-07-11 | 2018-10-17 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of monogenic diseases |
| AU2013293270B2 (en) * | 2012-07-25 | 2018-08-16 | Massachusetts Institute Of Technology | Inducible DNA binding proteins and genome perturbation tools and applications thereof |
| AU2013335451C1 (en) * | 2012-10-23 | 2024-07-04 | Toolgen Incorporated | Composition for cleaving a target DNA comprising a guide RNA specific for the target DNA and Cas protein-encoding nucleic acid or Cas protein, and use thereof |
| WO2014071182A1 (en) | 2012-11-01 | 2014-05-08 | Massachusetts Institute Of Technology | Directed evolution of synthetic gene cluster |
| PL3360964T3 (pl) | 2012-12-06 | 2020-03-31 | Sigma-Aldrich Co. Llc | Modyfikacja i regulacja genomu oparta na crispr |
| US20140186843A1 (en) | 2012-12-12 | 2014-07-03 | Massachusetts Institute Of Technology | Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| 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 |
| CN113355357B (zh) | 2012-12-12 | 2024-12-03 | 布罗德研究所有限公司 | 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化 |
| EP2840140B2 (en) | 2012-12-12 | 2023-02-22 | The Broad Institute, Inc. | Crispr-Cas based method for mutation of prokaryotic cells |
| WO2014093655A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| WO2014093709A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| EP3031921B1 (en) | 2012-12-12 | 2025-03-12 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| DK2931891T3 (da) * | 2012-12-17 | 2019-08-19 | Harvard College | Rna-styret modificering af menneskelige genomer |
| CA2898184A1 (en) | 2013-01-16 | 2014-07-24 | Emory University | Cas9-nucleic acid complexes and uses related thereto |
| US10660943B2 (en) | 2013-02-07 | 2020-05-26 | The Rockefeller University | Sequence specific antimicrobials |
| US11135273B2 (en) | 2013-02-07 | 2021-10-05 | The Rockefeller University | Sequence specific antimicrobials |
| EP2971184B1 (en) | 2013-03-12 | 2019-04-17 | President and Fellows of Harvard College | Method of generating a three-dimensional nucleic acid containing matrix |
| RU2662932C2 (ru) | 2013-03-14 | 2018-07-31 | Карибо Биосайенсиз, Инк. | Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты |
| WO2014204578A1 (en) | 2013-06-21 | 2014-12-24 | The General Hospital Corporation | Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing |
| CN112301024A (zh) | 2013-03-15 | 2021-02-02 | 通用医疗公司 | 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性 |
| US20140364333A1 (en) * | 2013-03-15 | 2014-12-11 | President And Fellows Of Harvard College | Methods for Live Imaging of Cells |
| US9234213B2 (en) | 2013-03-15 | 2016-01-12 | System Biosciences, Llc | Compositions and methods directed to CRISPR/Cas genomic engineering systems |
| US10760064B2 (en) | 2013-03-15 | 2020-09-01 | The General Hospital Corporation | RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| EP4286517A3 (en) | 2013-04-04 | 2024-03-13 | President and Fellows of Harvard College | Therapeutic uses of genome editing with crispr/cas systems |
| DK3456831T3 (da) | 2013-04-16 | 2021-09-06 | Regeneron Pharma | Målrettet modifikation af rottegenom |
| US9873907B2 (en) | 2013-05-29 | 2018-01-23 | Agilent Technologies, Inc. | Method for fragmenting genomic DNA using CAS9 |
| AU2014273082B2 (en) * | 2013-05-29 | 2018-11-08 | Cellectis | A method for producing precise DNA cleavage using Cas9 nickase activity |
| US9267135B2 (en) | 2013-06-04 | 2016-02-23 | President And Fellows Of Harvard College | RNA-guided transcriptional regulation |
| SG10201913068PA (en) * | 2013-06-04 | 2020-02-27 | Harvard College | Rna-guided transcriptional regulation |
| WO2014197748A2 (en) * | 2013-06-05 | 2014-12-11 | Duke University | Rna-guided gene editing and gene regulation |
| US9982277B2 (en) | 2013-06-11 | 2018-05-29 | The Regents Of The University Of California | Methods and compositions for target DNA modification |
| 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 |
| MX374532B (es) * | 2013-06-17 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos. |
| RU2716420C2 (ru) | 2013-06-17 | 2020-03-11 | Те Брод Инститьют Инк. | Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени |
| WO2014204724A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| EP3019595A4 (en) | 2013-07-09 | 2016-11-30 | THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS | |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | 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 |
| CN120574876A (zh) | 2013-08-22 | 2025-09-02 | 纳幕尔杜邦公司 | 使用向导rna/cas内切核酸酶系统的植物基因组修饰及其使用方法 |
| EP4074330A1 (en) * | 2013-09-05 | 2022-10-19 | Massachusetts Institute of Technology | Tuning microbial populations with programmable nucleases |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| EP3988649B1 (en) * | 2013-09-18 | 2024-11-27 | Kymab Limited | Methods, cells and organisms |
| WO2015054507A1 (en) | 2013-10-10 | 2015-04-16 | Pronutria, Inc. | Nutritive polypeptide production systems, and methods of manufacture and use thereof |
| WO2015065964A1 (en) | 2013-10-28 | 2015-05-07 | The Broad Institute Inc. | Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof |
| CN106459995B (zh) | 2013-11-07 | 2020-02-21 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
| US11326209B2 (en) | 2013-11-07 | 2022-05-10 | Massachusetts Institute Of Technology | Cell-based genomic recorded accumulative memory |
| KR102170502B1 (ko) | 2013-12-11 | 2020-10-28 | 리제너론 파마슈티칼스 인코포레이티드 | 게놈의 표적화된 변형을 위한 방법 및 조성물 |
| EP3470089A1 (en) | 2013-12-12 | 2019-04-17 | 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 |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| EP3835419A1 (en) | 2013-12-12 | 2021-06-16 | The Regents of The University of California | Methods and compositions for modifying a single stranded target nucleic acid |
| JP7103750B2 (ja) | 2013-12-12 | 2022-07-20 | ザ・ブロード・インスティテュート・インコーポレイテッド | ゲノム編集のためのCRISPR-Cas系及び組成物の送達、使用及び治療適用 |
| WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
| JP6712948B2 (ja) | 2013-12-12 | 2020-06-24 | ザ・ブロード・インスティテュート・インコーポレイテッド | 組成物、及びヌクレオチドリピート障害におけるcrispr−cas系の使用方法 |
| JP6793547B2 (ja) | 2013-12-12 | 2020-12-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 最適化機能CRISPR−Cas系による配列操作のための系、方法および組成物 |
| SG10201804975PA (en) | 2013-12-12 | 2018-07-30 | Broad Inst Inc | Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for HBV and Viral Diseases and Disorders |
| 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 |
| ES2918501T3 (es) | 2013-12-19 | 2022-07-18 | Novartis Ag | Receptores de antígenos quiméricos de mesotelina humana y usos de los mismos |
| EP3089989B1 (en) * | 2013-12-31 | 2020-06-24 | The Regents of The University of California | Cas9 crystals and methods of use thereof |
| EP3690044B1 (en) | 2014-02-11 | 2024-01-10 | The Regents of the University of Colorado, a body corporate | Crispr enabled multiplexed genome engineering |
| US10286084B2 (en) | 2014-02-18 | 2019-05-14 | Duke University | Compositions for the inactivation of virus replication and methods of making and using the same |
| EP3110971B1 (en) | 2014-02-27 | 2019-05-08 | The Broad Institute Inc. | T cell balance gene expression and methods of use thereof |
| ES2898460T3 (es) | 2014-02-27 | 2022-03-07 | Monsanto Technology Llc | Composiciones y procedimientos para la modificación genómica dirigida al sitio |
| US11028388B2 (en) | 2014-03-05 | 2021-06-08 | Editas Medicine, Inc. | CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa |
| EP3115457B1 (en) | 2014-03-05 | 2019-10-02 | National University Corporation Kobe University | Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same |
| 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 |
| ES2745769T3 (es) | 2014-03-10 | 2020-03-03 | Editas Medicine Inc | Procedimientos y composiciones relacionados con CRISPR/CAS para tratar la amaurosis congénita de Leber 10 (LCA10) |
| US20170335281A1 (en) | 2014-03-15 | 2017-11-23 | Novartis Ag | Treatment of cancer using chimeric antigen receptor |
| EP3122880B1 (en) | 2014-03-26 | 2021-05-05 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
| MX375361B (es) | 2014-03-26 | 2025-03-06 | Univ Maryland | Edicion de genoma dirigida en cigotos de animales grandes domesticos. |
| EP3540061A1 (en) * | 2014-04-02 | 2019-09-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| AU2015244039B2 (en) | 2014-04-07 | 2021-10-21 | Novartis Ag | Treatment of cancer using anti-CD19 chimeric antigen receptor |
| CN106536739B (zh) * | 2014-04-14 | 2021-08-03 | 内梅西斯生物有限公司 | 治疗剂 |
| GB201406968D0 (en) | 2014-04-17 | 2014-06-04 | Green Biologics Ltd | Deletion mutants |
| GB201406970D0 (en) * | 2014-04-17 | 2014-06-04 | Green Biologics Ltd | Targeted mutations |
| BR112016024945A2 (pt) | 2014-04-28 | 2017-10-24 | Recombinetics Inc | edição de gene multiplex em suínos |
| EP3140403A4 (en) | 2014-05-09 | 2017-12-20 | Université Laval | Prevention and treatment of alzheimer's disease by genome editing using the crispr/cas system |
| PL3145934T3 (pl) | 2014-05-19 | 2021-08-16 | Pfizer Inc. | Podstawione związki 6,8-dioksabicyklo[3.2.1]oktano-2,3-diolu jako środki kierujące do ASGPR |
| SG10201801654RA (en) | 2014-05-28 | 2018-04-27 | Childrens Hospital Med Ct | Methods and systems for converting precursor cells into gastric tissues through directed differentiation |
| HRP20200529T1 (hr) * | 2014-06-06 | 2020-09-04 | Regeneron Pharmaceuticals, Inc. | Postupci i pripravci za modificiranje ciljnog lokusa |
| CN106536056B (zh) | 2014-06-13 | 2021-07-16 | 儿童医学中心公司 | 分离线粒体的产品和方法 |
| CN106604994B (zh) | 2014-06-23 | 2021-12-14 | 通用医疗公司 | 通过测序评估的DSBs的全基因组无偏鉴定(GUIDE-Seq) |
| 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 |
| WO2016007948A1 (en) * | 2014-07-11 | 2016-01-14 | Pioneer Hi-Bred International, Inc. | Agronomic trait modification using guide rna/cas endonuclease systems and methods of use |
| US10676754B2 (en) | 2014-07-11 | 2020-06-09 | E I Du Pont De Nemours And Company | Compositions and methods for producing plants resistant to glyphosate herbicide |
| EP3169310A1 (en) | 2014-07-15 | 2017-05-24 | Life Technologies Corporation | Compositions with lipid aggregates and methods for efficient delivery of molecules to cells |
| US11542488B2 (en) | 2014-07-21 | 2023-01-03 | Novartis Ag | Sortase synthesized chimeric antigen receptors |
| TWI719942B (zh) | 2014-07-21 | 2021-03-01 | 瑞士商諾華公司 | 使用cd33嵌合抗原受體治療癌症 |
| JP2017528433A (ja) | 2014-07-21 | 2017-09-28 | ノバルティス アーゲー | 低い免疫増強用量のmTOR阻害剤とCARの組み合わせ |
| BR112017001183A2 (pt) | 2014-07-21 | 2017-11-28 | Novartis Ag | tratamento de câncer usando receptor de antígeno quimérico anti-bcma humanizado |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| EP4205749A1 (en) | 2014-07-31 | 2023-07-05 | Novartis AG | Subset-optimized chimeric antigen receptor-containing cells |
| US10513711B2 (en) | 2014-08-13 | 2019-12-24 | Dupont Us Holding, Llc | Genetic targeting in non-conventional yeast using an RNA-guided endonuclease |
| AU2015301460B2 (en) | 2014-08-14 | 2021-04-08 | Novartis Ag | Treatment of cancer using GFR alpha-4 chimeric antigen receptor |
| WO2016028682A1 (en) | 2014-08-17 | 2016-02-25 | The Broad Institute Inc. | Genome editing using cas9 nickases |
| MX2017002205A (es) | 2014-08-19 | 2017-08-21 | Novartis Ag | Receptor quimerico de antigeno (car) anti-cd123 para uso en el tratamiento de cancer. |
| SG11201701245QA (en) * | 2014-08-27 | 2017-03-30 | Caribou Biosciences Inc | Methods for increasing cas9-mediated engineering efficiency |
| EP3186375A4 (en) | 2014-08-28 | 2019-03-13 | North Carolina State University | Novel CAS9 PROTEINS AND CHARACTERISTICS FOR DNA TARGETING AND GENOME EDITING |
| EP3188763B1 (en) * | 2014-09-02 | 2020-05-13 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification |
| MX2017002930A (es) | 2014-09-12 | 2017-06-06 | Du Pont | Generacion de sitios de integracion especifica de sitio para loci de rasgos complejos en maiz y soja, y metodos de uso. |
| JP6839074B2 (ja) | 2014-09-17 | 2021-03-03 | ノバルティス アーゲー | 養子免疫療法のためのキメラ受容体での細胞毒性細胞のターゲティング |
| US9616114B1 (en) | 2014-09-18 | 2017-04-11 | David Gordon Bermudes | Modified bacteria having improved pharmacokinetics and tumor colonization enhancing antitumor activity |
| WO2016049182A1 (en) | 2014-09-23 | 2016-03-31 | Aggentics, Inc. | Materials and methods for producing animals with short hair |
| WO2016049024A2 (en) | 2014-09-24 | 2016-03-31 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo |
| 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 |
| 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 |
| WO2016049258A2 (en) | 2014-09-25 | 2016-03-31 | The Broad Institute Inc. | Functional screening with optimized functional crispr-cas systems |
| US10040048B1 (en) | 2014-09-25 | 2018-08-07 | Synthego Corporation | Automated modular system and method for production of biopolymers |
| AU2015330699B2 (en) | 2014-10-10 | 2021-12-02 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| WO2016061073A1 (en) * | 2014-10-14 | 2016-04-21 | Memorial Sloan-Kettering Cancer Center | Composition and method for in vivo engineering of chromosomal rearrangements |
| CN105602935B (zh) * | 2014-10-20 | 2020-11-13 | 聂凌云 | 一种新型线粒体基因组编辑工具 |
| GB201418965D0 (https=) | 2014-10-24 | 2014-12-10 | Ospedale San Raffaele And Fond Telethon | |
| EP3212788A2 (en) | 2014-10-27 | 2017-09-06 | The Broad Institute, Inc. | Compositions, methods and use of synthetic lethal screening |
| US9816080B2 (en) | 2014-10-31 | 2017-11-14 | President And Fellows Of Harvard College | Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs) |
| EP3215623A4 (en) | 2014-11-06 | 2018-09-26 | President and Fellows of Harvard College | Cells lacking b2m surface expression and methods for allogeneic administration of such cells |
| JP6823593B2 (ja) | 2014-11-06 | 2021-02-03 | イー・アイ・デュポン・ドウ・ヌムール・アンド・カンパニーE.I.Du Pont De Nemours And Company | Rna誘導型エンドヌクレアーゼの細胞へのペプチド媒介輸送 |
| EP4464338A3 (en) | 2014-11-07 | 2025-02-12 | Editas Medicine, Inc. | Systems for improving crispr/cas-mediated genome-editing |
| KR102531016B1 (ko) | 2014-11-21 | 2023-05-10 | 리제너론 파마슈티칼스 인코포레이티드 | 쌍 형성된 가이드 rna를 사용하는 표적화된 유전자 변형을 위한 방법 및 조성물 |
| EP3224381B1 (en) | 2014-11-25 | 2019-09-04 | The Brigham and Women's Hospital, Inc. | Method of identifying a person having a predisposition to or afflicted with a cardiometabolic disease |
| CA2969145A1 (en) | 2014-11-26 | 2016-06-02 | The Regents Of The University Of California | Therapeutic compositions comprising transcription factors and methods of making and using the same |
| US20180105834A1 (en) * | 2014-11-27 | 2018-04-19 | Institute Of Animal Sciences, Chinese Academy Of Agrigultural Sciences | A method of site-directed insertion to h11 locus in pigs by using site-directed cutting system |
| GB201421096D0 (en) | 2014-11-27 | 2015-01-14 | Imp Innovations Ltd | Genome editing methods |
| CA2969619A1 (en) | 2014-12-03 | 2016-06-09 | Agilent Technologies, Inc. | Guide rna with chemical modifications |
| CN116059378A (zh) | 2014-12-10 | 2023-05-05 | 明尼苏达大学董事会 | 用于治疗疾病的遗传修饰的细胞、组织和器官 |
| 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) |
| WO2016094874A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Escorted and functionalized guides for crispr-cas systems |
| WO2016094872A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Dead guides for crispr transcription factors |
| WO2016100333A1 (en) | 2014-12-15 | 2016-06-23 | Syngenta Participations Ag | Pesticidal microrna carriers and use thereof |
| FI3234150T3 (fi) * | 2014-12-16 | 2025-11-05 | Danisco Us Inc | Sienigenomin muokkausjärjestelmät ja niiden käyttömenetelmät |
| KR102424626B1 (ko) * | 2014-12-17 | 2022-07-25 | 이 아이 듀폰 디 네모아 앤드 캄파니 | 원형 폴리뉴클레오티드 변형 주형과 함께 가이드 RNA/Cas 엔도뉴클레아제 시스템을 이용하여 대장균에서 효율적으로 유전자 편집을 하기 위한 조성물 및 방법 |
| WO2016098078A2 (en) | 2014-12-19 | 2016-06-23 | Novartis Ag | Dimerization switches and uses thereof |
| 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 |
| US10190106B2 (en) | 2014-12-22 | 2019-01-29 | Univesity Of Massachusetts | Cas9-DNA targeting unit chimeras |
| WO2016106236A1 (en) | 2014-12-23 | 2016-06-30 | The Broad Institute Inc. | Rna-targeting system |
| WO2016106121A1 (en) | 2014-12-23 | 2016-06-30 | Syngenta Participations Ag | Methods and compositions for identifying and enriching for cells comprising site specific genomic modifications |
| CA2970370A1 (en) | 2014-12-24 | 2016-06-30 | Massachusetts Institute Of Technology | 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 |
| JP2018500037A (ja) | 2014-12-31 | 2018-01-11 | シンセティック ジェノミクス インコーポレーテッド | 高効率なインビボゲノム編集のための組成物及び方法 |
| EP3245232B1 (en) | 2015-01-12 | 2021-04-21 | The Regents of The University of California | Heterodimeric cas9 and methods of use thereof |
| EP3250689B1 (en) | 2015-01-28 | 2020-11-04 | The Regents of The University of California | Methods and compositions for labeling a single-stranded target nucleic acid |
| US9650617B2 (en) | 2015-01-28 | 2017-05-16 | Pioneer Hi-Bred International. Inc. | CRISPR hybrid DNA/RNA polynucleotides and methods of use |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| WO2016138488A2 (en) | 2015-02-26 | 2016-09-01 | 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 |
| KR20230156800A (ko) | 2015-03-03 | 2023-11-14 | 더 제너럴 하스피탈 코포레이션 | 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제 |
| CN104673816A (zh) * | 2015-03-05 | 2015-06-03 | 广东医学院 | 一种pCr-NHEJ载体及其构建方法及其用于细菌基因定点敲除的应用 |
| BR112017017260A2 (pt) | 2015-03-27 | 2018-04-17 | Du Pont | construções de dna, vetor, célula, plantas, semente, método de expressão de rna e método de modificação de local alvo |
| KR102888521B1 (ko) | 2015-04-06 | 2025-11-19 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna |
| WO2016168594A1 (en) * | 2015-04-16 | 2016-10-20 | President And Fellows Of Harvard College | Sensor systems for target ligands and uses thereof |
| GB201506509D0 (en) | 2015-04-16 | 2015-06-03 | Univ Wageningen | Nuclease-mediated genome editing |
| EP3286211A1 (en) | 2015-04-23 | 2018-02-28 | Novartis AG | Treatment of cancer using chimeric antigen receptor and protein kinase a blocker |
| 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 |
| WO2016176617A2 (en) | 2015-04-29 | 2016-11-03 | New York University | Method for treating high-grade gliomas |
| CN106191040B (zh) * | 2015-04-30 | 2021-09-14 | 杭州菁因康生物科技有限公司 | 基因打靶方法 |
| EP3711488A1 (en) | 2015-05-06 | 2020-09-23 | Snipr Technologies Limited | Altering microbial populations & modifying microbiota |
| CN107921148A (zh) | 2015-05-08 | 2018-04-17 | 哈佛学院校长同事会 | 通用供体干细胞和相关方法 |
| 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 |
| US10676723B2 (en) | 2015-05-11 | 2020-06-09 | David Gordon Bermudes | Chimeric protein toxins for expression by therapeutic bacteria |
| CA2986310A1 (en) | 2015-05-11 | 2016-11-17 | Editas Medicine, Inc. | Optimized crispr/cas9 systems and methods for gene editing in stem cells |
| US20180291372A1 (en) * | 2015-05-14 | 2018-10-11 | Massachusetts Institute Of Technology | Self-targeting genome editing system |
| AU2016263026A1 (en) | 2015-05-15 | 2017-11-09 | Pioneer Hi-Bred International, Inc. | Guide RNA/Cas endonuclease systems |
| WO2016187904A1 (zh) * | 2015-05-22 | 2016-12-01 | 深圳市第二人民医院 | CRISPR-Cas9特异性敲除猪CMAH基因的方法及用于特异性靶向CMAH基因的sgRNA |
| WO2016191684A1 (en) * | 2015-05-28 | 2016-12-01 | Finer Mitchell H | Genome editing vectors |
| EA201792663A1 (ru) * | 2015-05-29 | 2018-04-30 | Норт Каролина Стейт Юниверсити | Способы скрининга бактерий, архей, водорослей и дрожжей с использованием нуклеиновых кислот crispr |
| CN108025188A (zh) | 2015-06-01 | 2018-05-11 | 天普大学-联邦高等教育系统 | 用于hiv感染的rna导向治疗的方法和组合物 |
| EP3303585A4 (en) | 2015-06-03 | 2018-10-31 | Board of Regents of the University of Nebraska | Dna editing using single-stranded dna |
| WO2016196655A1 (en) | 2015-06-03 | 2016-12-08 | The Regents Of The University Of California | Cas9 variants and methods of use thereof |
| EP3302525A2 (en) | 2015-06-05 | 2018-04-11 | Novartis AG | Methods and compositions for diagnosing, treating, and monitoring treatment of shank3 deficiency associated disorders |
| CN108026526B (zh) | 2015-06-09 | 2023-05-12 | 爱迪塔斯医药公司 | 用于改善移植的crispr/cas相关方法和组合物 |
| WO2016198500A1 (en) * | 2015-06-10 | 2016-12-15 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and compositions for rna-guided treatment of human cytomegalovirus (hcmv) infection |
| EP3307890A1 (en) | 2015-06-10 | 2018-04-18 | Board of Regents, The University of Texas System | Use of exosomes for the treatment of disease |
| EP3307888A1 (en) * | 2015-06-12 | 2018-04-18 | Erasmus University Medical Center Rotterdam | New crispr assays |
| WO2016205276A1 (en) | 2015-06-15 | 2016-12-22 | North Carolina State University | Methods and compositions for efficient delivery of nucleic acids and rna-based antimicrobials |
| JP2018518182A (ja) | 2015-06-17 | 2018-07-12 | ザ ユーエービー リサーチ ファンデーション | ゲノム編集のためのcrispr/cas9複合体 |
| JP2018518181A (ja) | 2015-06-17 | 2018-07-12 | ザ ユーエービー リサーチ ファンデーション | 血液細胞系列の細胞に機能的ポリペプチドを導入するためのCRISPR/Cas9複合体 |
| WO2016205728A1 (en) | 2015-06-17 | 2016-12-22 | Massachusetts Institute Of Technology | Crispr mediated recording of cellular events |
| 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 |
| TWI906646B (zh) | 2015-06-18 | 2025-12-01 | 美商博得學院股份有限公司 | 降低脫靶效應的crispr酶突變 |
| US10648020B2 (en) | 2015-06-18 | 2020-05-12 | The Broad Institute, Inc. | CRISPR enzymes and systems |
| US9790490B2 (en) | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| WO2016205745A2 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Cell sorting |
| AU2016279077A1 (en) | 2015-06-18 | 2019-03-28 | Omar O. Abudayyeh | Novel CRISPR enzymes and systems |
| WO2016205749A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Novel crispr enzymes and systems |
| WO2017004261A1 (en) | 2015-06-29 | 2017-01-05 | Ionis Pharmaceuticals, Inc. | Modified crispr rna and modified single crispr rna and uses thereof |
| KR102804055B1 (ko) | 2015-07-13 | 2025-05-08 | 피벗 바이오, 인크. | 식물 형질 개선을 위한 방법 및 조성물 |
| JP7044373B2 (ja) | 2015-07-15 | 2022-03-30 | 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 | ヌクレアーゼ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 |
| US10676735B2 (en) | 2015-07-22 | 2020-06-09 | Duke University | High-throughput screening of regulatory element function with epigenome editing technologies |
| GB2592821B (en) * | 2015-07-31 | 2022-01-12 | Univ Minnesota | Modified cells and methods of therapy |
| US11667691B2 (en) | 2015-08-07 | 2023-06-06 | Novartis Ag | Treatment of cancer using chimeric CD3 receptor proteins |
| US9580727B1 (en) | 2015-08-07 | 2017-02-28 | Caribou Biosciences, Inc. | Compositions and methods of engineered CRISPR-Cas9 systems using split-nexus Cas9-associated polynucleotides |
| EP4512819A3 (en) | 2015-08-12 | 2025-08-06 | 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 |
| CA3033909A1 (en) | 2015-08-14 | 2017-02-23 | The University Of Sydney | Connexin 45 inhibition for therapy |
| WO2017031370A1 (en) | 2015-08-18 | 2017-02-23 | The Broad Institute, Inc. | Methods and compositions for altering function and structure of chromatin loops and/or domains |
| WO2017035416A2 (en) * | 2015-08-25 | 2017-03-02 | Duke University | Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases |
| US9512446B1 (en) | 2015-08-28 | 2016-12-06 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| US9926546B2 (en) | 2015-08-28 | 2018-03-27 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| JP6799586B2 (ja) | 2015-08-28 | 2020-12-16 | ザ ジェネラル ホスピタル コーポレイション | 遺伝子操作CRISPR−Cas9ヌクレアーゼ |
| ES2938623T3 (es) | 2015-09-09 | 2023-04-13 | Univ Kobe Nat Univ Corp | Método para convertir una secuencia del genoma de una bacteria gram-positiva mediante una conversión específica de una base de ácido nucleico de una secuencia de ADN seleccionada como diana y el complejo molecular utilizado en el mismo |
| IL241462A0 (en) | 2015-09-10 | 2015-11-30 | Yeda Res & Dev | Heterologous engineering of betalain pigments in plants |
| WO2017044843A1 (en) | 2015-09-11 | 2017-03-16 | The General Hospital Corporation | Full interrogation of nuclease dsbs and sequencing (find-seq) |
| EP3353296B1 (en) | 2015-09-24 | 2020-11-04 | Editas Medicine, Inc. | Use of exonucleases to improve crispr/cas-mediated genome editing |
| JP2018529353A (ja) | 2015-09-30 | 2018-10-11 | ザ ジェネラル ホスピタル コーポレイション | 配列決定法による切断事象の包括的生体外報告(CIRCLE−seq) |
| CA3001001A1 (en) | 2015-10-05 | 2017-04-13 | Massachusetts Institute Of Technology | Nitrogen fixation using refactored nif clusters |
| US12241053B2 (en) | 2015-10-09 | 2025-03-04 | The Brigham And Women's Hospital, Inc. | Modulation of novel immune checkpoint targets |
| EP4144844B1 (en) | 2015-10-12 | 2025-09-10 | DuPont US Holding, LLC | Protected dna templates for gene modification and increased homologous recombination in cells and methods of use |
| EP4089175A1 (en) | 2015-10-13 | 2022-11-16 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| JP6936952B2 (ja) | 2015-10-16 | 2021-09-22 | アストラゼネカ アクチボラグ | 細胞ゲノムの誘導性改変 |
| EP4636083A3 (en) | 2015-10-20 | 2026-01-21 | Institut National de la Santé et de la Recherche Médicale (INSERM) | Methods and products for genetic engineering |
| WO2017068077A1 (en) | 2015-10-20 | 2017-04-27 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods and products for genetic engineering |
| BR112018008134A2 (pt) | 2015-10-20 | 2018-11-06 | Pioneer Hi Bred Int | método para restaurar a função de um produto gênico não funcional no genoma de uma célula, método para editar uma sequência nucleotídica no genoma de uma célula, planta ou planta de progênie, método para editar uma sequência nucleotídica no genoma de uma célula sem o uso de um molde polinucleotídico de modificação e método para entregar um complexo de rna guia/endonuclease cas numa célula |
| CN116814590A (zh) | 2015-10-22 | 2023-09-29 | 布罗德研究所有限公司 | Vi-b型crispr酶和系统 |
| WO2017070598A1 (en) | 2015-10-23 | 2017-04-27 | Caribou Biosciences, Inc. | Engineered crispr class 2 cross-type nucleic-acid targeting nucleic acids |
| IL310721B2 (en) | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors and uses thereof |
| EP3368687B1 (en) | 2015-10-27 | 2021-09-29 | The Broad Institute, Inc. | Compositions and methods for targeting cancer-specific sequence variations |
| WO2017075478A2 (en) | 2015-10-28 | 2017-05-04 | The Broad Institute Inc. | Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures |
| US11092607B2 (en) | 2015-10-28 | 2021-08-17 | The Board Institute, Inc. | Multiplex analysis of single cell constituents |
| 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 |
| 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 |
| WO2017075294A1 (en) | 2015-10-28 | 2017-05-04 | The Board Institute Inc. | Assays for massively combinatorial perturbation profiling and cellular circuit reconstruction |
| AU2016349288A1 (en) | 2015-11-03 | 2018-05-31 | President And Fellows Of Harvard College | Method and apparatus for volumetric imaging of a three-dimensional nucleic acid containing matrix |
| CN108471731A (zh) * | 2015-11-06 | 2018-08-31 | 杰克逊实验室 | 大型基因组dna敲入及其用途 |
| CA2947904A1 (en) | 2015-11-12 | 2017-05-12 | Pfizer Inc. | Tissue-specific genome engineering using crispr-cas9 |
| EP4036228A1 (en) | 2015-11-13 | 2022-08-03 | Avellino Lab USA, Inc. | Methods for the treatment of corneal dystrophies |
| WO2017083766A1 (en) * | 2015-11-13 | 2017-05-18 | Massachusetts Institute Of Technology | High-throughput crispr-based library screening |
| 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 |
| US11905521B2 (en) | 2015-11-17 | 2024-02-20 | The Chinese University Of Hong Kong | Methods and systems for targeted gene manipulation |
| AU2016355178B9 (en) | 2015-11-19 | 2019-05-30 | Massachusetts Institute Of Technology | Lymphocyte antigen CD5-like (CD5L)-interleukin 12B (p40) heterodimers in immunity |
| WO2017087885A1 (en) | 2015-11-19 | 2017-05-26 | Dana-Farber Cancer Institute, Inc. | Methods of identifying compounds that interfere with erg-driven misguidance of baf complexes in tmprss2-erg driven prostate cancers |
| JP6923205B2 (ja) * | 2015-11-27 | 2021-08-18 | 国立大学法人神戸大学 | 標的化したdna配列の核酸塩基を特異的に変換する、単子葉植物のゲノム配列の変換方法、及びそれに用いる分子複合体 |
| US11903974B2 (en) | 2015-11-30 | 2024-02-20 | Flagship Pioneering Innovations V, Inc. | Methods and compositions relating to chondrisomes from cultured cells |
| KR102787119B1 (ko) | 2015-11-30 | 2025-03-27 | 듀크 유니버시티 | 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법 |
| CN105296518A (zh) * | 2015-12-01 | 2016-02-03 | 中国农业大学 | 一种用于CRISPR/Cas9技术的同源臂载体构建方法 |
| CN108473981B (zh) | 2015-12-04 | 2022-04-12 | 卡里布生物科学公司 | 工程化靶向核酸的核酸 |
| US11208649B2 (en) | 2015-12-07 | 2021-12-28 | Zymergen Inc. | HTP genomic engineering platform |
| BR112018011503A2 (pt) | 2015-12-07 | 2018-12-04 | Zymergen Inc | promotores da corynebacterium glutamicum |
| US9988624B2 (en) | 2015-12-07 | 2018-06-05 | Zymergen Inc. | Microbial strain improvement by a HTP genomic engineering platform |
| DK3387134T3 (da) | 2015-12-11 | 2020-12-21 | Danisco Us Inc | Fremgangsmåder og sammensætninger til øget nukleasemedieret genommodifikation og reducerede virkninger uden for målstedet |
| US12110490B2 (en) | 2015-12-18 | 2024-10-08 | The Broad Institute, Inc. | CRISPR enzymes and systems |
| EP3390631B1 (en) | 2015-12-18 | 2020-04-08 | Danisco US Inc. | Methods and compositions for t-rna based guide rna expression |
| US11542466B2 (en) | 2015-12-22 | 2023-01-03 | North Carolina State University | Methods and compositions for delivery of CRISPR based antimicrobials |
| RU2018127657A (ru) | 2015-12-30 | 2020-01-31 | Новартис Аг | Виды терапии на основе иммуноэффекторных клеток с улучшенной эффективностью |
| MX2018008345A (es) | 2016-01-11 | 2018-12-06 | Univ Leland Stanford Junior | Proteínas quiméricas y métodos de inmunoterapia. |
| IL260532B2 (en) | 2016-01-11 | 2023-12-01 | Univ Leland Stanford Junior | Chimeric proteins- containing systems and uses thereof in regulating gene expression |
| WO2017124037A1 (en) | 2016-01-15 | 2017-07-20 | The Children's Medical Center Corporation | Therapeutic use of mitochondria and combined mitochondrial agents |
| WO2017132239A1 (en) | 2016-01-26 | 2017-08-03 | Pioneer Hi-Bred International, Inc. | Waxy corn |
| US10876129B2 (en) | 2016-02-12 | 2020-12-29 | Ceres, Inc. | Methods and materials for high throughput testing of mutagenized allele combinations |
| US20190249172A1 (en) | 2016-02-18 | 2019-08-15 | The Regents Of The University Of California | Methods and compositions for gene editing in stem cells |
| US10973930B2 (en) | 2016-02-18 | 2021-04-13 | The Penn State Research Foundation | Generating GABAergic neurons in brains |
| US20190144942A1 (en) | 2016-02-22 | 2019-05-16 | Massachusetts Institute Of Technology | Methods for identifying and modulating immune phenotypes |
| WO2017151444A1 (en) | 2016-02-29 | 2017-09-08 | Agilent Technologies, Inc. | Methods and compositions for blocking off-target nucleic acids from cleavage by crispr proteins |
| JP2019507610A (ja) | 2016-03-04 | 2019-03-22 | インドア バイオテクノロジーズ インコーポレイテッド | CRISPR−Casゲノム編集に基づく、Fel d1ノックアウト並びに関連組成物及び方法 |
| EP3426778A1 (en) | 2016-03-11 | 2019-01-16 | Pioneer Hi-Bred International, Inc. | Novel cas9 systems and methods of use |
| WO2017155715A1 (en) | 2016-03-11 | 2017-09-14 | Pioneer Hi-Bred International, Inc. | Novel cas9 systems and methods of use |
| EP3426780A1 (en) | 2016-03-11 | 2019-01-16 | Pioneer Hi-Bred International, Inc. | Novel cas9 systems and methods of use |
| CN109414414A (zh) | 2016-03-16 | 2019-03-01 | 戴维·格拉德斯通研究所 | 用于治疗肥胖症和/或糖尿病以及用于鉴定候选治疗剂的方法和组合物 |
| 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 |
| EP3433362B1 (en) | 2016-03-23 | 2021-05-05 | Dana-Farber Cancer Institute, Inc. | Methods for enhancing the efficiency of gene editing |
| US11512311B2 (en) | 2016-03-25 | 2022-11-29 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency |
| US11597924B2 (en) | 2016-03-25 | 2023-03-07 | Editas Medicine, Inc. | Genome editing systems comprising repair-modulating enzyme molecules and methods of their use |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| US11236313B2 (en) | 2016-04-13 | 2022-02-01 | Editas Medicine, Inc. | Cas9 fusion molecules, gene editing systems, and methods of use thereof |
| CN109715808A (zh) | 2016-04-15 | 2019-05-03 | 诺华股份有限公司 | 用于选择性蛋白质表达的组合物和方法 |
| KR20260004568A (ko) | 2016-04-19 | 2026-01-08 | 더 브로드 인스티튜트, 인코퍼레이티드 | 신규한 crispr 효소 및 시스템 |
| EP3445853A1 (en) | 2016-04-19 | 2019-02-27 | The Broad Institute, Inc. | Cpf1 complexes with reduced indel activity |
| HUE055862T2 (hu) * | 2016-04-20 | 2021-12-28 | Centro De Investig Energeticas | Készítmények és eljárások PKLR fokozott génexpressziójára |
| CN105861485B (zh) * | 2016-04-20 | 2021-08-17 | 上海伊丽萨生物科技有限公司 | 一种提高基因置换效率的方法 |
| EP4613756A3 (en) | 2016-04-25 | 2025-11-12 | President And Fellows Of Harvard College | Hybridization chain reaction methods for in situ molecular detection |
| WO2017201425A1 (en) * | 2016-05-20 | 2017-11-23 | The Trustees Columbia University In The City Of New York | Anabolic enhancers for ameliorating neurodegeneration |
| WO2017205503A1 (en) | 2016-05-24 | 2017-11-30 | Indiana University Research And Technology Corporation | Ku inhibitors and their use |
| 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 |
| CN109642231A (zh) | 2016-06-17 | 2019-04-16 | 博德研究所 | Vi型crispr直向同源物和系统 |
| WO2017223128A1 (en) | 2016-06-21 | 2017-12-28 | The Curators Of The University Of Missouri | Modified dystrophin proteins |
| AU2017280353B2 (en) | 2016-06-24 | 2021-11-11 | Inscripta, Inc. | Methods for generating barcoded combinatorial libraries |
| WO2018005445A1 (en) | 2016-06-27 | 2018-01-04 | The Broad Institute, Inc. | Compositions and methods for detecting and treating diabetes |
| US12595478B2 (en) | 2016-06-29 | 2026-04-07 | The Broad Institute, Inc. | Crispr-Cas systems having destabilization domain |
| US10544390B2 (en) | 2016-06-30 | 2020-01-28 | Zymergen Inc. | Methods for generating a bacterial hemoglobin library and uses thereof |
| EP3478845A4 (en) | 2016-06-30 | 2019-07-31 | Zymergen, Inc. | METHOD OF GENERATING A GLUCOSE PERMEASE LIBRARY AND USES THEREOF |
| US20180004537A1 (en) | 2016-07-01 | 2018-01-04 | Microsoft Technology Licensing, Llc | Molecular State Machines |
| US11359234B2 (en) | 2016-07-01 | 2022-06-14 | Microsoft Technology Licensing, Llc | Barcoding sequences for identification of gene expression |
| ES2817973T3 (es) | 2016-07-01 | 2021-04-08 | Microsoft Technology Licensing Llc | Almacenamiento a través de la edición iterativa del ADN |
| MA45670A (fr) | 2016-07-13 | 2019-05-22 | Vertex Pharma | Procédés, compositions et kits pour augmenter l'efficacité d'édition du génome |
| SG11201900344YA (en) | 2016-07-15 | 2019-02-27 | Novartis Ag | Treatment and prevention of cytokine release syndrome using a chimeric antigen receptor in combination with a kinase inhibitor |
| JP7490211B2 (ja) | 2016-07-19 | 2024-05-27 | デューク ユニバーシティ | Cpf1に基づくゲノム編集の治療適用 |
| BR112019001887A2 (pt) | 2016-08-02 | 2019-07-09 | Editas Medicine Inc | composições e métodos para o tratamento de doença associada a cep290 |
| US11078481B1 (en) | 2016-08-03 | 2021-08-03 | KSQ Therapeutics, Inc. | Methods for screening for cancer targets |
| CN110214183A (zh) | 2016-08-03 | 2019-09-06 | 哈佛大学的校长及成员们 | 腺苷核碱基编辑器及其用途 |
| WO2018031683A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| EP4485466A3 (en) | 2016-08-17 | 2025-04-02 | The Broad Institute Inc. | Novel crispr enzymes and systems |
| WO2018035387A1 (en) | 2016-08-17 | 2018-02-22 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
| EP3500967A1 (en) | 2016-08-17 | 2019-06-26 | The Broad Institute, Inc. | Methods for identifying class 2 crispr-cas systems |
| WO2018035364A1 (en) | 2016-08-17 | 2018-02-22 | The Broad Institute Inc. | Product and methods useful for modulating and evaluating immune responses |
| WO2020225754A1 (en) | 2019-05-06 | 2020-11-12 | Mcmullen Tara | Crispr gene editing for autosomal dominant diseases |
| JP2019524149A (ja) * | 2016-08-20 | 2019-09-05 | アベリノ ラボ ユーエスエー インコーポレイテッドAvellino Lab USA, Inc. | 一本鎖ガイドRNA、CRISPR/Cas9システム、及びそれらの使用方法 |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| CN118853848A (zh) | 2016-08-31 | 2024-10-29 | 哈佛学院董事及会员团体 | 将生物分子的检测组合到使用荧光原位测序的单个试验的方法 |
| US11078483B1 (en) | 2016-09-02 | 2021-08-03 | KSQ Therapeutics, Inc. | Methods for measuring and improving CRISPR reagent function |
| US11525119B2 (en) | 2016-09-06 | 2022-12-13 | The Children's Medical Center Corporation | Immune cells derived from induced pluripotent stem cell |
| CA3035534A1 (en) | 2016-09-07 | 2018-03-15 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
| WO2018049025A2 (en) | 2016-09-07 | 2018-03-15 | The Broad Institute Inc. | Compositions and methods for evaluating and modulating immune responses |
| IL247752A0 (en) | 2016-09-11 | 2016-11-30 | Yeda Res & Dev | Compositions and methods for modulating gene expression for site-directed mutagenesis |
| US20190225974A1 (en) | 2016-09-23 | 2019-07-25 | BASF Agricultural Solutions Seed US LLC | Targeted genome optimization in plants |
| CN109844126A (zh) | 2016-09-28 | 2019-06-04 | 诺华股份有限公司 | 基于多孔膜的大分子递送系统 |
| US12499971B2 (en) | 2016-09-28 | 2025-12-16 | The Broad Institute, Inc. | Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof |
| BR112019006388A2 (pt) | 2016-09-30 | 2019-06-25 | Univ California | enzimas modificadoras de ácido nucleico guiadas por rna e métodos de uso das mesmas |
| MX2019003674A (es) | 2016-09-30 | 2021-01-08 | Univ California | Enzimas modificadoras de ácido nucleico guiadas por arn y métodos de uso de estas. |
| US11730823B2 (en) | 2016-10-03 | 2023-08-22 | President And Fellows Of Harvard College | Delivery of therapeutic RNAs via ARRDC1-mediated microvesicles |
| US10669539B2 (en) | 2016-10-06 | 2020-06-02 | Pioneer Biolabs, Llc | Methods and compositions for generating CRISPR guide RNA libraries |
| US12447213B2 (en) | 2016-10-07 | 2025-10-21 | The Broad Institute, Inc. | Modulation of novel immune checkpoint targets |
| US12012605B2 (en) | 2016-10-13 | 2024-06-18 | Pioneer Hi-Bred International, Inc. | Generating northern leaf blight resistant maize |
| KR102622411B1 (ko) | 2016-10-14 | 2024-01-10 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| GB201617559D0 (en) | 2016-10-17 | 2016-11-30 | University Court Of The University Of Edinburgh The | Swine comprising modified cd163 and associated methods |
| US10738338B2 (en) | 2016-10-18 | 2020-08-11 | The Research Foundation for the State University | Method and composition for biocatalytic protein-oligonucleotide conjugation and protein-oligonucleotide conjugate |
| 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 |
| CN110234762A (zh) * | 2016-10-28 | 2019-09-13 | 吉尼松公司 | 用于治疗肌强直性营养不良的组合物和方法 |
| WO2018081531A2 (en) | 2016-10-28 | 2018-05-03 | Ariad Pharmaceuticals, Inc. | Methods for human t-cell activation |
| WO2018076335A1 (en) | 2016-10-31 | 2018-05-03 | Institute Of Genetics And Developmental Biology, Chinese Academy Of Sciences | Compositions and methods for enhancing abiotic stress tolerance |
| EP3535396A1 (en) | 2016-11-01 | 2019-09-11 | Novartis AG | Methods and compositions for enhancing gene editing |
| US20190264218A1 (en) | 2016-11-04 | 2019-08-29 | Flagship Pioneering Innovations V, Inc. | Novel Plant Cells, Plants, and Seeds |
| EP4553082A3 (en) | 2016-11-04 | 2025-08-20 | Children's Hospital Medical Center | Liver organoid compositions and methods of making and using same |
| 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 |
| WO2018091971A1 (en) | 2016-11-15 | 2018-05-24 | Genomic Vision | Method for the monitoring of modified nucleases induced-gene editing events by molecular combing |
| CN110199031A (zh) | 2016-11-29 | 2019-09-03 | 基因组影像公司 | 设计用于分析感兴趣的遗传区域中的特定事件的一组多核苷酸序列的方法 |
| US9816093B1 (en) | 2016-12-06 | 2017-11-14 | Caribou Biosciences, Inc. | Engineered nucleic acid-targeting nucleic acids |
| US11129906B1 (en) | 2016-12-07 | 2021-09-28 | David Gordon Bermudes | Chimeric protein toxins for expression by therapeutic bacteria |
| US11180535B1 (en) | 2016-12-07 | 2021-11-23 | David Gordon Bermudes | Saccharide binding, tumor penetration, and cytotoxic antitumor chimeric peptides from therapeutic bacteria |
| US12404514B2 (en) | 2016-12-09 | 2025-09-02 | The Broad Institute, Inc. | CRISPR-systems for modifying a trait of interest in a plant |
| AU2017378427A1 (en) | 2016-12-14 | 2019-06-20 | Ligandal, Inc. | Methods and compositions for nucleic acid and protein payload delivery |
| EP3555297A1 (en) | 2016-12-19 | 2019-10-23 | Editas Medicine, Inc. | Assessing nuclease cleavage |
| CN110213961A (zh) | 2016-12-22 | 2019-09-06 | 孟山都技术公司 | 基于基因组编辑的作物工程化和生产矮秆植物 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| EP3565608A4 (en) | 2017-01-05 | 2020-12-16 | Rutgers, The State University of New Jersey | TARGETED GENE EDITING PLATFORM INDEPENDENT OF BICATENARY DNA BREAKAGE AND ITS USES |
| WO2018127585A1 (en) | 2017-01-06 | 2018-07-12 | Txcell | Monospecific regulatory t cell population with cytotoxicity for b cells |
| JP7229923B2 (ja) | 2017-01-06 | 2023-02-28 | エディタス・メディシン、インコーポレイテッド | ヌクレアーゼ切断を評価する方法 |
| EP3346001A1 (en) | 2017-01-06 | 2018-07-11 | TXCell | Monospecific regulatory t cell population with cytotoxicity for b cells |
| WO2018129440A1 (en) | 2017-01-09 | 2018-07-12 | University Of Massachusetts | Complexes for gene deletion and editing |
| CN116904333A (zh) | 2017-01-12 | 2023-10-20 | 皮沃特生物公司 | 用于改良植物性状的方法及组合物 |
| ES2912408T3 (es) | 2017-01-26 | 2022-05-25 | Novartis Ag | Composiciones de CD28 y métodos para terapia con receptores quiméricos para antígenos |
| WO2018140899A1 (en) | 2017-01-28 | 2018-08-02 | Inari Agriculture, Inc. | Novel plant cells, plants, and seeds |
| CN110582509A (zh) | 2017-01-31 | 2019-12-17 | 诺华股份有限公司 | 使用具有多特异性的嵌合t细胞受体蛋白治疗癌症 |
| 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 |
| JP2020507312A (ja) | 2017-02-10 | 2020-03-12 | ザイマージェン インコーポレイテッド | 複数の宿主用の複数のdnaコンストラクトのアセンブリ及び編集のためのモジュラーユニバーサルプラスミド設計戦略 |
| US10828330B2 (en) | 2017-02-22 | 2020-11-10 | IO Bioscience, Inc. | Nucleic acid constructs comprising gene editing multi-sites and uses thereof |
| US12440578B2 (en) | 2017-02-22 | 2025-10-14 | Io Biosciences, Inc. | Nucleic acid constructs comprising gene editing multi-sites and uses thereof |
| WO2018160731A1 (en) | 2017-02-28 | 2018-09-07 | Novartis Ag | Shp inhibitor compositions and uses for chimeric antigen receptor therapy |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| WO2018170184A1 (en) | 2017-03-14 | 2018-09-20 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| JP2020511141A (ja) | 2017-03-15 | 2020-04-16 | ザ・ブロード・インスティテュート・インコーポレイテッド | 新規Cas13bオルソログCRISPR酵素及び系 |
| WO2018170436A1 (en) | 2017-03-16 | 2018-09-20 | Jacobs Farm Del Cabo | Basil with high tolerance to downy mildew |
| WO2018170515A1 (en) | 2017-03-17 | 2018-09-20 | The Broad Institute, Inc. | Methods for identifying and modulating co-occurant cellular phenotypes |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| WO2018175924A1 (en) | 2017-03-24 | 2018-09-27 | The Broad Institute, Inc. | Methods and compositions for regulating innate lymphoid cell inflammatory responses |
| AU2018243654B2 (en) | 2017-03-31 | 2024-07-25 | Pioneer Hi-Bred International, Inc. | Expression modulating elements and use thereof |
| US11963966B2 (en) | 2017-03-31 | 2024-04-23 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for treating ovarian tumors |
| WO2018183921A1 (en) | 2017-04-01 | 2018-10-04 | The Broad Institute, Inc. | Methods and compositions for detecting and modulating an immunotherapy resistance gene signature in cancer |
| EP3610266A4 (en) | 2017-04-12 | 2021-04-21 | Massachusetts Eye and Ear Infirmary | TUMOR SIGNATURE OF METASTASIS, COMPOSITIONS OF SUBSTANCES AND USES THEREOF |
| US12590288B2 (en) | 2017-04-12 | 2026-03-31 | The Broad Institute, Inc. | Method of treating an inflammatory disease by administering an agent which binds a surface receptor on a tuft cell that induces an ILC class 2 inflammatory response |
| US12331319B2 (en) | 2017-04-12 | 2025-06-17 | The Broad Institute, Inc. | Respiratory and sweat gland ionocytes |
| AU2018251801B2 (en) | 2017-04-12 | 2024-11-07 | Massachusetts Institute Of Technology | Novel type VI CRISPR orthologs and systems |
| WO2018191750A2 (en) | 2017-04-14 | 2018-10-18 | The Broad Institute Inc. | Novel delivery of large payloads |
| JP7248586B2 (ja) | 2017-04-14 | 2023-03-29 | チルドレンズ ホスピタル メディカル センター | 複数ドナー幹細胞組成物およびそれを作製する方法 |
| MX2019012398A (es) | 2017-04-18 | 2020-09-25 | Broad Inst Inc | Composiciones para detectar secreciones y metodos de uso. |
| US11834670B2 (en) | 2017-04-19 | 2023-12-05 | Global Life Sciences Solutions Usa Llc | Site-specific DNA modification using a donor DNA repair template having tandem repeat sequences |
| KR102746223B1 (ko) | 2017-04-20 | 2024-12-27 | 이제네시스, 인크. | 유전자 변형 동물의 생성 방법 |
| US12208140B2 (en) | 2017-04-21 | 2025-01-28 | The Broad Institute, Inc. | Targeted delivery to beta cells |
| US11591589B2 (en) | 2017-04-21 | 2023-02-28 | The General Hospital Corporation | Variants of Cpf1 (Cas12a) with altered PAM specificity |
| US11499151B2 (en) | 2017-04-28 | 2022-11-15 | Editas Medicine, Inc. | Methods and systems for analyzing guide RNA molecules |
| US20200179511A1 (en) | 2017-04-28 | 2020-06-11 | Novartis Ag | Bcma-targeting agent, and combination therapy with a gamma secretase inhibitor |
| EP3615055A1 (en) | 2017-04-28 | 2020-03-04 | Novartis AG | Cells expressing a bcma-targeting chimeric antigen receptor, and combination therapy with a gamma secretase inhibitor |
| US11591601B2 (en) | 2017-05-05 | 2023-02-28 | The Broad Institute, Inc. | Methods for identification and modification of lncRNA associated with target genotypes and phenotypes |
| EP3622070A2 (en) | 2017-05-10 | 2020-03-18 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| EP3622092A4 (en) | 2017-05-11 | 2021-06-23 | The Broad Institute, Inc. | METHODS AND COMPOSITIONS OF USE OF CD8 + TUMOR-INFILTRATING LYMPHOCYTE SUBTYPES AND GENE SIGNATURES THEREOF |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| US11866697B2 (en) | 2017-05-18 | 2024-01-09 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| WO2018213726A1 (en) | 2017-05-18 | 2018-11-22 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| EP3630849A4 (en) | 2017-05-25 | 2021-01-13 | The General Hospital Corporation | BIPARTITE BASIC EDITOR (BBE) AND TYPE II-C-CAS9 ZINC FINGER EDITOR ARCHITECTURES |
| EP3409104A1 (en) | 2017-05-31 | 2018-12-05 | Vilmorin et Cie | Tomato plant resistant to tomato yellow leaf curl virus, powdery mildew, and nematodes |
| EP3409106A1 (en) | 2017-06-01 | 2018-12-05 | Vilmorin et Cie | Tolerance in plants of solanum lycopersicum to the tobamovirus tomato brown rugose fruit virus (tbrfv) |
| WO2020249996A1 (en) | 2019-06-14 | 2020-12-17 | Vilmorin & Cie | Resistance in plants of solanum lycopersicum to the tobamovirus tomato brown rugose fruit virus |
| CA3102054A1 (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 |
| CA3065946A1 (en) | 2017-06-05 | 2018-12-13 | Research Institute At Nationwide Children's Hospital | Enhanced modified viral capsid proteins |
| WO2018226900A2 (en) | 2017-06-06 | 2018-12-13 | Zymergen Inc. | A htp genomic engineering platform for improving fungal strains |
| WO2018226880A1 (en) | 2017-06-06 | 2018-12-13 | Zymergen Inc. | A htp genomic engineering platform for improving escherichia coli |
| DK3636753T5 (da) * | 2017-06-08 | 2024-08-26 | Univ Osaka | Fremgangsmåde til fremstilling af DNA-redigeret eukaryotisk celle |
| MX2019014640A (es) | 2017-06-09 | 2020-10-05 | Editas Medicine Inc | Nucleasas de repeticiones palíndromicas agrupadas y regularmente interespaciadas de proteina asociada 9 (cas9) genomanipuladas. |
| WO2018226972A2 (en) | 2017-06-09 | 2018-12-13 | Vilmorin & Cie | Compositions and methods for genome editing |
| IL271275B1 (en) | 2017-06-13 | 2026-04-01 | Flagship Pioneering Innovations V Inc | Preparations containing croons and their uses |
| WO2018232195A1 (en) | 2017-06-14 | 2018-12-20 | The Broad Institute, Inc. | Compositions and methods targeting complement component 3 for inhibiting tumor growth |
| WO2018232356A1 (en) | 2017-06-15 | 2018-12-20 | The Regents Of The University Of California | Targeted non-viral dna insertions |
| US9982279B1 (en) | 2017-06-23 | 2018-05-29 | Inscripta, Inc. | Nucleic acid-guided nucleases |
| US10011849B1 (en) | 2017-06-23 | 2018-07-03 | Inscripta, Inc. | Nucleic acid-guided nucleases |
| KR102761791B1 (ko) | 2017-06-26 | 2025-02-05 | 더 브로드 인스티튜트, 인코퍼레이티드 | 표적화된 핵산 편집을 위한 crispr/cas-아데닌 데아미나아제 기반 조성물, 시스템 및 방법 |
| EP3645021A4 (en) | 2017-06-30 | 2021-04-21 | Intima Bioscience, Inc. | ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY |
| US10392616B2 (en) | 2017-06-30 | 2019-08-27 | Arbor Biotechnologies, Inc. | CRISPR RNA targeting enzymes and systems and uses thereof |
| US20200140896A1 (en) | 2017-06-30 | 2020-05-07 | Novartis Ag | Methods for the treatment of disease with gene editing systems |
| US12049643B2 (en) | 2017-07-14 | 2024-07-30 | The Broad Institute, Inc. | Methods and compositions for modulating cytotoxic lymphocyte activity |
| US11866726B2 (en) | 2017-07-14 | 2024-01-09 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| US12105089B2 (en) | 2017-07-17 | 2024-10-01 | The Broad Institute, Inc. | Cell atlas of the healthy and ulcerative colitis human colon |
| US20200231974A1 (en) * | 2017-07-18 | 2020-07-23 | Genovie Ab | A two-component vector library system for rapid assembly and diversification of full-length t-cell receptor open reading frames |
| US10510743B2 (en) * | 2017-07-18 | 2019-12-17 | Hong Kong Applied Science and Technology Research Institute Company, Limited | Step fin field-effect-transistor (FinFET) with slim top of fin and thick bottom of fin for electro-static-discharge (ESD) or electrical over-stress (EOS) protection |
| US12239646B2 (en) | 2017-07-19 | 2025-03-04 | Dana-Farber Cancer Institute, Inc. | Cancer diagnostic and treatment |
| WO2019023291A2 (en) | 2017-07-25 | 2019-01-31 | Dana-Farber Cancer Institute, Inc. | COMPOSITIONS AND METHODS FOR PRODUCTION AND DECODING OF GUIDE RNA LIBRARIES AND USES THEREOF |
| 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 |
| EP3625356B1 (en) | 2017-08-08 | 2021-05-19 | Depixus | In vitro isolation and enrichment of nucleic acids using site-specific nucleases |
| US10476825B2 (en) | 2017-08-22 | 2019-11-12 | Salk Institue for Biological Studies | RNA targeting methods and compositions |
| US11970720B2 (en) | 2017-08-22 | 2024-04-30 | Salk Institute For Biological Studies | RNA targeting methods and compositions |
| CA3073448A1 (en) | 2017-08-23 | 2019-02-28 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
| IL272786B2 (en) | 2017-08-25 | 2025-03-01 | Codiak Biosciences Inc | Preparation of therapeutic exosomes using membrane proteins |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| EP3679143A1 (en) | 2017-09-08 | 2020-07-15 | Life Technologies Corporation | Methods for improved homologous recombination and compositions thereof |
| US20190076814A1 (en) | 2017-09-11 | 2019-03-14 | Synthego Corporation | Biopolymer synthesis system and method |
| EP4595770A3 (en) | 2017-09-15 | 2025-10-08 | Covercress Inc. | Low fiber pennycress meal and methods of making |
| SG11202002520UA (en) | 2017-09-19 | 2020-04-29 | Univ British Columbia | Anti-hla-a2 antibodies and methods of using the same |
| EP3684822A4 (en) | 2017-09-20 | 2021-06-16 | The University of British Columbia | NEW ANTI-HLA-A2 ANTIBODIES AND USES THEREOF |
| CN109517820B (zh) | 2017-09-20 | 2021-09-24 | 北京宇繁生物科技有限公司 | 一种靶向HPK1的gRNA以及HPK1基因编辑方法 |
| US11252928B2 (en) | 2017-09-21 | 2022-02-22 | The Condard-Pyle Company | Miniature rose plant named ‘Meibenbino’ |
| US11917978B2 (en) | 2017-09-21 | 2024-03-05 | The Conard Pyle Company | Miniature rose plant named ‘meibenbino’ |
| AU2018338318B2 (en) | 2017-09-21 | 2022-12-22 | Massachusetts Institute Of Technology | Systems, methods, and compositions for targeted nucleic acid editing |
| CA3076249A1 (en) | 2017-09-21 | 2019-03-28 | The Brigham And Women's Hospital, Inc. | Isolation, preservation, compositions and uses of extracts from justicia plants |
| CA3073449A1 (en) | 2017-09-25 | 2019-03-28 | Agrospheres, Inc. | Compositions and methods for scalable production and delivery of biologicals |
| 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 |
| EP3694603B1 (en) | 2017-10-10 | 2026-04-08 | Children's Hospital Medical Center | Esophageal tissue and/or organoid compositions and methods of making same |
| WO2019075197A1 (en) | 2017-10-11 | 2019-04-18 | The General Hospital Corporation | METHODS OF DETECTION OF INDIVIDUAL SITE-SPECIFIC PARASITE GENOMIC DEAMINATION BY BASE EDITING TECHNOLOGIES |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| SG11202002263TA (en) | 2017-10-16 | 2020-04-29 | Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas | Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency |
| US11680296B2 (en) | 2017-10-16 | 2023-06-20 | Massachusetts Institute Of Technology | Mycobacterium tuberculosis host-pathogen interaction |
| CA3079681A1 (en) | 2017-10-20 | 2019-04-25 | Fred Hutchinson Cancer Research Center | Systems and methods to produce b cells genetically modified to express selected antibodies |
| WO2019135816A2 (en) | 2017-10-23 | 2019-07-11 | The Broad Institute, Inc. | Novel nucleic acid modifiers |
| CN111527057A (zh) | 2017-10-25 | 2020-08-11 | 皮沃特生物股份有限公司 | 靶向改良植物性状的固氮的基因靶标 |
| MX2020004343A (es) | 2017-10-25 | 2021-01-08 | Pivot Bio Inc | Métodos y composiciones para mejorar microbios modificados que fijan nitrógeno. |
| JP7101419B2 (ja) | 2017-10-27 | 2022-07-15 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 内因性t細胞受容体の標的置換 |
| WO2019089803A1 (en) | 2017-10-31 | 2019-05-09 | The Broad Institute, Inc. | Methods and compositions for studying cell evolution |
| US20210179709A1 (en) | 2017-10-31 | 2021-06-17 | Novartis Ag | Anti-car compositions and methods |
| AU2018358051B2 (en) | 2017-11-01 | 2025-01-09 | The Regents Of The University Of California | CasZ compositions and methods of use |
| WO2019089804A1 (en) | 2017-11-01 | 2019-05-09 | The Regents Of The University Of California | Casy compositions and methods of use |
| EP3704245A1 (en) | 2017-11-01 | 2020-09-09 | Novartis AG | Synthetic rnas and methods of use |
| WO2019090175A1 (en) | 2017-11-02 | 2019-05-09 | Arbor Biotechnologies, Inc. | Novel crispr-associated transposon systems and components |
| EP3707263A4 (en) | 2017-11-06 | 2021-06-16 | Shenzhen Cell Inspire Biotechnology Co., Ltd. | NOVEL, HUMAN INDUCED PLURIPOTENT STEM CELL LINES FOR MODELING AND USING ALZHEIMER'S MORBUS |
| WO2019094955A1 (en) | 2017-11-13 | 2019-05-16 | The Broad Institute, Inc. | Methods and compositions for targeting developmental and oncogenic programs in h3k27m gliomas |
| EP3710039A4 (en) | 2017-11-13 | 2021-08-04 | The Broad Institute, Inc. | METHODS AND COMPOSITIONS FOR TREATMENT OF CANCER BY TARGETING THE CLEC2D-KLRB1 PATH |
| US12221720B2 (en) | 2017-11-13 | 2025-02-11 | The Broad Institute, Inc. | Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells |
| CR20200251A (es) | 2017-11-17 | 2020-07-17 | Iovance Biotherapeutics Inc | Expansión de til de aspirados con aguja fina y biopsias por punción |
| EP3714041A1 (en) | 2017-11-22 | 2020-09-30 | Iovance Biotherapeutics, Inc. | Expansion of peripheral blood lymphocytes (pbls) from peripheral blood |
| JP2021505144A (ja) | 2017-12-05 | 2021-02-18 | シンセティック ジェノミクス インコーポレーテッド | シグナル伝達タンパク質の遺伝子改変を介した藻類脂質生産性の改善方法 |
| WO2019113506A1 (en) | 2017-12-07 | 2019-06-13 | The Broad Institute, Inc. | Methods and compositions for multiplexing single cell and single nuclei sequencing |
| CA3082956A1 (en) | 2017-12-08 | 2019-06-13 | Synthetic Genomics, Inc. | Improving algal lipid productivity via genetic modification of a tpr domain containing protein |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| EP3724216A1 (en) | 2017-12-15 | 2020-10-21 | Dana Farber Cancer Institute, Inc. | Stabilized peptide-mediated targeted protein degradation |
| BR112020011011A2 (pt) | 2017-12-15 | 2020-11-17 | Danisco Us Inc. | variantes de cas9 e métodos de uso |
| EP3727394A4 (en) | 2017-12-21 | 2021-09-08 | Children's Hospital Medical Center | DIGITALIZED HUMAN ORGANOIDS AND METHOD OF USING THEREOF |
| CN111655271B (zh) | 2017-12-28 | 2025-09-23 | 隆萨销售股份公司 | 用于免疫肿瘤学和抗炎疗法的外来体 |
| WO2019133881A1 (en) | 2017-12-29 | 2019-07-04 | Rubius Therapeutics, Inc. | Gene editing and targeted transcriptional modulation for enginerering erythroid cells |
| 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 |
| AU2019207409B2 (en) | 2018-01-12 | 2023-02-23 | Basf Se | Gene underlying the number of spikelets per spike qtl in wheat on chromosome 7a |
| MA51619A (fr) | 2018-01-17 | 2021-04-14 | Vertex Pharma | Inhibiteurs de la protéine kinase dépendante de l'adn |
| JP7466448B2 (ja) | 2018-01-17 | 2024-04-12 | 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 | Dna-pk阻害剤 |
| BR112020013626A2 (pt) | 2018-01-17 | 2020-12-01 | Vertex Pharmaceuticals Incorporated | compostos de quinoxalinona, composições, métodos e kits para aumentar a eficiência de edição de genoma |
| EP3740580A4 (en) | 2018-01-19 | 2021-10-20 | Duke University | GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS |
| WO2019143926A1 (en) | 2018-01-19 | 2019-07-25 | Covercress Inc. | Low glucosinolate pennycress meal and methods of making |
| WO2019147743A1 (en) | 2018-01-26 | 2019-08-01 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
| US11926835B1 (en) | 2018-01-29 | 2024-03-12 | Inari Agriculture Technology, Inc. | Methods for efficient tomato genome editing |
| US11497752B2 (en) | 2018-01-30 | 2022-11-15 | Foghorn Therapeutics Inc. | Compounds and uses thereof |
| AR114350A1 (es) | 2018-01-31 | 2020-08-26 | Res Institute At Nationwide ChildrenS Hospital | Terapia genética para la distrofia muscular de cinturas del tipo 2c |
| CN111699254A (zh) | 2018-02-08 | 2020-09-22 | 齐默尔根公司 | 使用crispr在棒状杆菌属中进行基因组编辑 |
| KR102465067B1 (ko) * | 2018-02-15 | 2022-11-10 | 시그마-알드리치 컴퍼니., 엘엘씨 | 진핵 게놈 변형을 위한 조작된 cas9 시스템 |
| WO2019165168A1 (en) | 2018-02-23 | 2019-08-29 | Pioneer Hi-Bred International, Inc. | Novel cas9 orthologs |
| WO2019173125A1 (en) | 2018-03-09 | 2019-09-12 | Pioneer Hi-Bred International, Inc. | Compositions and methods for modification of fatty acids in soybean |
| WO2019178428A1 (en) | 2018-03-14 | 2019-09-19 | Arbor Biotechnologies, Inc. | Novel crispr dna and rna targeting enzymes and systems |
| WO2019178427A1 (en) | 2018-03-14 | 2019-09-19 | Arbor Biotechnologies, Inc. | Novel crispr dna targeting enzymes and systems |
| KR102907245B1 (ko) | 2018-03-14 | 2026-01-05 | 에디타스 메디신, 인코포레이티드 | 혈색소병증 치료를 위한 시스템 및 방법 |
| US10760075B2 (en) | 2018-04-30 | 2020-09-01 | Snipr Biome Aps | Treating and preventing microbial infections |
| GB201805287D0 (en) | 2018-03-29 | 2018-05-16 | Univ Edinburgh | Haematoietic stem cell treatment |
| JP7522038B2 (ja) | 2018-04-06 | 2024-07-24 | ザ チルドレンズ メディカル センター コーポレーション | 体細胞リプログラミングおよびインプリンティングのモジュレートのための組成物および方法 |
| WO2019200167A1 (en) | 2018-04-11 | 2019-10-17 | Rocket Pharmaceuticals, Ltd. | Compositions and methods for stem cell transplant |
| AU2019250692A1 (en) | 2018-04-13 | 2020-11-05 | Sangamo Therapeutics France | Chimeric antigen receptor specific for Interleukin-23 receptor |
| US11898203B2 (en) | 2018-04-17 | 2024-02-13 | The General Hospital Corporation | Highly sensitive in vitro assays to define substrate preferences and sites of nucleic-acid binding, modifying, and cleaving agents |
| WO2019204585A1 (en) | 2018-04-19 | 2019-10-24 | Massachusetts Institute Of Technology | Single-stranded break detection in double-stranded dna |
| KR102922694B1 (ko) | 2018-04-19 | 2026-02-03 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 유전자 편집을 위한 조성물 및 방법 |
| HUE059223T2 (hu) | 2018-04-24 | 2022-10-28 | Kws Saat Se & Co Kgaa | Jobb emészthetõségû növények és marker haplotípusok |
| US11957695B2 (en) | 2018-04-26 | 2024-04-16 | The Broad Institute, Inc. | Methods and compositions targeting glucocorticoid signaling for modulating immune responses |
| WO2019210153A1 (en) | 2018-04-27 | 2019-10-31 | Novartis Ag | Car t cell therapies with enhanced efficacy |
| WO2019210268A2 (en) | 2018-04-27 | 2019-10-31 | The Broad Institute, Inc. | Sequencing-based proteomics |
| WO2019210131A1 (en) | 2018-04-27 | 2019-10-31 | Iovance Biotherapeutics, Inc. | Closed process for expansion and gene editing of tumor infiltrating lymphocytes and uses of same in immunotherapy |
| EP3788094A2 (en) | 2018-04-27 | 2021-03-10 | Genedit Inc. | Cationic polymer and use for biomolecule delivery |
| WO2019213660A2 (en) | 2018-05-04 | 2019-11-07 | The Broad Institute, Inc. | Compositions and methods for modulating cgrp signaling to regulate innate lymphoid cell inflammatory responses |
| KR20210045360A (ko) | 2018-05-16 | 2021-04-26 | 신테고 코포레이션 | 가이드 rna 설계 및 사용을 위한 방법 및 시스템 |
| WO2019222555A1 (en) | 2018-05-16 | 2019-11-21 | Arbor Biotechnologies, Inc. | Novel crispr-associated systems and components |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| CA3096859A1 (en) | 2018-05-25 | 2019-11-28 | Pioneer Hi-Bred International, Inc. | Systems and methods for improved breeding by modulating recombination rates |
| CN108707628B (zh) * | 2018-05-28 | 2021-11-23 | 上海海洋大学 | 斑马鱼notch2基因突变体的制备方法 |
| WO2019232542A2 (en) | 2018-06-01 | 2019-12-05 | Massachusetts Institute Of Technology | Methods and compositions for detecting and modulating microenvironment gene signatures from the csf of metastasis patients |
| US11866719B1 (en) | 2018-06-04 | 2024-01-09 | Inari Agriculture Technology, Inc. | Heterologous integration of regulatory elements to alter gene expression in wheat cells and wheat plants |
| 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 |
| WO2019241315A1 (en) | 2018-06-12 | 2019-12-19 | Obsidian Therapeutics, Inc. | Pde5 derived regulatory constructs and methods of use in immunotherapy |
| TWI890660B (zh) | 2018-06-13 | 2025-07-21 | 瑞士商諾華公司 | Bcma 嵌合抗原受體及其用途 |
| US12036240B2 (en) | 2018-06-14 | 2024-07-16 | The Broad Institute, Inc. | Compositions and methods targeting complement component 3 for inhibiting tumor growth |
| WO2019246173A1 (en) | 2018-06-19 | 2019-12-26 | Lunella Biotech, Inc. | "energetic" cancer stem cells (e-cscs): a new hyper-metabolic and proliferative tumor cell phenotype, driven by mitochondrial energy |
| EP3814519A1 (en) | 2018-06-26 | 2021-05-05 | The Broad Institute, Inc. | Crispr/cas and transposase based amplification compositions, systems and methods |
| WO2020006036A1 (en) | 2018-06-26 | 2020-01-02 | Massachusetts Institute Of Technology | Crispr effector system based amplification methods, systems, and diagnostics |
| EP3813511A4 (en) | 2018-06-27 | 2022-08-03 | Pivot Bio, Inc. | GUIDED MICROBIAL REMODELING, PLATFORM FOR RATIONAL IMPROVEMENT OF MICROBIAL SPECIES FOR AGRICULTURE |
| WO2020006064A2 (en) | 2018-06-27 | 2020-01-02 | Pivot Bio, Inc. | Agricultural compositions comprising remodeled nitrogen fixing microbes |
| EP3814369A1 (en) | 2018-06-29 | 2021-05-05 | Stichting Het Nederlands Kanker Instituut- Antoni van Leeuwenhoek Ziekenhuis | Tweak-receptor agonists for use in combination with immunotherapy of a cancer |
| 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 |
| EP3823633A4 (en) | 2018-06-29 | 2023-05-03 | Editas Medicine, Inc. | SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| KR20210060434A (ko) | 2018-07-11 | 2021-05-26 | 피벗 바이오, 인크. | 리모델링된 미생물에 의한 시간적 및 공간적 표적화 동적 질소 전달 |
| JP2021530212A (ja) | 2018-07-13 | 2021-11-11 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California | レトロトランスポゾンベースの送達媒体及びその使用方法 |
| WO2020018142A1 (en) | 2018-07-16 | 2020-01-23 | Arbor Biotechnologies, Inc. | Novel crispr dna targeting enzymes and systems |
| EP4678727A3 (en) | 2018-07-26 | 2026-03-11 | Children's Hospital Medical Center | Hepato-biliary-pancreatic tissues and methods of making same |
| SG11202102068TA (en) | 2018-07-31 | 2021-03-30 | Broad Inst Inc | Novel crispr enzymes and systems |
| EP3830301B1 (en) | 2018-08-01 | 2024-05-22 | Mammoth Biosciences, Inc. | Programmable nuclease compositions and methods of use thereof |
| JP2021532815A (ja) | 2018-08-07 | 2021-12-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 新規Cas12b酵素およびシステム |
| KR20210046006A (ko) | 2018-08-10 | 2021-04-27 | 상가모 테라퓨틱스 프랑스 | Tnfr2 도메인을 포함하는 신규한 car 작제물 |
| EP3607819A1 (en) | 2018-08-10 | 2020-02-12 | Vilmorin et Cie | Resistance to xanthomonas campestris pv. campestris (xcc) in cauliflower |
| CA3107002A1 (en) | 2018-08-15 | 2020-04-30 | Zymergen Inc. | Applications of crispri in high throughput metabolic engineering |
| US12421507B2 (en) | 2018-08-20 | 2025-09-23 | 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 |
| US12275964B2 (en) | 2018-08-22 | 2025-04-15 | The Regents Of The University Of California | Variant type V CRISPR/Cas effector polypeptides and methods of use thereof |
| AU2019326408A1 (en) | 2018-08-23 | 2021-03-11 | Sangamo Therapeutics, Inc. | Engineered target specific base editors |
| MX2021002418A (es) | 2018-08-30 | 2021-04-28 | Univ North Carolina Chapel Hill | Genes sinteticos activados por retroalimentacion, casetes de coincidencia de semilla objetivo y sus usos. |
| US12545910B2 (en) | 2018-08-31 | 2026-02-10 | Inari Agriculture Technology, Inc. | Compositions, systems, and methods for genome editing |
| US11479762B1 (en) | 2018-08-31 | 2022-10-25 | Inari Agriculture Technology, Inc. | Compositions, systems, and methods for genome editing |
| US12534743B2 (en) | 2018-08-31 | 2026-01-27 | Inari Agriculture Technology, Inc. | Compositions, systems, and methods for genome editing |
| WO2020051507A1 (en) | 2018-09-06 | 2020-03-12 | The Broad Institute, Inc. | Nucleic acid assemblies for use in targeted delivery |
| CA3112026A1 (en) | 2018-09-12 | 2020-03-19 | Children's Hospital Medical Center | Organoid compositions for the production of hematopoietic stem cells and derivatives thereof |
| WO2020056170A1 (en) | 2018-09-12 | 2020-03-19 | Fred Hutchinson Cancer Research Center | Reducing cd33 expression to selectively protect therapeutic cells |
| AU2019343045A1 (en) | 2018-09-18 | 2021-05-13 | Vnv Newco Inc. | ARC-based capsids and uses thereof |
| CN109265562B (zh) * | 2018-09-26 | 2021-03-30 | 北京市农林科学院 | 一种切刻酶及其在基因组碱基替换中的应用 |
| GB201815820D0 (en) | 2018-09-28 | 2018-11-14 | Univ Wageningen | Off-target activity inhibitors for guided endonucleases |
| WO2020072250A1 (en) | 2018-10-01 | 2020-04-09 | North Carolina State University | Recombinant type i crispr-cas system and uses thereof for genome modification and alteration of expression |
| US12203123B2 (en) | 2018-10-01 | 2025-01-21 | North Carolina State University | Recombinant type I CRISPR-Cas system and uses thereof for screening for variant cells |
| WO2020072254A1 (en) | 2018-10-01 | 2020-04-09 | North Carolina State University | Recombinant type i crispr-cas system and uses thereof for killing target cells |
| WO2020072248A1 (en) | 2018-10-01 | 2020-04-09 | North Carolina State University | Recombinant type i crispr-cas system |
| 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 |
| US11851663B2 (en) | 2018-10-14 | 2023-12-26 | Snipr Biome Aps | Single-vector type I vectors |
| US20210379057A1 (en) | 2018-10-16 | 2021-12-09 | Massachusetts Institute Of Technology | Nutlin-3a for use in treating a mycobacterium tuberculosis infection |
| WO2020081588A1 (en) | 2018-10-17 | 2020-04-23 | Dana-Farber Cancer Institute, Inc. | Swi/snf family chromatin remodeling complexes and uses thereof |
| US12116458B2 (en) | 2018-10-24 | 2024-10-15 | Genedit Inc. | Cationic polymer with alkyl side chains and use for biomolecule delivery |
| US20210386788A1 (en) | 2018-10-24 | 2021-12-16 | Obsidian Therapeutics, Inc. | Er tunable protein regulation |
| US11407995B1 (en) | 2018-10-26 | 2022-08-09 | Inari Agriculture Technology, Inc. | RNA-guided nucleases and DNA binding proteins |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| KR102929494B1 (ko) * | 2018-10-29 | 2026-02-20 | 차이나 어그리컬처럴 유니버시티 | 신규 CRISPR/Cas12f 효소 및 시스템 |
| SG11202104409YA (en) | 2018-10-31 | 2021-05-28 | Zymergen Inc | Multiplexed deterministic assembly of dna libraries |
| AU2019369418A1 (en) | 2018-10-31 | 2021-03-18 | Pioneer Hi-Bred International, Inc. | Compositions and methods for Ochrobactrum-mediated gene editing |
| US11970733B2 (en) | 2018-11-01 | 2024-04-30 | Synthego Corporation | Methods for analyzing nucleic acid sequences |
| US11434477B1 (en) | 2018-11-02 | 2022-09-06 | Inari Agriculture Technology, Inc. | RNA-guided nucleases and DNA binding proteins |
| MY210603A (en) | 2018-11-05 | 2025-10-01 | Iovance Biotherapeutics Inc | Processes for production of tumor infiltrating lymphocytes and uses of the same in immunotherapy |
| JP7854297B2 (ja) | 2018-11-05 | 2026-05-01 | アイオバンス バイオセラピューティクス,インコーポレイテッド | 改良された腫瘍反応性t細胞の選択 |
| US12611427B2 (en) | 2018-11-05 | 2026-04-28 | Iovance Biotherapeutics, Inc. | Treatment of NSCLC patients refractory for anti-PD-1 antibody |
| WO2020096927A1 (en) | 2018-11-05 | 2020-05-14 | Iovance Biotherapeutics, Inc. | Expansion of tils utilizing akt pathway inhibitors |
| WO2020097445A1 (en) | 2018-11-09 | 2020-05-14 | Inari Agriculture, Inc. | Rna-guided nucleases and dna binding proteins |
| 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 |
| US20220282275A1 (en) | 2018-11-15 | 2022-09-08 | The Broad Institute, Inc. | G-to-t base editors and uses thereof |
| KR20220022107A (ko) | 2018-12-05 | 2022-02-24 | 아베오나 테라퓨틱스 인코퍼레이티드 | 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터 |
| US11166996B2 (en) | 2018-12-12 | 2021-11-09 | Flagship Pioneering Innovations V, Inc. | Anellovirus compositions and methods of use |
| WO2020123887A2 (en) | 2018-12-14 | 2020-06-18 | Pioneer Hi-Bred International, Inc. | Novel crispr-cas systems for genome editing |
| US12264323B2 (en) | 2018-12-17 | 2025-04-01 | The Broad Institute, Inc. | CRISPR CPF1 direct repeat variants |
| CN113544266A (zh) | 2018-12-17 | 2021-10-22 | 博德研究所 | Crispr相关转座酶系统和其使用方法 |
| US20220062394A1 (en) | 2018-12-17 | 2022-03-03 | The Broad Institute, Inc. | Methods for identifying neoantigens |
| JP2022515078A (ja) | 2018-12-18 | 2022-02-17 | ブラスケム エス.エー. | マロン酸セミアルデヒドからの3-HPおよびアセチル-CoA誘導体の共産生経路 |
| CA3123392A1 (en) | 2018-12-19 | 2020-06-25 | Iovance Biotherapeutics, Inc. | Methods of expanding tumor infiltrating lymphocytes using engineered cytokine receptor pairs and uses thereof |
| US12390812B2 (en) | 2018-12-19 | 2025-08-19 | Nuclein, Llc | Apparatus and methods for molecular diagnostics |
| US20220017911A1 (en) | 2018-12-21 | 2022-01-20 | Pivot Bio, Inc. | Methods, compositions, and media for improving plant traits |
| WO2020132737A2 (en) | 2018-12-28 | 2020-07-02 | Braskem S.A. | Modulation of carbon flux through the meg and c3 pathways for the improved production of monoethylene glycol and c3 compounds |
| CN113574386A (zh) | 2019-01-03 | 2021-10-29 | 国家医疗保健研究所 | 用于增强癌症患者的cd8+t细胞依赖性免疫应答的方法和药物组合物 |
| EP3931313A2 (en) | 2019-01-04 | 2022-01-05 | Mammoth Biosciences, Inc. | Programmable nuclease improvements and compositions and methods for nucleic acid amplification and detection |
| US11739156B2 (en) | 2019-01-06 | 2023-08-29 | The Broad Institute, Inc. Massachusetts Institute of Technology | Methods and compositions for overcoming immunosuppression |
| AU2020206751A1 (en) | 2019-01-07 | 2021-07-01 | Pivot Bio, Inc. | Plant colonization assays using natural microbial barcodes |
| WO2020148206A1 (en) | 2019-01-14 | 2020-07-23 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and kits for generating and selecting a variant of a binding protein with increased binding affinity and/or specificity |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| US12509453B2 (en) | 2019-01-29 | 2025-12-30 | Foghorn Therapeutics Inc. | BRM/BRG1 inhibitors and uses thereof |
| US12384776B2 (en) | 2019-01-29 | 2025-08-12 | Foghorn Therapeutics Inc. | Compounds and uses thereof |
| US12600943B2 (en) | 2019-02-01 | 2026-04-14 | The University Of Hong Kong | Innervated organoid compositions and methods of making same |
| US11946040B2 (en) | 2019-02-04 | 2024-04-02 | The General Hospital Corporation | Adenine DNA base editor variants with reduced off-target RNA editing |
| TW202045190A (zh) * | 2019-02-06 | 2020-12-16 | 美商蒂克利爾公司 | 自包含錯合劑之眼用調配物移除保存劑之系統及方法 |
| CA3128507A1 (en) | 2019-02-06 | 2020-08-13 | Fred Hutchinson Cancer Research Center | Minicircle producing bacteria engineered to differentially methylate nucleic acid molecules therein |
| WO2020163856A1 (en) | 2019-02-10 | 2020-08-13 | The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone | Modified mitochondrion and methods of use thereof |
| AU2020229340A1 (en) | 2019-02-26 | 2021-09-16 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of B-sarcoglycan and the treatment of muscular dystrophy |
| WO2020180733A1 (en) | 2019-03-01 | 2020-09-10 | Iovance Biotherapeutics, Inc. | Expansion of tumor infiltrating lymphocytes from liquid tumors and therapeutic uses thereof |
| WO2020176958A1 (en) | 2019-03-01 | 2020-09-10 | Braskem S.A. | Method for the in vivo synthesis of 4-hydroxymethylfurfural and derivatives thereof |
| CN110177061B (zh) * | 2019-03-01 | 2023-04-07 | 致讯科技(天津)有限公司 | 一种异构网络中信号干扰的协调方法 |
| WO2020181178A1 (en) | 2019-03-06 | 2020-09-10 | The Broad Institute, Inc. | T:a to a:t base editing through thymine alkylation |
| WO2020181180A1 (en) | 2019-03-06 | 2020-09-10 | The Broad Institute, Inc. | A:t to c:g base editors and uses thereof |
| WO2020181193A1 (en) | 2019-03-06 | 2020-09-10 | The Broad Institute, Inc. | T:a to a:t base editing through adenosine methylation |
| WO2020181195A1 (en) | 2019-03-06 | 2020-09-10 | The Broad Institute, Inc. | T:a to a:t base editing through adenine excision |
| WO2020181202A1 (en) | 2019-03-06 | 2020-09-10 | The Broad Institute, Inc. | A:t to t:a base editing through adenine deamination and oxidation |
| MX2021010559A (es) | 2019-03-07 | 2021-12-15 | Univ California | Polipéptidos efectores de crispr-cas y métodos de uso de estos. |
| MX2021010840A (es) | 2019-03-08 | 2022-01-19 | Obsidian Therapeutics Inc | Composiciones de anhidrasa carbónica 2 (ca2) humana y métodos de regulación ajustable. |
| CN113728106A (zh) | 2019-03-08 | 2021-11-30 | 齐默尔根公司 | 微生物中的迭代基因组编辑 |
| US11053515B2 (en) | 2019-03-08 | 2021-07-06 | Zymergen Inc. | Pooled genome editing in microbes |
| US12534714B2 (en) | 2019-03-18 | 2026-01-27 | The Broad Institute, Inc. | Type VII CRISPR proteins and systems |
| EP3769090B1 (en) | 2019-03-18 | 2023-11-15 | Regeneron Pharmaceuticals, Inc. | Crispr/cas dropout screening platform to reveal genetic vulnerabilities associated with tau aggregation |
| CN120648640A (zh) | 2019-03-18 | 2025-09-16 | 瑞泽恩制药公司 | 用于鉴定tau接种或聚集的基因修饰因子的CRISPR/Cas筛选平台 |
| WO2020190363A1 (en) | 2019-03-19 | 2020-09-24 | Massachusetts Institute Of Technology | Control of nitrogen fixation in rhizobia that associate with cereals |
| WO2020191233A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| WO2020206036A1 (en) | 2019-04-01 | 2020-10-08 | The Broad Institute, Inc. | Novel nucleic acid modifier |
| RU2710731C1 (ru) * | 2019-04-02 | 2020-01-10 | Общество с ограниченной ответственностью "Зеленые линии" | Система редактирования генома дрожжей debaryomyces hansenii на основе crispr/cas9 |
| EP3935154A1 (en) | 2019-04-04 | 2022-01-12 | Braskem S.A. | Metabolic engineering for simultaneous consumption of xylose and glucose for production of chemicals from second generation sugars |
| JP7641233B2 (ja) * | 2019-04-12 | 2025-03-06 | アストラゼネカ・アクチエボラーグ | 改善された遺伝子編集のための組成物及び方法 |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CA3137382A1 (en) | 2019-04-23 | 2020-10-29 | Genedit Inc. | Cationic polymer with alkyl side chains |
| EP3959320A1 (en) | 2019-04-24 | 2022-03-02 | Novartis AG | Compositions and methods for selective protein degradation |
| EP3959229A1 (en) | 2019-04-24 | 2022-03-02 | Pivot Bio, Inc. | Gene targets for nitrogen fixation targeting for improving plant traits |
| CA3129539A1 (en) | 2019-04-25 | 2020-10-29 | Min-Hyung RYU | High-throughput methods for isolating and characterizing ammonium-excreting mutant libraries generated by chemical mutagenesis |
| EP3966334A1 (en) | 2019-05-10 | 2022-03-16 | Basf Se | Regulatory nucleic acid molecules for enhancing gene expression in plants |
| US11162079B2 (en) | 2019-05-10 | 2021-11-02 | The Regents Of The University Of California | Blood type O Rh-hypo-immunogenic pluripotent cells |
| WO2020232029A1 (en) | 2019-05-13 | 2020-11-19 | Iovance Biotherapeutics, Inc. | Methods and compositions for selecting tumor infiltrating lymphocytes and uses of the same in immunotherapy |
| WO2020229533A1 (en) | 2019-05-13 | 2020-11-19 | KWS SAAT SE & Co. KGaA | Drought tolerance in corn |
| US12116583B2 (en) | 2019-05-14 | 2024-10-15 | Yield10 Bioscience, Inc. | Modified plants comprising a polynucleotide comprising a non-cognate promoter operably linked to a coding sequence that encodes a transcription factor |
| WO2020236972A2 (en) | 2019-05-20 | 2020-11-26 | The Broad Institute, Inc. | Non-class i multi-component nucleic acid targeting systems |
| US20220235340A1 (en) | 2019-05-20 | 2022-07-28 | The Broad Institute, Inc. | Novel crispr-cas systems and uses thereof |
| US20220249697A1 (en) | 2019-05-20 | 2022-08-11 | The Broad Institute, Inc. | Aav delivery of nucleobase editors |
| JP2022533713A (ja) | 2019-05-21 | 2022-07-25 | サンガモ セラピューティクス, インコーポレイテッド | 調節性t細胞における制御された導入遺伝子発現 |
| AR118995A1 (es) | 2019-05-25 | 2021-11-17 | Kws Saat Se & Co Kgaa | Mejorador de la inducción de haploides |
| US20220340712A1 (en) | 2019-05-28 | 2022-10-27 | GenEdit, Inc. | Polymer comprising multiple functionalized sidechains for biomolecule delivery |
| BR112021021149A2 (pt) | 2019-05-29 | 2021-12-14 | Monsanto Technology Llc | Métodos e composições para a geração de alelos dominantes utilizando a edição de genomas |
| US12497597B2 (en) | 2019-05-31 | 2025-12-16 | Children's Hospital Medical Center | Methods of generating and expanding hematopoietic stem cells |
| AU2020283048A1 (en) | 2019-05-31 | 2021-12-23 | Children's Hospital Medical Center | Shaped organoid compositions and methods of making same |
| WO2020243661A1 (en) | 2019-05-31 | 2020-12-03 | The Broad Institute, Inc. | Methods for treating metabolic disorders by targeting adcy5 |
| JP7644722B2 (ja) * | 2019-06-01 | 2025-03-12 | シベック バイオテクノロジーズ,インコーポレイティド | 真核細胞への遺伝子編集システムの送達のための細菌プラットフォーム |
| WO2020244759A1 (en) | 2019-06-05 | 2020-12-10 | Klemm & Sohn Gmbh & Co. Kg | New plants having a white foliage phenotype |
| CN113906134B (zh) | 2019-06-14 | 2025-06-24 | 瑞泽恩制药公司 | Tau蛋白病模型 |
| WO2020254850A1 (en) | 2019-06-21 | 2020-12-24 | Vilmorin & Cie | Improvement of quality and permanence of green color of peppers at maturity and over-maturity |
| CN114450326B (zh) | 2019-06-24 | 2025-02-11 | 普罗美加公司 | 用于将生物分子递送到细胞中的改性聚胺聚合物 |
| CN114630910A (zh) | 2019-06-25 | 2022-06-14 | 伊纳瑞农业技术有限公司 | 改善的同源依赖修复基因组编辑 |
| SG11202111943UA (en) | 2019-07-02 | 2021-11-29 | Hutchinson Fred Cancer Res | Recombinant ad35 vectors and related gene therapy improvements |
| GB201909597D0 (en) | 2019-07-03 | 2019-08-14 | Univ Wageningen | Crispr type v-u1 system from mycobacterium mucogenicum and uses thereof |
| AU2020311897A1 (en) | 2019-07-08 | 2022-02-03 | Research Institute At Nationwide Children's Hospital | Antibody compositions for disrupting biofilms |
| WO2021009299A1 (en) | 2019-07-17 | 2021-01-21 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Bcl-xl:fkbp12 fusion proteins suitable for screening agents capable of slowing down the aging process |
| CN110387405A (zh) * | 2019-07-17 | 2019-10-29 | 浙江善测禾骑士生物科技有限公司 | 一种快速检测核酸的(rt)raa-crispr系统 |
| CN110452966A (zh) * | 2019-07-17 | 2019-11-15 | 浙江善测禾骑士生物科技有限公司 | 一种利用raa-crispr蛋白酶系统快速检测方法 |
| EP3999103A4 (en) | 2019-07-19 | 2023-11-22 | Inari Agriculture Technology, Inc. | GENOMIC EDITING FOR ENHANCED HOMOLOGY-DIRECTED REPAIR |
| WO2021019272A1 (en) | 2019-07-31 | 2021-02-04 | Vilmorin & Cie | Tolerance to tolcndv in cucumber |
| WO2021026294A1 (en) | 2019-08-06 | 2021-02-11 | Children's Medical Center Corporation | Methods and compositions for reconstituting microglia |
| 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 |
| WO2021028359A1 (en) | 2019-08-09 | 2021-02-18 | Sangamo Therapeutics France | Controlled expression of chimeric antigen receptors in t cells |
| WO2021030666A1 (en) | 2019-08-15 | 2021-02-18 | The Broad Institute, Inc. | Base editing by transglycosylation |
| EP4013859A4 (en) * | 2019-08-15 | 2023-10-11 | The Rockefeller University | CRISPR-BASED GENOME EDITING WITH CELL SURFACE DISPLAY TO PRODUCE HOMOZYGOTICLY ENGINEERED EUKARYOTIC CELLS |
| RS65421B1 (sr) | 2019-08-21 | 2024-05-31 | Res Inst Nationwide Childrens Hospital | Isporuka adeno-asociranog virusnog vektora alfa-sarkoglikana i lečenje mišićne distrofije |
| WO2021041922A1 (en) | 2019-08-30 | 2021-03-04 | The Broad Institute, Inc. | Crispr-associated mu transposase systems |
| WO2021046451A1 (en) | 2019-09-06 | 2021-03-11 | Obsidian Therapeutics, Inc. | Compositions and methods for dhfr tunable protein regulation |
| EP4028047A4 (en) | 2019-09-09 | 2023-07-26 | Arbor Biotechnologies, Inc. | Novel crispr dna targeting enzymes and systems |
| CN114729382A (zh) | 2019-09-12 | 2022-07-08 | 巴斯夫欧洲公司 | 增强植物中基因表达的调节性核酸分子 |
| CN115175996A (zh) | 2019-09-20 | 2022-10-11 | 博德研究所 | 新颖vi型crispr酶和系统 |
| CN110541001A (zh) * | 2019-09-20 | 2019-12-06 | 福建上源生物科学技术有限公司 | 精确大片段基因删除结合终止密码子插入的基因敲除法 |
| EP4048807A1 (en) | 2019-09-23 | 2022-08-31 | Omega Therapeutics, Inc. | Compositions and methods for modulating apolipoprotein b (apob) gene expression |
| CN114729376A (zh) | 2019-09-23 | 2022-07-08 | 欧米茄治疗公司 | 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法 |
| WO2021062196A1 (en) | 2019-09-26 | 2021-04-01 | President And Fellows Of Harvard College | Minimal arrestin domain containing protein 1 (arrdc1) constructs |
| 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 |
| WO2021069387A1 (en) | 2019-10-07 | 2021-04-15 | Basf Se | Regulatory nucleic acid molecules for enhancing gene expression in plants |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| EP3808766A1 (en) | 2019-10-15 | 2021-04-21 | Sangamo Therapeutics France | Chimeric antigen receptor specific for interleukin-23 receptor |
| EP4045522A1 (en) | 2019-10-17 | 2022-08-24 | KWS SAAT SE & Co. KGaA | Enhanced disease resistance of crops by downregulation of repressor genes |
| EP4048295A1 (en) | 2019-10-25 | 2022-08-31 | Iovance Biotherapeutics, Inc. | Gene editing of tumor infiltrating lymphocytes and uses of same in immunotherapy |
| WO2021094805A1 (en) | 2019-11-14 | 2021-05-20 | Vilmorin & Cie | Resistance to acidovorax valerianellae in corn salad |
| CA3158963A1 (en) | 2019-11-22 | 2021-05-27 | Samir Mitragotri | Ionic liquids for drug delivery |
| US20230086199A1 (en) | 2019-11-26 | 2023-03-23 | The Broad Institute, Inc. | Systems and methods for evaluating cas9-independent off-target editing of nucleic acids |
| CN114945273B (zh) | 2019-11-29 | 2025-08-12 | 巴斯夫欧洲公司 | 增加植物对抗真菌感染的抗性 |
| WO2021111466A1 (en) * | 2019-12-02 | 2021-06-10 | Council Of Scientific & Industrial Research | Method and kit for detection of polynucleotide |
| CA3159805A1 (en) | 2019-12-03 | 2021-06-10 | Frank Meulewaeter | Regulatory nucleic acid molecules for enhancing gene expression in plants |
| BR112022011372A2 (pt) | 2019-12-10 | 2022-08-23 | Univ Indiana Trustees | Inibidores de interação de proteína a de replicação (rpa)-dna |
| US12516291B2 (en) | 2019-12-11 | 2026-01-06 | Iovance Biotherapeutics, Inc. | Processes for the production of tumor infiltrating lymphocytes (TILs) and methods of using the same |
| CN120683228A (zh) | 2019-12-13 | 2025-09-23 | 中外制药株式会社 | 检测细胞外嘌呤受体配体的系统和导入该系统的非人动物 |
| EP4058032A4 (en) | 2019-12-19 | 2024-01-10 | Entrada Therapeutics, Inc. | Compositions for delivery of antisense compounds |
| EP4077699A1 (en) | 2019-12-19 | 2022-10-26 | Basf Se | Increasing space-time-yield, carbon-conversion-efficiency and carbon substrate flexibility in the production of fine chemicals |
| US20230041211A1 (en) | 2019-12-20 | 2023-02-09 | Basf Se | Decreasing toxicity of terpenes and increasing the production potential in micro-organisms |
| WO2021146222A1 (en) | 2020-01-13 | 2021-07-22 | Sana Biotechnology, Inc. | Modification of blood type antigens |
| WO2021221690A1 (en) | 2020-05-01 | 2021-11-04 | Pivot Bio, Inc. | Modified bacterial strains for improved fixation of nitrogen |
| WO2021150919A1 (en) | 2020-01-23 | 2021-07-29 | The Children's Medical Center Corporation | Stroma-free t cell differentiation from human pluripotent stem cells |
| WO2021151073A2 (en) | 2020-01-24 | 2021-07-29 | The General Hospital Corporation | Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants |
| WO2021151085A2 (en) | 2020-01-24 | 2021-07-29 | The General Hospital Corporation | Crispr-cas enzymes with enhanced on-target activity |
| EP4097251A1 (en) | 2020-01-29 | 2022-12-07 | 10X Genomics, Inc. | Compositions and methods for analyte detection |
| AU2021213811A1 (en) | 2020-01-29 | 2022-07-28 | Foghorn Therapeutics Inc. | Compounds and uses thereof |
| EP4100519A2 (en) | 2020-02-05 | 2022-12-14 | The Broad Institute, Inc. | Adenine base editors and uses thereof |
| US12116580B2 (en) | 2020-02-21 | 2024-10-15 | The Board Of Trustees Of The University Of Illinois | Genetic toolbox for metabolic engineering of non-conventional yeast |
| EP3872190A1 (en) | 2020-02-26 | 2021-09-01 | Antibodies-Online GmbH | A method of using cut&run or cut&tag to validate crispr-cas targeting |
| CA3173528A1 (en) | 2020-03-11 | 2021-09-16 | Omega Therapeutics, Inc. | Compositions and methods for modulating forkhead box p3 (foxp3) gene expression |
| WO2021186056A1 (en) | 2020-03-20 | 2021-09-23 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Chimeric antigen receptor specific for human cd45rc and uses thereof |
| US12057197B2 (en) | 2020-04-03 | 2024-08-06 | Creyon Bio, Inc. | Oligonucleotide-based machine learning |
| WO2021216623A1 (en) | 2020-04-21 | 2021-10-28 | Aspen Neuroscience, Inc. | Gene editing of lrrk2 in stem cells and method of use of cells differentiated therefrom |
| US20250263752A1 (en) | 2020-04-21 | 2025-08-21 | Aspen Neuroscience, Inc. | Gene editing of gba1 in stem cells and method of use of cells differentiated therefrom |
| WO2021217082A1 (en) | 2020-04-23 | 2021-10-28 | Genedit Inc. | Polymer with cationic and hydrophobic side chains |
| WO2021222567A2 (en) | 2020-05-01 | 2021-11-04 | Pivot Bio, Inc. | Modified bacterial strains for improved fixation of nitrogen |
| CA3172321A1 (en) | 2020-05-01 | 2021-11-04 | Farzaneh REZAEI | Stable liquid formulations for nitrogen-fixing microorganisms |
| AU2020445067A1 (en) | 2020-05-01 | 2022-12-01 | Pivot Bio, Inc. | Measurement of nitrogen fixation and incorporation |
| CA3176826A1 (en) | 2020-05-04 | 2021-11-11 | Iovance Biotherapeutics, Inc. | Processes for production of tumor infiltrating lymphocytes and uses of the same in immunotherapy |
| EP4146797A1 (en) | 2020-05-06 | 2023-03-15 | Orchard Therapeutics (Europe) Limited | Treatment for neurodegenerative diseases |
| IL297761A (en) | 2020-05-08 | 2022-12-01 | Broad Inst Inc | Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence |
| EP4149245A2 (en) | 2020-05-13 | 2023-03-22 | Pivot Bio, Inc. | De-repression of nitrogen fixation in gram-positive microorganisms |
| US12383555B2 (en) | 2020-05-20 | 2025-08-12 | Foghorn Therapeutics Inc. | Methods of treating cancers |
| CN115698335A (zh) | 2020-05-22 | 2023-02-03 | 因斯特罗公司 | 使用机器学习模型预测疾病结果 |
| GB202007943D0 (en) | 2020-05-27 | 2020-07-08 | Snipr Biome Aps | Products & methods |
| AR122206A1 (es) | 2020-05-29 | 2022-08-24 | Kws Saat Se & Co Kgaa | Inducción de haploides en plantas |
| WO2021245435A1 (en) | 2020-06-03 | 2021-12-09 | Vilmorin & Cie | Melon plants resistant to scab disease, aphids and powdery mildew |
| AU2021285274A1 (en) | 2020-06-05 | 2023-02-02 | Vilmorin & Cie | Resistance in plants of solanum lycopersicum to the TOBRFV |
| JP2023530234A (ja) | 2020-06-05 | 2023-07-14 | ザ・ブロード・インスティテュート・インコーポレイテッド | 新生物を治療するための組成物および方法 |
| JP2023529211A (ja) | 2020-06-11 | 2023-07-07 | ノバルティス アーゲー | Zbtb32阻害剤及びその使用 |
| WO2022008935A1 (en) | 2020-07-10 | 2022-01-13 | Horizon Discovery Limited | Method for producing genetically modified cells |
| US20230287458A1 (en) | 2020-07-14 | 2023-09-14 | Abeona Therapeutics Inc. | Recombinant adeno-associated viral vectors for multipartite gene delivery |
| EP4189071A1 (en) | 2020-08-03 | 2023-06-07 | Institut National de la Santé et de la Recherche Médicale (INSERM) | Population of treg cells functionally committed to exert a regulatory activity and their use for adoptive therapy |
| WO2022034474A1 (en) | 2020-08-10 | 2022-02-17 | Novartis Ag | Treatments for retinal degenerative diseases |
| US20220049303A1 (en) | 2020-08-17 | 2022-02-17 | Readcoor, Llc | Methods and systems for spatial mapping of genetic variants |
| BR112023002885A2 (pt) | 2020-08-18 | 2023-03-21 | Pioneer Hi Bred Int | Genes de resistência a múltiplas doenças e seus empilhamentos genômicos |
| CA3188867A1 (en) | 2020-08-20 | 2022-02-24 | Xueyin Wang | Compositions and methods for treating ceacam positive cancers |
| WO2022040444A1 (en) | 2020-08-20 | 2022-02-24 | A2 Biotherapeutics, Inc. | Compositions and methods for treating egfr positive cancers |
| IL300500A (en) | 2020-08-20 | 2023-04-01 | A2 Biotherapeutics Inc | Preparations and methods for the treatment of mesothelin positive cancer |
| CN112080587A (zh) * | 2020-08-31 | 2020-12-15 | 上海海关动植物与食品检验检疫技术中心 | 用于高效检测新型冠状病毒的raa-crispr扩增引物组、试剂盒及方法 |
| US11944063B2 (en) | 2020-09-30 | 2024-04-02 | Spring Meadow Nursery, Inc. | Hydrangea ‘SMNHPH’ |
| WO2022188039A1 (en) | 2021-03-09 | 2022-09-15 | Huigene Therapeutics Co., Ltd. | Engineered crispr/cas13 system and uses thereof |
| WO2022068912A1 (en) | 2020-09-30 | 2022-04-07 | Huigene Therapeutics Co., Ltd. | Engineered crispr/cas13 system and uses thereof |
| EP4221492A1 (en) | 2020-10-02 | 2023-08-09 | Vilmorin & Cie | Melon with extended shelf life |
| JP2023546359A (ja) | 2020-10-06 | 2023-11-02 | アイオバンス バイオセラピューティクス,インコーポレイテッド | 腫瘍浸潤リンパ球療法によるnsclc患者の治療 |
| WO2022076606A1 (en) | 2020-10-06 | 2022-04-14 | Iovance Biotherapeutics, Inc. | Treatment of nsclc patients with tumor infiltrating lymphocyte therapies |
| US11155884B1 (en) | 2020-10-16 | 2021-10-26 | Klemm & Sohn Gmbh & Co. Kg | Double-flowering dwarf Calibrachoa |
| EP3984355B1 (en) | 2020-10-16 | 2024-05-15 | Klemm & Sohn GmbH & Co. KG | Double-flowering dwarf calibrachoa |
| US11530419B2 (en) | 2020-10-30 | 2022-12-20 | Fortiphyte, Inc. | Pathogen resistance in plants |
| CA3198447A1 (en) | 2020-11-13 | 2022-05-19 | Novartis Ag | Combination therapies with chimeric antigen receptor (car)-expressing cells |
| EP4001429A1 (en) | 2020-11-16 | 2022-05-25 | Antibodies-Online GmbH | Analysis of crispr-cas binding and cleavage sites followed by high-throughput sequencing (abc-seq) |
| IL303102A (en) | 2020-12-03 | 2023-07-01 | Vilmorin & Cie | Tomato plants resistant to TOMMV, TOMV, TMV, TOBRFV and suitable resistant genes |
| AU2021401302A1 (en) | 2020-12-17 | 2023-07-06 | Iovance Biotherapeutics, Inc. | Treatment with tumor infiltrating lymphocyte therapies in combination with ctla-4 and pd-1 inhibitors |
| EP4262827A1 (en) | 2020-12-17 | 2023-10-25 | Iovance Biotherapeutics, Inc. | Treatment of cancers with tumor infiltrating lymphocytes |
| TW202235622A (zh) | 2020-12-23 | 2022-09-16 | 美商旗艦先鋒創新公司 | 包裹rna之指環病毒(anellovirus)蛋白殼之活體外組裝 |
| CA3207144A1 (en) | 2021-01-05 | 2022-07-14 | Horizon Discovery Limited | Method for producing genetically modified cells |
| WO2022150776A1 (en) * | 2021-01-11 | 2022-07-14 | Vedere Bio Ii, Inc. | OPTOGENETIC COMPOSITIONS COMPRISING A CBh PROMOTER SEQUENCE AND METHODS FOR USE |
| JP2024502630A (ja) | 2021-01-12 | 2024-01-22 | マーチ セラピューティクス, インコーポレイテッド | コンテキスト依存性二本鎖dna特異的デアミナーゼ及びその使用 |
| WO2022165260A1 (en) | 2021-01-29 | 2022-08-04 | Iovance Biotherapeutics, Inc. | Methods of making modified tumor infiltrating lymphocytes and their use in adoptive cell therapy |
| US12171813B2 (en) | 2021-02-05 | 2024-12-24 | Christiana Care Gene Editing Institute, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| WO2022177979A1 (en) | 2021-02-16 | 2022-08-25 | A2 Biotherapeutics, Inc. | Compositions and methods for treating her2 positive cancers |
| US12606553B2 (en) | 2021-03-09 | 2026-04-21 | Foghorn Therapeutics Inc. | Crystalline forms, compositions containing same, and methods of their use |
| EP4305157A1 (en) | 2021-03-09 | 2024-01-17 | Huidagene Therapeutics (Singapore) Pte. Ltd. | Engineered crispr/cas13 system and uses thereof |
| US20240191191A1 (en) | 2021-03-19 | 2024-06-13 | Iovance Biotherapeutics, Inc. | Methods for infiltrating lymphocyte (til) expansion related to cd39/cd69 selection and gene knockout in tils |
| JP2024512029A (ja) | 2021-03-25 | 2024-03-18 | アイオバンス バイオセラピューティクス,インコーポレイテッド | T細胞共培養効力アッセイのための方法及び組成物、ならびに細胞療法製品との使用 |
| EP4314016A1 (en) | 2021-03-31 | 2024-02-07 | Entrada Therapeutics, Inc. | Cyclic cell penetrating peptides |
| WO2022208489A1 (en) | 2021-04-02 | 2022-10-06 | Vilmorin & Cie | Semi-determinate or determinate growth habit trait in cucurbita |
| KR20240007651A (ko) | 2021-04-16 | 2024-01-16 | 빔 테라퓨틱스, 인크. | 간세포의 유전적 변형 |
| CA3215830A1 (en) | 2021-04-19 | 2022-10-27 | Rafael CUBAS | Chimeric costimulatory receptors, chemokine receptors, and the use of same in cellular immunotherapies |
| WO2022235929A1 (en) | 2021-05-05 | 2022-11-10 | Radius Pharmaceuticals, Inc. | Animal model having homologous recombination of mouse pth1 receptor |
| AU2022271873A1 (en) | 2021-05-10 | 2024-01-04 | Entrada Therapeutics, Inc. | Compositions and methods for intracellular therapeutics |
| WO2022240760A2 (en) | 2021-05-10 | 2022-11-17 | Entrada Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR MODULATING mRNA SPLICING |
| EP4337263A1 (en) | 2021-05-10 | 2024-03-20 | Entrada Therapeutics, Inc. | Compositions and methods for modulating interferon regulatory factor-5 (irf-5) activity |
| US20240352456A1 (en) | 2021-05-13 | 2024-10-24 | The United States Of America, As Represented By The Secretary, Department Of Health & Human Services | Compositions and methods for treating sickle cell diseases |
| JP2024519029A (ja) | 2021-05-17 | 2024-05-08 | アイオバンス バイオセラピューティクス,インコーポレイテッド | Pd-1遺伝子編集された腫瘍浸潤リンパ球及び免疫療法におけるその使用 |
| EP4347818A2 (en) | 2021-06-01 | 2024-04-10 | Arbor Biotechnologies, Inc. | Gene editing systems comprising a crispr nuclease and uses thereof |
| WO2022261509A1 (en) | 2021-06-11 | 2022-12-15 | The Broad Institute, Inc. | Improved cytosine to guanine base editors |
| US20240285781A1 (en) | 2021-06-11 | 2024-08-29 | GenEdit, Inc. | Biodegradable polymer comprising side chains with polyamine and polyalkylene oxide groups |
| WO2022266105A1 (en) * | 2021-06-14 | 2022-12-22 | The University Of Chicago | Characterization and treatment of asthma |
| AU2022298774A1 (en) | 2021-06-23 | 2023-12-14 | Entrada Therapeutics, Inc. | Antisense compounds and methods for targeting cug repeats |
| EP4362661A1 (en) | 2021-07-02 | 2024-05-08 | Pivot Bio, Inc. | Genetically-engineered bacterial strains for improved fixation of nitrogen |
| EP4367242A2 (en) | 2021-07-07 | 2024-05-15 | Omega Therapeutics, Inc. | Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression |
| EP4373270A2 (en) | 2021-07-22 | 2024-05-29 | Iovance Biotherapeutics, Inc. | Method for cryopreservation of solid tumor fragments |
| JP2024527961A (ja) | 2021-07-28 | 2024-07-26 | アイオバンス バイオセラピューティクス,インコーポレイテッド | Kras阻害剤と併用した腫瘍浸潤リンパ球療法によるがん患者の治療 |
| WO2023006933A1 (en) | 2021-07-30 | 2023-02-02 | KWS SAAT SE & Co. KGaA | Plants with improved digestibility and marker haplotypes |
| EP4424698A3 (en) | 2021-07-30 | 2024-11-27 | Helaina, Inc. | Methods and compositions for protein synthesis and secretion |
| MX2024001624A (es) | 2021-08-06 | 2024-04-30 | Vilmorin & Cie | Resistencia a leveillula taurica en el pimiento. |
| EP4396357A1 (en) | 2021-09-02 | 2024-07-10 | SESVanderHave NV | Methods and compositions for ppo herbicide tolerance |
| JP2024534945A (ja) | 2021-09-10 | 2024-09-26 | アジレント・テクノロジーズ・インク | 化学修飾を有するプライム編集のためのガイドrna |
| EP4596696A3 (en) | 2021-09-28 | 2025-08-27 | Acrigen Biosciences | Compositions and methods for nucleic acid modifications |
| WO2023056367A1 (en) | 2021-09-30 | 2023-04-06 | The Board Of Regents Of The Universityof Texas System | Slc13a5 gene therapy vectors and uses thereof |
| EP4412587A1 (en) | 2021-10-08 | 2024-08-14 | President And Fellows Of Harvard College | Ionic liquids for drug delivery |
| JP2024537991A (ja) | 2021-10-14 | 2024-10-18 | アーセナル バイオサイエンシズ インコーポレイテッド | 共発現されるshRNAと論理ゲートシステムとを有する免疫細胞 |
| 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 |
| WO2023077015A2 (en) | 2021-10-27 | 2023-05-04 | Iovance Biotherapeutics, Inc. | Systems and methods for coordinating manufacturing of cells for patient-specific immunotherapy |
| 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 |
| CA3237482A1 (en) | 2021-11-03 | 2023-05-11 | The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone | Precise genome editing using retrons |
| WO2023081762A2 (en) * | 2021-11-03 | 2023-05-11 | The Regents Of The University Of California | Serine recombinases |
| CA3237410A1 (en) | 2021-11-10 | 2023-05-19 | Friedrich Graf Finck VON FINCKENSTEIN | Methods of expansion treatment utilizing cd8 tumor infiltrating lymphocytes |
| US20250011781A1 (en) * | 2021-11-22 | 2025-01-09 | The Texas A&M University System | Methods and compositions targeting nucleus accumbens-associated protein-1 for treatment of autoimmune disorders and cancers |
| US20250295718A1 (en) | 2021-11-24 | 2025-09-25 | Research Institute At Nationwide Children's Hospital | Chimeric hsv expressing hil21 to boost anti-tumor immune activity |
| WO2023093862A1 (en) | 2021-11-26 | 2023-06-01 | Epigenic Therapeutics Inc. | Method of modulating pcsk9 and uses thereof |
| WO2023102406A1 (en) | 2021-12-01 | 2023-06-08 | The Board Of Regents Of The Univesity Of Texas System | Vector genome design to express optimized cln7 transgene |
| WO2023102518A1 (en) | 2021-12-03 | 2023-06-08 | The Board Of Regents Of The University Of Texas System | Gnao1 gene therapy vectors and uses thereof |
| GB202118058D0 (en) | 2021-12-14 | 2022-01-26 | Univ Warwick | Methods to increase yields in crops |
| US20230279442A1 (en) | 2021-12-15 | 2023-09-07 | Versitech Limited | Engineered cas9-nucleases and method of use thereof |
| WO2023115041A1 (en) | 2021-12-17 | 2023-06-22 | Sana Biotechnology, Inc. | Modified paramyxoviridae attachment glycoproteins |
| US20250059239A1 (en) | 2021-12-17 | 2025-02-20 | Sana Biotechnology, Inc. | Modified paramyxoviridae fusion glycoproteins |
| WO2023122805A1 (en) | 2021-12-20 | 2023-06-29 | Vestaron Corporation | Sorbitol driven selection pressure method |
| AU2022420615A1 (en) | 2021-12-22 | 2024-07-04 | Tome Biosciences, Inc. | Co-delivery of a gene editor construct and a donor template |
| JP2025501755A (ja) | 2021-12-23 | 2025-01-23 | ユニバーシティー オブ マサチューセッツ | 脆弱x関連障害に対する治療的処置 |
| EP4463135A2 (en) | 2022-01-10 | 2024-11-20 | Sana Biotechnology, Inc. | Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses |
| WO2023141602A2 (en) | 2022-01-21 | 2023-07-27 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
| WO2023147476A1 (en) | 2022-01-28 | 2023-08-03 | The Board Of Regents Of The University Of Texas System | Transgene casette designed to express the human codon-optimized gene fmr1 |
| EP4469065A1 (en) | 2022-01-28 | 2024-12-04 | Iovance Biotherapeutics, Inc. | Cytokine associated tumor infiltrating lymphocytes compositions and methods |
| EP4472646A1 (en) | 2022-02-01 | 2024-12-11 | Sana Biotechnology, Inc. | Cd3-targeted lentiviral vectors and uses thereof |
| WO2023150647A1 (en) | 2022-02-02 | 2023-08-10 | Sana Biotechnology, Inc. | Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses |
| EP4479143A1 (en) | 2022-02-18 | 2024-12-25 | Institut National de la Santé et de la Recherche Médicale (INSERM) | Use of tcr-deficient car-tregs in combination with anti-tcr complex monoclonal antibodies for inducing durable tolerance |
| WO2023196802A1 (en) | 2022-04-04 | 2023-10-12 | The Broad Institute, Inc. | Cas9 variants having non-canonical pam specificities and uses thereof |
| WO2023196877A1 (en) | 2022-04-06 | 2023-10-12 | Iovance Biotherapeutics, Inc. | Treatment of nsclc patients with tumor infiltrating lymphocyte therapies |
| EP4256950A1 (en) | 2022-04-06 | 2023-10-11 | Vilmorin et Cie | Tolerance to cgmmv in cucumber |
| JP2025512401A (ja) | 2022-04-15 | 2025-04-17 | アイオバンス バイオセラピューティクス,インコーポレイテッド | 特定のサイトカインの組み合わせ及び/またはAKTi処理を使用したTIL拡張プロセス |
| WO2023205744A1 (en) | 2022-04-20 | 2023-10-26 | Tome Biosciences, Inc. | Programmable gene insertion compositions |
| JP2025515503A (ja) | 2022-04-28 | 2025-05-15 | ザ ブロード インスティテュート,インコーポレーテッド | 塩基エディタをコードするaavベクターおよびその使用 |
| WO2023215831A1 (en) | 2022-05-04 | 2023-11-09 | Tome Biosciences, Inc. | Guide rna compositions for programmable gene insertion |
| WO2023220040A1 (en) | 2022-05-09 | 2023-11-16 | Synteny Therapeutics, Inc. | Erythroparvovirus with a modified capsid for gene therapy |
| US20250302994A1 (en) | 2022-05-09 | 2025-10-02 | Synteny Therapeutics, Inc. | Erythroparvovirus with a modified genome for gene therapy |
| EP4522201A1 (en) | 2022-05-09 | 2025-03-19 | Synteny Therapeutics, Inc. | Erythroparvovirus compositions and methods for gene therapy |
| WO2023219933A1 (en) | 2022-05-09 | 2023-11-16 | Entrada Therapeutics, Inc. | Compositions and methods for delivery of nucleic acid therapeutics |
| CA3251533A1 (en) | 2022-05-10 | 2023-11-16 | Iovance Biotherapeutics, Inc. | TREATMENT OF CANCER PATIENTS WITH TUMOR-INFILTRATING LYMPHOCYTE THERAPIES IN COMBINATION WITH AN IL-15R AGONIST |
| WO2023225670A2 (en) | 2022-05-20 | 2023-11-23 | Tome Biosciences, Inc. | Ex vivo programmable gene insertion |
| CN119563038A (zh) | 2022-05-25 | 2025-03-04 | 旗舰创业创新七公司 | 用于调节免疫应答的组合物和方法 |
| AU2023276757A1 (en) | 2022-05-25 | 2024-11-21 | Flagship Pioneering Innovations Vii, Llc | Compositions and methods for modulation of tumor suppressors and oncogenes |
| WO2023230570A2 (en) | 2022-05-25 | 2023-11-30 | Flagship Pioneering Innovations Vii, Llc | Compositions and methods for modulating genetic drivers |
| CA3254388A1 (en) | 2022-05-25 | 2023-11-30 | Flagship Pioneering Innovations Vii, Llc | COMPOSITIONS AND METHODS OF MODULATING TRAFFIC FACTORS |
| US20250319115A1 (en) | 2022-05-25 | 2025-10-16 | Flagship Pioneering Innovations Vii, Llc | Compositions and Methods for Modulating Cytokines |
| IL317874A (en) | 2022-06-24 | 2025-02-01 | Tune Therapeutics Inc | Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression |
| GB202209518D0 (en) | 2022-06-29 | 2022-08-10 | Snipr Biome Aps | Treating & preventing E coli infections |
| WO2024005864A1 (en) | 2022-06-30 | 2024-01-04 | Inari Agriculture Technology, Inc. | Compositions, systems, and methods for genome editing |
| WO2024005863A1 (en) | 2022-06-30 | 2024-01-04 | Inari Agriculture Technology, Inc. | Compositions, systems, and methods for genome editing |
| EP4299733A1 (en) | 2022-06-30 | 2024-01-03 | Inari Agriculture Technology, Inc. | Compositions, systems, and methods for genome editing |
| GB2621813A (en) | 2022-06-30 | 2024-02-28 | Univ Newcastle | Preventing disease recurrence in Mitochondrial replacement therapy |
| EP4548351A1 (en) | 2022-06-30 | 2025-05-07 | Pioneer Hi-Bred International, Inc. | Artificial intelligence-mediated methods and systems for genome editing |
| EP4299739A1 (en) | 2022-06-30 | 2024-01-03 | Inari Agriculture Technology, Inc. | Compositions, systems, and methods for genome editing |
| JP2025525569A (ja) | 2022-07-18 | 2025-08-05 | レナゲード セラピューティクス マネージメント インコーポレイテッド | 遺伝子編集成分、システム、及び使用方法 |
| WO2024020587A2 (en) | 2022-07-22 | 2024-01-25 | Tome Biosciences, Inc. | Pleiopluripotent stem cell programmable gene insertion |
| EP4562168A1 (en) | 2022-07-28 | 2025-06-04 | Institut Pasteur | Hsc70-4 in host-induced and spray-induced gene silencing |
| WO2024026406A2 (en) | 2022-07-29 | 2024-02-01 | Vestaron Corporation | Next Generation ACTX Peptides |
| WO2024036190A2 (en) | 2022-08-09 | 2024-02-15 | Pioneer Hi-Bred International, Inc. | Guide polynucleotide multiplexing |
| WO2024040083A1 (en) | 2022-08-16 | 2024-02-22 | The Broad Institute, Inc. | Evolved cytosine deaminases and methods of editing dna using same |
| US20260055426A1 (en) | 2022-08-19 | 2026-02-26 | Generation Bio Co. | CLEAVABLE CLOSED-ENDED DNA (ceDNA) AND METHODS OF USE THEREOF |
| US20260055146A1 (en) | 2022-08-24 | 2026-02-26 | Sana Biotechnology, Inc. | Delivery of heterologous proteins |
| WO2024044723A1 (en) | 2022-08-25 | 2024-02-29 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
| CA3265393A1 (en) | 2022-08-26 | 2024-02-29 | KWS SAAT SE & Co. KGaA | USE OF PAIRED GENES FOR HYBRID SELECTION |
| WO2024050544A2 (en) | 2022-09-01 | 2024-03-07 | J.R. Simplot Company | Enhanced targeted knock-in frequency in host genomes through crispr exonuclease processing |
| WO2024047605A1 (en) | 2022-09-01 | 2024-03-07 | SESVanderHave NV | Methods and compositions for ppo herbicide tolerance |
| EP4583905A1 (en) | 2022-09-06 | 2025-07-16 | Institut National de la Santé et de la Recherche Médicale | Novel dual split car-t cells for the treatment of cd38-positive hematological malignancies |
| WO2024064824A2 (en) | 2022-09-21 | 2024-03-28 | Yale University | Compositions and methods for identification of membrane targets for enhancement of nk cell therapy |
| WO2024064838A1 (en) | 2022-09-21 | 2024-03-28 | Sana Biotechnology, Inc. | Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof |
| WO2024081820A1 (en) | 2022-10-13 | 2024-04-18 | Sana Biotechnology, Inc. | Viral particles targeting hematopoietic stem cells |
| JP7353602B1 (ja) | 2022-10-14 | 2023-10-02 | 株式会社インプランタイノベーションズ | ゲノム編集方法およびゲノム編集用組成物 |
| EP4605524A1 (en) | 2022-10-20 | 2025-08-27 | Basf Se | Regulatory nucleic acid molecules for enhancing gene expression in plants |
| EP4612277A1 (en) | 2022-11-04 | 2025-09-10 | Iovance Biotherapeutics, Inc. | Methods for tumor infiltrating lymphocyte (til) expansion related to cd39/cd103 selection |
| WO2024098024A1 (en) | 2022-11-04 | 2024-05-10 | Iovance Biotherapeutics, Inc. | Expansion of tumor infiltrating lymphocytes from liquid tumors and therapeutic uses thereof |
| WO2024102434A1 (en) | 2022-11-10 | 2024-05-16 | Senda Biosciences, Inc. | Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs |
| JP2025539816A (ja) | 2022-11-21 | 2025-12-09 | アイオバンス バイオセラピューティクス,インコーポレイテッド | 腫瘍浸潤リンパ球の増幅のための2次元プロセス及びそれからの治療法 |
| WO2024118836A1 (en) | 2022-11-30 | 2024-06-06 | Iovance Biotherapeutics, Inc. | Processes for production of tumor infiltrating lymphocytes with shortened rep step |
| WO2024119157A1 (en) | 2022-12-02 | 2024-06-06 | Sana Biotechnology, Inc. | Lipid particles with cofusogens and methods of producing and using the same |
| WO2024123786A1 (en) | 2022-12-06 | 2024-06-13 | Pioneer Hi-Bred International, Inc. | Methods and compositions for co-delivery of t-dnas expressing multiple guide polynucleotides into plants |
| WO2024138194A1 (en) | 2022-12-22 | 2024-06-27 | Tome Biosciences, Inc. | Platforms, compositions, and methods for in vivo programmable gene insertion |
| CN120583887A (zh) | 2022-12-23 | 2025-09-02 | 皮沃特生物股份有限公司 | 各种固氮细菌与各种生物产品的组合用于实现协同效应 |
| CN120417757A (zh) | 2022-12-29 | 2025-08-01 | 维尔莫林公司 | 对抗性突破型tswv毒株具有抗性的番茄植物和相应的抗性基因 |
| WO2024155745A1 (en) | 2023-01-18 | 2024-07-25 | The Broad Institute, Inc. | Base editing-mediated readthrough of premature termination codons (bert) |
| EP4658061A1 (en) | 2023-02-01 | 2025-12-10 | United Beet Seeds | Beet yellows virus resistance |
| WO2024168265A1 (en) | 2023-02-10 | 2024-08-15 | Possible Medicines Llc | Aav delivery of rna guided recombination system |
| WO2024168253A1 (en) | 2023-02-10 | 2024-08-15 | Possible Medicines Llc | Delivery of an rna guided recombination system |
| KR20250151430A (ko) | 2023-02-15 | 2025-10-21 | 아버 바이오테크놀로지스, 인크. | 스타스민 2(stmn2) 전사체에서 비정상적인 스플라이싱을 억제하는 유전자 편집 방법 |
| AU2024239266A1 (en) | 2023-03-20 | 2025-09-25 | Pioneer Hi-Bred International, Inc. | Cas polypeptides with altered pam recognition |
| US12383615B2 (en) | 2023-03-23 | 2025-08-12 | Carbon Biosciences, Inc. | Protoparvovirus compositions comprising a protoparvovirus variant VP1 capsid polypeptide and related methods |
| US12225874B2 (en) | 2023-03-27 | 2025-02-18 | Redsea Science And Technology Inc. | Tomato plant designated ‘X22-31’ |
| WO2024206687A1 (en) * | 2023-03-29 | 2024-10-03 | William Marsh Rice University | Methods and compostions for engineered mitochondrial biogenesis |
| WO2024206911A2 (en) | 2023-03-30 | 2024-10-03 | Children's Hospital Medical Center | Clinical-grade organoids |
| WO2024211287A1 (en) | 2023-04-03 | 2024-10-10 | Seagen Inc. | Production cell lines with targeted integration sites |
| WO2024220719A1 (en) | 2023-04-18 | 2024-10-24 | Crystal Ronald G | Modified aav capsids for gene delivery |
| WO2024220560A1 (en) | 2023-04-18 | 2024-10-24 | Sana Biotechnology, Inc. | Engineered protein g fusogens and related lipid particles and methods thereof |
| EP4698666A1 (en) | 2023-04-18 | 2026-02-25 | Sana Biotechnology, Inc. | Universal protein g fusogens and adapter systems thereof and related lipid particles and uses |
| WO2024220598A2 (en) | 2023-04-18 | 2024-10-24 | Sana Biotechnology, Inc. | Lentiviral vectors with two or more genomes |
| 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 |
| EP4697940A1 (en) | 2023-04-21 | 2026-02-25 | Vib Vzw | Allelic combinations in crops for yield increase |
| WO2024226499A1 (en) | 2023-04-24 | 2024-10-31 | The Broad Institute, Inc. | Compositions and methods for modifying fertility |
| WO2024223900A1 (en) | 2023-04-28 | 2024-10-31 | Vilmorin & Cie | Genetic determinants conferring improved tobrfv resistance |
| WO2024234006A1 (en) | 2023-05-11 | 2024-11-14 | Tome Biosciences, Inc. | Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs) |
| WO2024238977A2 (en) | 2023-05-18 | 2024-11-21 | Children's Hospital Medical Center | Liver organoids with intrahepatic sympathetic nerves, and methods of use thereof |
| 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 |
| EP4716750A1 (en) | 2023-05-23 | 2026-04-01 | Sana Biotechnology, Inc. | Tandem fusogens and related lipid particles |
| EP4716741A2 (en) | 2023-05-23 | 2026-04-01 | Flagship Labs 114, Inc. | Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression |
| EP4731750A2 (en) | 2023-06-23 | 2026-04-29 | Children's Hospital Medical Center | Methods of matrix-free suspension culture |
| WO2025006419A1 (en) | 2023-06-26 | 2025-01-02 | Flagship Pioneering Innovations Vii, Llc | Engineered plasmodia and related methods |
| EP4734755A1 (en) | 2023-06-28 | 2026-05-06 | Vilmorin & Cie | Pepper resistance to meloidogyne enterolobii |
| CN121548346A (zh) | 2023-07-24 | 2026-02-17 | 维尔莫林公司 | 甜瓜对cysdv的抗性 |
| WO2025027165A1 (en) | 2023-08-01 | 2025-02-06 | Basf Plant Science Company Gmbh | Increased resistance by expression of an ics protein |
| CN121605117A (zh) | 2023-08-01 | 2026-03-03 | 巴斯夫植物科学有限公司 | 通过表达msbp1蛋白增加抗性 |
| WO2025030048A2 (en) | 2023-08-03 | 2025-02-06 | J.R. Simplot Company | Fusarium wilt resistance genes |
| WO2025038723A1 (en) | 2023-08-14 | 2025-02-20 | The Texas A&M University System | Corn endotype-derived polypeptides capable of metabolizing fumonisin |
| 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 |
| AU2024335327A1 (en) | 2023-09-01 | 2026-03-26 | 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 |
| WO2025054202A1 (en) | 2023-09-05 | 2025-03-13 | Sana Biotechnology, Inc. | Method of screening a sample comprising a transgene with a unique barcode |
| WO2025054540A1 (en) | 2023-09-08 | 2025-03-13 | Iovance Biotherapeutics, Inc. | Methods of gene-editing using programmable nucleases |
| WO2025064408A1 (en) | 2023-09-18 | 2025-03-27 | The Broad Institute, Inc. | Compositions and methods for treating cardiovascular disease |
| GB202314578D0 (en) | 2023-09-22 | 2023-11-08 | Univ Manchester | Methods of producing homoplasmic modified plants or parts thereof |
| WO2025072803A1 (en) | 2023-09-29 | 2025-04-03 | Children's Hospital Medical Center | Ntrk2 signaling-mediated alveolar capillary injury and repair |
| WO2025076141A1 (en) | 2023-10-03 | 2025-04-10 | Inari Agriculture Technology, Inc. | Viral delivery of grna to the scion |
| AU2024357861A1 (en) | 2023-10-12 | 2026-04-23 | Renagade Therapeutics Management Inc. | Nickase-retron template-based precision editing system and methods of use |
| WO2025101484A1 (en) | 2023-11-06 | 2025-05-15 | Iovance Biotherapeutics, Inc. | Treatment of endometrial cancers with tumor infiltrating lymphocyte therapies |
| US20250161347A1 (en) | 2023-11-22 | 2025-05-22 | Flagship Pioneering Innovations Vii, Llc | Methods and compositions for treating non-alcoholic fatty liver disease |
| WO2025151838A1 (en) | 2024-01-12 | 2025-07-17 | Sana Biotechnology, Inc. | Safety switches to control in vitro and in vivo proliferation of cell therapy products |
| WO2025155753A2 (en) | 2024-01-17 | 2025-07-24 | Renagade Therapeutics Management Inc. | Improved gene editing system, guides, and methods |
| WO2025155518A1 (en) | 2024-01-18 | 2025-07-24 | Pivot Bio, Inc. | Host cells for enhanced plant growth |
| WO2025155521A1 (en) | 2024-01-18 | 2025-07-24 | Pivot Bio, Inc. | Host cells for enhanced plant growth |
| WO2025158400A1 (en) | 2024-01-24 | 2025-07-31 | Yale University | Compositions and methods of natural killer cell hyperboosts for enhancement of nk cell therapy |
| WO2025162985A1 (en) | 2024-01-30 | 2025-08-07 | Basf Plant Science Company Gmbh | Increased plant disease resistance by expression of a glycine-rich protein |
| WO2025168705A1 (en) | 2024-02-08 | 2025-08-14 | Vib Vzw | Means and methods for the production of saponins with endosomal escape-enhancing properties |
| 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 |
| WO2025184529A1 (en) | 2024-03-01 | 2025-09-04 | Sana Biotechnology, Inc. | Viral particles with fusogen display and related compositions and methods |
| WO2025184759A1 (en) | 2024-03-04 | 2025-09-12 | Pioneer Hi-Bred International, Inc. | Maize plants comprising resistance to gray leaf spot and compositions and methods for selecting and producing the same |
| WO2025186284A1 (en) | 2024-03-05 | 2025-09-12 | Vilmorin & Cie | Increased yield in cucumber |
| WO2025194019A1 (en) | 2024-03-14 | 2025-09-18 | Flagship Pioneering Innovations Vii, Llc | Methods for treating liver fibrosis and non-alcoholic fatty liver disease |
| WO2025202473A1 (en) | 2024-03-28 | 2025-10-02 | Revvity Discovery Limited | A nucleic acid deaminase, a base editor and uses thereof |
| WO2025212920A1 (en) | 2024-04-03 | 2025-10-09 | Children's Hospital Medical Center | Multi-zonal liver organoids |
| WO2025217202A1 (en) | 2024-04-08 | 2025-10-16 | Children's Hospital Medical Center | Bile duct organoid |
| EP4677108A1 (en) | 2024-04-22 | 2026-01-14 | 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 |
| WO2025232898A1 (en) | 2024-05-10 | 2025-11-13 | Yoltech Therapeutics Co., Ltd | Lipid compounds and lipid nanoparticle compositions |
| WO2025240947A1 (en) | 2024-05-17 | 2025-11-20 | University Of Massachusetts | Therapeutic treatment for fragile x-associated disorder |
| WO2025245188A2 (en) | 2024-05-21 | 2025-11-27 | Flagship Pioneering Innovations Vii, Llc | Methods of treating liver steatosis and non-alcoholic fatty liver disease |
| US20250382627A1 (en) | 2024-06-13 | 2025-12-18 | J.R. Simplot Company | Engineering remontant flowering in rosaceae |
| WO2026006542A2 (en) | 2024-06-26 | 2026-01-02 | Yale University | Compositions and methods for crispr/cas9 based reactivation of human angelman syndrome |
| WO2026015523A1 (en) | 2024-07-09 | 2026-01-15 | Pioneer Hi-Bred International, Inc. | Methods and systems for use in plant breeding |
| WO2026015832A2 (en) | 2024-07-12 | 2026-01-15 | Arbor Biotechnologies, Inc. | Reverse transcriptases and gene editing systems comprising such |
| WO2026015829A2 (en) | 2024-07-12 | 2026-01-15 | Arbor Biotechnologies, Inc. | Small reverse transcriptases and gene editing systems comprising such |
| WO2026017833A1 (en) | 2024-07-17 | 2026-01-22 | Vilmorin & Cie | Tswv-resistant pepper |
| WO2026036000A1 (en) | 2024-08-08 | 2026-02-12 | Invaio Sciences, Inc. | Nature-derived lipid particles for use in agriculture |
| WO2026047626A1 (en) | 2024-08-30 | 2026-03-05 | Inceptor Bio, Llc | Compositions and methods involving immune cells engineered for binding bispecific antibodies for use in cell therapy |
| WO2026072421A2 (en) | 2024-09-27 | 2026-04-02 | The Broad Institute, Inc. | Base editing methods and compositions for editing prnp in the treatment of prion disease |
| CN119601083A (zh) * | 2024-11-16 | 2025-03-11 | 大连易康生物科技有限公司 | 一种基因测序数据处理方法及系统 |
| CN119874861B (zh) * | 2025-01-10 | 2025-11-25 | 中国科学院遗传与发育生物学研究所 | SlABCG45基因在提高番茄的列当抗性和产量中的应用 |
Family Cites Families (83)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US2011A (en) | 1841-03-18 | Appabatxts for piling saws | ||
| US44790A (en) | 1864-10-25 | Improvement in force-pumps | ||
| US2011008A (en) | 1929-04-15 | 1935-08-13 | Rca Corp | Electric discharge tube |
| 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 |
| 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 |
| 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 |
| 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 |
| DE122007000007I2 (de) | 1986-04-09 | 2010-12-30 | Genzyme Corp | Genetisch transformierte Tiere, die ein gewünschtes Protein in Milch absondern |
| 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 |
| 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 |
| 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 |
| 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 |
| US20090100536A1 (en) | 2001-12-04 | 2009-04-16 | Monsanto Company | Transgenic plants with enhanced agronomic traits |
| US7539579B2 (en) | 2002-04-09 | 2009-05-26 | Beattie Kenneth L | Oligonucleotide probes for genosensor chips |
| US20070134796A1 (en) | 2005-07-26 | 2007-06-14 | Sangamo Biosciences, Inc. | Targeted integration and expression of exogenous nucleic acid sequences |
| US8053232B2 (en) | 2004-01-23 | 2011-11-08 | Virxsys Corporation | Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing |
| US20050220796A1 (en) | 2004-03-31 | 2005-10-06 | Dynan William S | Compositions and methods for modulating DNA repair |
| 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 |
| US9677123B2 (en) | 2006-03-15 | 2017-06-13 | Siemens Healthcare Diagnostics Inc. | Degenerate nucleobase analogs |
| WO2008093152A1 (en) * | 2007-02-01 | 2008-08-07 | Cellectis | Obligate heterodimer meganucleases and uses thereof |
| CN101688241B (zh) | 2007-03-02 | 2015-01-21 | 杜邦营养生物科学有限公司 | 具有改善的噬菌体抗性的培养物 |
| US8586709B2 (en) * | 2007-09-25 | 2013-11-19 | Pastoral Greenhouse Research Limited | Cell-permeabilising peptides and polypeptides for microbial cells |
| 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 |
| US9404098B2 (en) | 2008-11-06 | 2016-08-02 | University Of Georgia Research Foundation, Inc. | Method for cleaving a target RNA using a Cas6 polypeptide |
| EP2352369B1 (en) | 2008-12-04 | 2017-04-26 | Sangamo BioSciences, Inc. | Genome editing in rats using zinc-finger nucleases |
| US20110016540A1 (en) | 2008-12-04 | 2011-01-20 | Sigma-Aldrich Co. | Genome editing of genes associated with trinucleotide repeat expansion disorders in animals |
| 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 |
| US8586526B2 (en) | 2010-05-17 | 2013-11-19 | Sangamo Biosciences, Inc. | DNA-binding proteins and uses thereof |
| US8889394B2 (en) | 2009-09-07 | 2014-11-18 | Empire Technology Development Llc | Multiple domain proteins |
| EP2504439B1 (en) | 2009-11-27 | 2016-03-02 | BASF Plant Science Company GmbH | Optimized endonucleases and uses thereof |
| US10087431B2 (en) | 2010-03-10 | 2018-10-02 | The Regents Of The University Of California | Methods of generating nucleic acid fragments |
| MX2012013037A (es) | 2010-05-10 | 2013-07-29 | Univ California | Composiciones de endorribonucleasa y metodos de uso de las mismas. |
| KR101613612B1 (ko) | 2010-07-29 | 2016-04-20 | 다카라 바이오 가부시키가이샤 | 표적 염기를 검출하기 위한 rna를 함유한 프로브를 제조하기 위한 방법 |
| DK2601611T3 (da) | 2010-08-02 | 2021-02-01 | Integrated Dna Tech Inc | Fremgangsmåder til forudsigelse af stabilitet og smeltetemperaturer for nukleinsyreduplekser |
| US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
| AU2012249390B2 (en) * | 2011-04-27 | 2017-03-30 | Amyris, Inc. | Methods for genomic modification |
| WO2012164565A1 (en) | 2011-06-01 | 2012-12-06 | Yeda Research And Development Co. Ltd. | Compositions and methods for downregulating prokaryotic genes |
| US8555733B2 (en) | 2011-09-23 | 2013-10-15 | Airgas, Inc. | System and method for analyzing a refrigerant sample |
| US20130122096A1 (en) | 2011-11-14 | 2013-05-16 | Silenseed Ltd. | Compositions for drug delivery and methods of manufacturing and using same |
| 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 |
| WO2013130824A1 (en) | 2012-02-29 | 2013-09-06 | Sangamo Biosciences, Inc. | Methods and compositions for treating huntington's disease |
| WO2013141680A1 (en) * | 2012-03-20 | 2013-09-26 | Vilnius University | RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX |
| US9637739B2 (en) * | 2012-03-20 | 2017-05-02 | Vilnius University | RNA-directed DNA cleavage by the Cas9-crRNA complex |
| AU2013266968B2 (en) | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| AU2013335451C1 (en) | 2012-10-23 | 2024-07-04 | Toolgen Incorporated | Composition for cleaving a target DNA comprising a guide RNA specific for the target DNA and Cas protein-encoding nucleic acid or Cas protein, and use thereof |
| PL3360964T3 (pl) | 2012-12-06 | 2020-03-31 | Sigma-Aldrich Co. Llc | Modyfikacja i regulacja genomu oparta na 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 |
| WO2014093655A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| IL239344B2 (en) | 2012-12-12 | 2024-06-01 | Broad Inst Inc | Systems engineering, methods and optimal guiding components for sequence manipulation |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| CN113355357B (zh) | 2012-12-12 | 2024-12-03 | 布罗德研究所有限公司 | 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化 |
| EP2840140B2 (en) | 2012-12-12 | 2023-02-22 | The Broad Institute, Inc. | Crispr-Cas based method for mutation of prokaryotic cells |
| EP3031921B1 (en) | 2012-12-12 | 2025-03-12 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| 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 |
| US20140186843A1 (en) | 2012-12-12 | 2014-07-03 | Massachusetts Institute Of Technology | Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| WO2014093709A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| DK2931891T3 (da) | 2012-12-17 | 2019-08-19 | Harvard College | Rna-styret modificering af menneskelige genomer |
| US11135273B2 (en) * | 2013-02-07 | 2021-10-05 | The Rockefeller University | Sequence specific antimicrobials |
| US10660943B2 (en) * | 2013-02-07 | 2020-05-26 | The Rockefeller University | Sequence specific antimicrobials |
| EP4286517A3 (en) * | 2013-04-04 | 2024-03-13 | President and Fellows of Harvard College | Therapeutic uses of genome editing with crispr/cas systems |
| MX374532B (es) | 2013-06-17 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos. |
| 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 |
| WO2014204724A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| EP3470089A1 (en) | 2013-12-12 | 2019-04-17 | 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 |
| EP3215623A4 (en) * | 2014-11-06 | 2018-09-26 | President and Fellows of Harvard College | Cells lacking b2m surface expression and methods for allogeneic administration of such cells |
-
2013
- 2013-12-12 EP EP14188820.6A patent/EP2840140B2/en active Active
- 2013-12-12 EP EP23192951.4A patent/EP4286404A3/en active Pending
- 2013-12-12 US US14/105,035 patent/US20140189896A1/en not_active Abandoned
- 2013-12-12 CA CA2894668A patent/CA2894668A1/en active Pending
- 2013-12-12 EP EP25158803.4A patent/EP4570817A3/en active Pending
- 2013-12-12 EP EP17162030.5A patent/EP3252160B1/en active Active
- 2013-12-12 EP EP13812430.0A patent/EP2825654B1/en not_active Revoked
- 2013-12-12 EP EP20198492.9A patent/EP3825401A1/en active Pending
- 2013-12-12 WO PCT/US2013/074611 patent/WO2014093595A1/en not_active Ceased
- 2013-12-12 CN CN201910829026.XA patent/CN110982844B/zh active Active
- 2013-12-12 JP JP2015547530A patent/JP2016505256A/ja active Pending
- 2013-12-12 EP EP23192260.0A patent/EP4286402A3/en active Pending
- 2013-12-12 BR BR122021008308-8A patent/BR122021008308B1/pt active IP Right Grant
- 2013-12-12 EP EP23192949.8A patent/EP4286403A3/en active Pending
- 2013-12-12 CN CN202111233527.5A patent/CN114634950A/zh active Pending
- 2013-12-12 CN CN201380072868.6A patent/CN105658796B/zh active Active
- 2013-12-12 DK DK17162030.5T patent/DK3252160T3/da active
- 2013-12-12 AU AU2013359262A patent/AU2013359262C1/en active Active
- 2013-12-12 EP EP23161559.2A patent/EP4234696A3/en active Pending
- 2013-12-12 BR BR112015013786-5A patent/BR112015013786B1/pt active IP Right Grant
-
2014
- 2014-02-18 US US14/183,486 patent/US8795965B2/en active Active
- 2014-04-23 US US14/259,420 patent/US8871445B2/en active Active
- 2014-09-26 US US14/497,627 patent/US20150031134A1/en not_active Abandoned
- 2014-10-24 US US14/523,799 patent/US9840713B2/en active Active
-
2016
- 2016-01-07 US US14/990,444 patent/US9822372B2/en active Active
- 2016-01-08 US US14/991,083 patent/US20160115489A1/en not_active Abandoned
- 2016-08-05 US US15/230,161 patent/US20180327756A1/en active Pending
-
2017
- 2017-12-11 US US15/838,064 patent/US20180305704A1/en not_active Abandoned
-
2018
- 2018-02-02 US US15/887,377 patent/US20180179547A1/en not_active Abandoned
- 2018-04-30 US US15/967,495 patent/US20190017058A1/en active Pending
- 2018-04-30 US US15/967,510 patent/US20190040399A1/en not_active Abandoned
- 2018-11-01 US US16/178,551 patent/US20190292550A1/en not_active Abandoned
-
2019
- 2019-04-22 JP JP2019080952A patent/JP6896786B2/ja active Active
- 2019-06-18 US US16/445,156 patent/US20200032278A1/en not_active Abandoned
- 2019-06-18 US US16/445,150 patent/US20200032277A1/en not_active Abandoned
- 2019-08-05 US US16/532,442 patent/US20200063147A1/en active Pending
- 2019-08-07 US US16/535,043 patent/US20200080094A1/en active Pending
- 2019-12-16 AU AU2019280394A patent/AU2019280394B2/en active Active
-
2020
- 2020-06-19 US US16/906,580 patent/US20200318123A1/en active Pending
- 2020-09-28 US US17/034,754 patent/US20210079407A1/en active Pending
-
2021
- 2021-06-09 JP JP2021096264A patent/JP7269990B2/ja active Active
- 2021-10-18 US US17/503,928 patent/US20220135985A1/en active Pending
-
2022
- 2022-06-01 AU AU2022203763A patent/AU2022203763B2/en active Active
-
2023
- 2023-02-14 US US18/109,550 patent/US20240182913A1/en active Pending
- 2023-03-29 US US18/128,122 patent/US20230340505A1/en active Pending
- 2023-04-13 US US18/134,317 patent/US20230374527A1/en active Pending
- 2023-04-24 JP JP2023070572A patent/JP7542681B2/ja active Active
- 2023-08-23 US US18/454,343 patent/US20240117365A1/en active Pending
-
2024
- 2024-08-20 JP JP2024138377A patent/JP7847620B2/ja active Active
-
2025
- 2025-01-17 US US19/028,666 patent/US20250304977A1/en active Pending
- 2025-01-17 US US19/028,841 patent/US20250250578A1/en active Pending
- 2025-05-22 AU AU2025203777A patent/AU2025203777A1/en active Pending
- 2025-09-29 US US19/342,889 patent/US20260022392A1/en active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7542681B2 (ja) | 配列操作のためのCRISPR-Cas成分系、方法および組成物 | |
| JP7542595B2 (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 | |
| JP7463436B2 (ja) | 遺伝子産物の発現を変更するためのCRISPR-Cas系および方法 | |
| DK2921557T3 (en) | Design of systems, methods and optimized sequence manipulation guide compositions | |
| KR102649341B1 (ko) | H1 프로모터를 사용하여 crispr 가이드 rna를 발현시키기 위한 조성물 및 방법 | |
| DK2931898T3 (en) | CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS | |
| DK2825654T3 (en) | SYSTEMS, PROCEDURES, AND COMPOSITIONS WITH CRISPR-CAS COMPONENTS FOR SEQUENCE MANIPULATION. | |
| HK1216759B (zh) | 用於序列操纵的系统、方法和优化的指导组合物的工程化 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| B09A | Decision: intention to grant [chapter 9.1 patent gazette] | ||
| B16A | Patent or certificate of addition of invention granted [chapter 16.1 patent gazette] |
Free format text: PRAZO DE VALIDADE: 20 (VINTE) ANOS CONTADOS A PARTIR DE 12/12/2013, OBSERVADAS AS CONDICOES LEGAIS |