CA2361191A1 - Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site - Google Patents
Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site Download PDFInfo
- Publication number
- CA2361191A1 CA2361191A1 CA002361191A CA2361191A CA2361191A1 CA 2361191 A1 CA2361191 A1 CA 2361191A1 CA 002361191 A CA002361191 A CA 002361191A CA 2361191 A CA2361191 A CA 2361191A CA 2361191 A1 CA2361191 A1 CA 2361191A1
- Authority
- CA
- Canada
- Prior art keywords
- dna
- interest
- site
- cell
- targeting
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Abandoned
Links
- 108090000623 proteins and genes Proteins 0.000 title claims abstract description 83
- 108020004414 DNA Proteins 0.000 title claims description 200
- 238000003776 cleavage reaction Methods 0.000 title claims description 46
- 230000007017 scission Effects 0.000 title claims description 46
- 230000008439 repair process Effects 0.000 title claims description 36
- 230000006698 induction Effects 0.000 title description 9
- 102000053602 DNA Human genes 0.000 title description 8
- 230000002759 chromosomal effect Effects 0.000 title description 5
- 239000013611 chromosomal DNA Substances 0.000 claims abstract description 76
- 238000000034 method Methods 0.000 claims abstract description 74
- 108091008146 restriction endonucleases Proteins 0.000 claims abstract description 74
- 238000011321 prophylaxis Methods 0.000 claims abstract description 8
- 230000008685 targeting Effects 0.000 claims description 116
- 230000035772 mutation Effects 0.000 claims description 31
- 230000001939 inductive effect Effects 0.000 claims description 30
- 230000006798 recombination Effects 0.000 claims description 27
- 238000005215 recombination Methods 0.000 claims description 27
- 230000007018 DNA scission Effects 0.000 claims description 14
- 230000004568 DNA-binding Effects 0.000 claims description 10
- 230000002068 genetic effect Effects 0.000 claims description 10
- 230000003902 lesion Effects 0.000 claims description 10
- 208000026350 Inborn Genetic disease Diseases 0.000 claims description 7
- 208000016361 genetic disease Diseases 0.000 claims description 7
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 claims description 6
- 230000027455 binding Effects 0.000 claims description 6
- 239000011701 zinc Substances 0.000 claims description 6
- 229910052725 zinc Inorganic materials 0.000 claims description 6
- 238000012937 correction Methods 0.000 claims description 4
- 230000004048 modification Effects 0.000 claims description 3
- 238000012986 modification Methods 0.000 claims description 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract description 10
- 201000010099 disease Diseases 0.000 abstract description 8
- 230000000415 inactivating effect Effects 0.000 abstract description 2
- 230000005782 double-strand break Effects 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 description 139
- 239000013612 plasmid Substances 0.000 description 62
- 230000014509 gene expression Effects 0.000 description 26
- 238000006243 chemical reaction Methods 0.000 description 22
- 239000013598 vector Substances 0.000 description 21
- 239000012634 fragment Substances 0.000 description 16
- BRZYSWJRSDMWLG-CAXSIQPQSA-N geneticin Chemical compound O1C[C@@](O)(C)[C@H](NC)[C@@H](O)[C@H]1O[C@@H]1[C@@H](O)[C@H](O[C@@H]2[C@@H]([C@@H](O)[C@H](O)[C@@H](C(C)O)O2)N)[C@@H](N)C[C@H]1N BRZYSWJRSDMWLG-CAXSIQPQSA-N 0.000 description 14
- 238000002474 experimental method Methods 0.000 description 13
- 102000004190 Enzymes Human genes 0.000 description 12
- 108090000790 Enzymes Proteins 0.000 description 12
- 150000007523 nucleic acids Chemical class 0.000 description 11
- 108091028043 Nucleic acid sequence Proteins 0.000 description 10
- 238000001890 transfection Methods 0.000 description 10
- 108020004707 nucleic acids Proteins 0.000 description 9
- 102000039446 nucleic acids Human genes 0.000 description 9
- 108700028369 Alleles Proteins 0.000 description 8
- 241001465754 Metazoa Species 0.000 description 8
- 241000700605 Viruses Species 0.000 description 8
- 230000006801 homologous recombination Effects 0.000 description 8
- 238000002744 homologous recombination Methods 0.000 description 8
- 102000004169 proteins and genes Human genes 0.000 description 8
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 7
- 101100282111 Caenorhabditis elegans gap-2 gene Proteins 0.000 description 7
- 101150013552 LDLR gene Proteins 0.000 description 7
- 108091081024 Start codon Proteins 0.000 description 7
- 230000001413 cellular effect Effects 0.000 description 6
- 238000012217 deletion Methods 0.000 description 6
- 230000037430 deletion Effects 0.000 description 6
- 238000010586 diagram Methods 0.000 description 6
- 238000003780 insertion Methods 0.000 description 6
- 230000037431 insertion Effects 0.000 description 6
- 238000010369 molecular cloning Methods 0.000 description 6
- 230000008488 polyadenylation Effects 0.000 description 6
- 238000001556 precipitation Methods 0.000 description 6
- 101000583086 Bunodosoma granuliferum Delta-actitoxin-Bgr2b Proteins 0.000 description 5
- 108091026890 Coding region Proteins 0.000 description 5
- 241000282414 Homo sapiens Species 0.000 description 5
- 230000001404 mediated effect Effects 0.000 description 5
- 108020004705 Codon Proteins 0.000 description 4
- 108010014303 DNA-directed DNA polymerase Proteins 0.000 description 4
- 102000016928 DNA-directed DNA polymerase Human genes 0.000 description 4
- 241000124008 Mammalia Species 0.000 description 4
- 108700008625 Reporter Genes Proteins 0.000 description 4
- 150000005829 chemical entities Chemical class 0.000 description 4
- 210000001671 embryonic stem cell Anatomy 0.000 description 4
- 238000001727 in vivo Methods 0.000 description 4
- 239000000203 mixture Substances 0.000 description 4
- 238000003752 polymerase chain reaction Methods 0.000 description 4
- 238000010561 standard procedure Methods 0.000 description 4
- 238000003786 synthesis reaction Methods 0.000 description 4
- 241000193738 Bacillus anthracis Species 0.000 description 3
- 101100346152 Drosophila melanogaster modSP gene Proteins 0.000 description 3
- 108091034117 Oligonucleotide Proteins 0.000 description 3
- 108700026244 Open Reading Frames Proteins 0.000 description 3
- 108020004511 Recombinant DNA Proteins 0.000 description 3
- 238000002105 Southern blotting Methods 0.000 description 3
- 101710192266 Tegument protein VP22 Proteins 0.000 description 3
- 230000004913 activation Effects 0.000 description 3
- 238000004458 analytical method Methods 0.000 description 3
- 229910000389 calcium phosphate Inorganic materials 0.000 description 3
- 239000001506 calcium phosphate Substances 0.000 description 3
- 235000011010 calcium phosphates Nutrition 0.000 description 3
- 230000000694 effects Effects 0.000 description 3
- 238000005516 engineering process Methods 0.000 description 3
- 238000002347 injection Methods 0.000 description 3
- 239000007924 injection Substances 0.000 description 3
- 101150066555 lacZ gene Proteins 0.000 description 3
- 238000004519 manufacturing process Methods 0.000 description 3
- 238000000520 microinjection Methods 0.000 description 3
- 239000002773 nucleotide Substances 0.000 description 3
- 125000003729 nucleotide group Chemical group 0.000 description 3
- 230000008263 repair mechanism Effects 0.000 description 3
- 210000000130 stem cell Anatomy 0.000 description 3
- 239000003053 toxin Substances 0.000 description 3
- 231100000765 toxin Toxicity 0.000 description 3
- 238000013518 transcription Methods 0.000 description 3
- 230000035897 transcription Effects 0.000 description 3
- 230000009261 transgenic effect Effects 0.000 description 3
- 238000011282 treatment Methods 0.000 description 3
- QORWJWZARLRLPR-UHFFFAOYSA-H tricalcium bis(phosphate) Chemical compound [Ca+2].[Ca+2].[Ca+2].[O-]P([O-])([O-])=O.[O-]P([O-])([O-])=O QORWJWZARLRLPR-UHFFFAOYSA-H 0.000 description 3
- 241001430294 unidentified retrovirus Species 0.000 description 3
- OPIFSICVWOWJMJ-AEOCFKNESA-N 5-bromo-4-chloro-3-indolyl beta-D-galactoside Chemical compound O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@H]1OC1=CNC2=CC=C(Br)C(Cl)=C12 OPIFSICVWOWJMJ-AEOCFKNESA-N 0.000 description 2
- 241000271566 Aves Species 0.000 description 2
- 241000283690 Bos taurus Species 0.000 description 2
- 241000282693 Cercopithecidae Species 0.000 description 2
- 101100364969 Dictyostelium discoideum scai gene Proteins 0.000 description 2
- 102100031780 Endonuclease Human genes 0.000 description 2
- 108010042407 Endonucleases Proteins 0.000 description 2
- 241000713666 Lentivirus Species 0.000 description 2
- 241001529936 Murinae Species 0.000 description 2
- 241000699666 Mus <mouse, genus> Species 0.000 description 2
- 101100364971 Mus musculus Scai gene Proteins 0.000 description 2
- 229930193140 Neomycin Natural products 0.000 description 2
- 238000010240 RT-PCR analysis Methods 0.000 description 2
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 2
- 241000251539 Vertebrata <Metazoa> Species 0.000 description 2
- 210000002459 blastocyst Anatomy 0.000 description 2
- 244000309466 calf Species 0.000 description 2
- 210000000170 cell membrane Anatomy 0.000 description 2
- 230000017858 demethylation Effects 0.000 description 2
- 238000010520 demethylation reaction Methods 0.000 description 2
- 208000035475 disorder Diseases 0.000 description 2
- 241001493065 dsRNA viruses Species 0.000 description 2
- 238000004520 electroporation Methods 0.000 description 2
- 208000015181 infectious disease Diseases 0.000 description 2
- 238000005304 joining Methods 0.000 description 2
- 238000001638 lipofection Methods 0.000 description 2
- 210000004962 mammalian cell Anatomy 0.000 description 2
- 239000011159 matrix material Substances 0.000 description 2
- 229960004927 neomycin Drugs 0.000 description 2
- 150000003904 phospholipids Chemical class 0.000 description 2
- 210000002966 serum Anatomy 0.000 description 2
- 210000001082 somatic cell Anatomy 0.000 description 2
- 241000894007 species Species 0.000 description 2
- 241000701161 unidentified adenovirus Species 0.000 description 2
- 239000013603 viral vector Substances 0.000 description 2
- 241000251468 Actinopterygii Species 0.000 description 1
- 241000710929 Alphavirus Species 0.000 description 1
- 241000714230 Avian leukemia virus Species 0.000 description 1
- 241001485018 Baboon endogenous virus Species 0.000 description 1
- 241000894006 Bacteria Species 0.000 description 1
- 241000714266 Bovine leukemia virus Species 0.000 description 1
- 241000700198 Cavia Species 0.000 description 1
- 241000711573 Coronaviridae Species 0.000 description 1
- 241000938605 Crocodylia Species 0.000 description 1
- 241000701022 Cytomegalovirus Species 0.000 description 1
- 241000450599 DNA viruses Species 0.000 description 1
- 108010008532 Deoxyribonuclease I Proteins 0.000 description 1
- 241000702421 Dependoparvovirus Species 0.000 description 1
- 241000196324 Embryophyta Species 0.000 description 1
- 241000283086 Equidae Species 0.000 description 1
- 241000289695 Eutheria Species 0.000 description 1
- 241000714165 Feline leukemia virus Species 0.000 description 1
- 241000714174 Feline sarcoma virus Species 0.000 description 1
- 108090000331 Firefly luciferases Proteins 0.000 description 1
- 241000710831 Flavivirus Species 0.000 description 1
- 208000000666 Fowlpox Diseases 0.000 description 1
- 241000713813 Gibbon ape leukemia virus Species 0.000 description 1
- 241000941423 Grom virus Species 0.000 description 1
- 241000238631 Hexapoda Species 0.000 description 1
- 241000282412 Homo Species 0.000 description 1
- 241000701044 Human gammaherpesvirus 4 Species 0.000 description 1
- 102000000853 LDL receptors Human genes 0.000 description 1
- 108010001831 LDL receptors Proteins 0.000 description 1
- 241000713821 Mason-Pfizer monkey virus Species 0.000 description 1
- 201000005505 Measles Diseases 0.000 description 1
- 241000289419 Metatheria Species 0.000 description 1
- 241000713862 Moloney murine sarcoma virus Species 0.000 description 1
- 241000289390 Monotremata Species 0.000 description 1
- 241000713333 Mouse mammary tumor virus Species 0.000 description 1
- 241000714177 Murine leukemia virus Species 0.000 description 1
- 241000699670 Mus sp. Species 0.000 description 1
- 108091061960 Naked DNA Proteins 0.000 description 1
- 238000000636 Northern blotting Methods 0.000 description 1
- 241000714209 Norwalk virus Species 0.000 description 1
- 241000702244 Orthoreovirus Species 0.000 description 1
- 241000283973 Oryctolagus cuniculus Species 0.000 description 1
- 241000282579 Pan Species 0.000 description 1
- 241000709664 Picornaviridae Species 0.000 description 1
- 241000288906 Primates Species 0.000 description 1
- 102000029797 Prion Human genes 0.000 description 1
- 108091000054 Prion Proteins 0.000 description 1
- 241000125945 Protoparvovirus Species 0.000 description 1
- 206010037742 Rabies Diseases 0.000 description 1
- 241000711798 Rabies lyssavirus Species 0.000 description 1
- 241000700159 Rattus Species 0.000 description 1
- 102000007056 Recombinant Fusion Proteins Human genes 0.000 description 1
- 108010008281 Recombinant Fusion Proteins Proteins 0.000 description 1
- 241000712907 Retroviridae Species 0.000 description 1
- 241000283984 Rodentia Species 0.000 description 1
- 241000714474 Rous sarcoma virus Species 0.000 description 1
- 206010039491 Sarcoma Diseases 0.000 description 1
- 241000713311 Simian immunodeficiency virus Species 0.000 description 1
- 241000700584 Simplexvirus Species 0.000 description 1
- 241000713675 Spumavirus Species 0.000 description 1
- 241000282887 Suidae Species 0.000 description 1
- 208000000389 T-cell leukemia Diseases 0.000 description 1
- 208000028530 T-cell lymphoblastic leukemia/lymphoma Diseases 0.000 description 1
- 210000001744 T-lymphocyte Anatomy 0.000 description 1
- 108700019146 Transgenes Proteins 0.000 description 1
- 206010046865 Vaccinia virus infection Diseases 0.000 description 1
- 241000711975 Vesicular stomatitis virus Species 0.000 description 1
- 108010067390 Viral Proteins Proteins 0.000 description 1
- 241000714205 Woolly monkey sarcoma virus Species 0.000 description 1
- 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 1
- 239000002253 acid Substances 0.000 description 1
- 230000009471 action Effects 0.000 description 1
- 238000000246 agarose gel electrophoresis Methods 0.000 description 1
- 210000004102 animal cell Anatomy 0.000 description 1
- 238000010171 animal model Methods 0.000 description 1
- 238000003556 assay Methods 0.000 description 1
- 210000001130 astrocyte Anatomy 0.000 description 1
- 208000004668 avian leukosis Diseases 0.000 description 1
- 210000003719 b-lymphocyte Anatomy 0.000 description 1
- 230000001580 bacterial effect Effects 0.000 description 1
- 108010005774 beta-Galactosidase Proteins 0.000 description 1
- 230000003115 biocidal effect Effects 0.000 description 1
- 238000001574 biopsy Methods 0.000 description 1
- 230000015572 biosynthetic process Effects 0.000 description 1
- 210000000601 blood cell Anatomy 0.000 description 1
- 230000037396 body weight Effects 0.000 description 1
- 210000001185 bone marrow Anatomy 0.000 description 1
- 210000004413 cardiac myocyte Anatomy 0.000 description 1
- 230000008859 change Effects 0.000 description 1
- 239000007795 chemical reaction product Substances 0.000 description 1
- 238000010367 cloning Methods 0.000 description 1
- 230000001332 colony forming effect Effects 0.000 description 1
- 238000010276 construction Methods 0.000 description 1
- 230000008878 coupling Effects 0.000 description 1
- 238000010168 coupling process Methods 0.000 description 1
- 238000005859 coupling reaction Methods 0.000 description 1
- 238000005520 cutting process Methods 0.000 description 1
- 210000000805 cytoplasm Anatomy 0.000 description 1
- 230000006378 damage Effects 0.000 description 1
- 230000002950 deficient Effects 0.000 description 1
- 210000004443 dendritic cell Anatomy 0.000 description 1
- 238000011161 development Methods 0.000 description 1
- 230000037213 diet Effects 0.000 description 1
- 235000005911 diet Nutrition 0.000 description 1
- 239000012895 dilution Substances 0.000 description 1
- 238000010790 dilution Methods 0.000 description 1
- 239000003937 drug carrier Substances 0.000 description 1
- 210000002308 embryonic cell Anatomy 0.000 description 1
- 239000003623 enhancer Substances 0.000 description 1
- 238000006911 enzymatic reaction Methods 0.000 description 1
- 210000002919 epithelial cell Anatomy 0.000 description 1
- 210000003743 erythrocyte Anatomy 0.000 description 1
- 210000003527 eukaryotic cell Anatomy 0.000 description 1
- 238000011156 evaluation Methods 0.000 description 1
- 239000013604 expression vector Substances 0.000 description 1
- 210000002950 fibroblast Anatomy 0.000 description 1
- 238000012239 gene modification Methods 0.000 description 1
- 238000010363 gene targeting Methods 0.000 description 1
- 238000001415 gene therapy Methods 0.000 description 1
- 230000009395 genetic defect Effects 0.000 description 1
- 230000005017 genetic modification Effects 0.000 description 1
- 235000013617 genetically modified food Nutrition 0.000 description 1
- 230000036541 health Effects 0.000 description 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 description 1
- 208000006454 hepatitis Diseases 0.000 description 1
- 231100000283 hepatitis Toxicity 0.000 description 1
- 230000001744 histochemical effect Effects 0.000 description 1
- 239000007943 implant Substances 0.000 description 1
- 238000001802 infusion Methods 0.000 description 1
- 230000010354 integration Effects 0.000 description 1
- 238000007918 intramuscular administration Methods 0.000 description 1
- 238000007912 intraperitoneal administration Methods 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 239000002502 liposome Substances 0.000 description 1
- 210000002540 macrophage Anatomy 0.000 description 1
- 239000002609 medium Substances 0.000 description 1
- 108020004999 messenger RNA Proteins 0.000 description 1
- 210000004498 neuroglial cell Anatomy 0.000 description 1
- 210000002569 neuron Anatomy 0.000 description 1
- 230000017448 oviposition Effects 0.000 description 1
- 244000045947 parasite Species 0.000 description 1
- 230000003169 placental effect Effects 0.000 description 1
- 229920000642 polymer Polymers 0.000 description 1
- 239000000047 product Substances 0.000 description 1
- 210000001236 prokaryotic cell Anatomy 0.000 description 1
- 230000010076 replication Effects 0.000 description 1
- 238000010839 reverse transcription Methods 0.000 description 1
- 210000000329 smooth muscle myocyte Anatomy 0.000 description 1
- 239000011780 sodium chloride Substances 0.000 description 1
- 239000000243 solution Substances 0.000 description 1
- 238000010186 staining Methods 0.000 description 1
- 239000008223 sterile water Substances 0.000 description 1
- 238000007920 subcutaneous administration Methods 0.000 description 1
- 238000006467 substitution reaction Methods 0.000 description 1
- 208000024891 symptom Diseases 0.000 description 1
- -1 systemic Substances 0.000 description 1
- 230000009885 systemic effect Effects 0.000 description 1
- 210000001519 tissue Anatomy 0.000 description 1
- 239000003104 tissue culture media Substances 0.000 description 1
- 230000000699 topical effect Effects 0.000 description 1
- 230000009466 transformation Effects 0.000 description 1
- 230000001131 transforming effect Effects 0.000 description 1
- 230000014616 translation Effects 0.000 description 1
- 210000004881 tumor cell Anatomy 0.000 description 1
- 241001529453 unidentified herpesvirus Species 0.000 description 1
- 241000712461 unidentified influenza virus Species 0.000 description 1
- 208000007089 vaccinia Diseases 0.000 description 1
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Chemical compound O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 1
- 210000005253 yeast cell Anatomy 0.000 description 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—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
-
- 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]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Mycology (AREA)
- Cell Biology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US11866999P | 1999-02-03 | 1999-02-03 | |
| US60/118,669 | 1999-02-03 | ||
| PCT/US2000/003014 WO2000046386A2 (en) | 1999-02-03 | 2000-02-03 | Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2361191A1 true CA2361191A1 (en) | 2000-08-10 |
Family
ID=22380031
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA002361191A Abandoned CA2361191A1 (en) | 1999-02-03 | 2000-02-03 | Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site |
Country Status (6)
| Country | Link |
|---|---|
| US (7) | US20020107214A1 (enExample) |
| EP (1) | EP1147209A2 (enExample) |
| JP (1) | JP2002535995A (enExample) |
| AU (1) | AU2982900A (enExample) |
| CA (1) | CA2361191A1 (enExample) |
| WO (1) | WO2000046386A2 (enExample) |
Families Citing this family (144)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2000046386A2 (en) | 1999-02-03 | 2000-08-10 | The Children's Medical Center Corporation | Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site |
| CA2360878A1 (en) | 1999-02-03 | 2000-08-10 | The Children's Medical Center Corporation | Gene repair involving excision of targeting dna |
| WO2003087341A2 (en) | 2002-01-23 | 2003-10-23 | The University Of Utah Research Foundation | Targeted chromosomal mutagenesis using zinc finger nucleases |
| WO2009095742A1 (en) | 2008-01-31 | 2009-08-06 | Cellectis | New i-crei derived single-chain meganuclease and uses thereof |
| WO2003078619A1 (en) | 2002-03-15 | 2003-09-25 | Cellectis | Hybrid and single chain meganucleases and use thereof |
| US20030232410A1 (en) * | 2002-03-21 | 2003-12-18 | Monika Liljedahl | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
| US7273758B2 (en) * | 2002-07-19 | 2007-09-25 | Centre National De La Recherche Scientifique | Methods for inducing targeted stimulation of meiotic recombination and kits for performing said methods |
| JP2006502748A (ja) | 2002-09-05 | 2006-01-26 | カリフォルニア インスティテュート オブ テクノロジー | 遺伝子ターゲッティングを誘発するキメラヌクレアーゼの使用方法 |
| US7563600B2 (en) | 2002-09-12 | 2009-07-21 | Combimatrix Corporation | Microarray synthesis and assembly of gene-length polynucleotides |
| EP1590468A2 (en) | 2003-01-28 | 2005-11-02 | Cellectis | Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof |
| ITMI20030821A1 (it) * | 2003-04-18 | 2004-10-19 | Internat Ct For Genetic En Gineering And | Polipeptidi chimerici e loro uso. |
| US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
| EP3222715A1 (en) * | 2003-08-08 | 2017-09-27 | Sangamo BioSciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| US20120196370A1 (en) | 2010-12-03 | 2012-08-02 | Fyodor Urnov | Methods and compositions for targeted genomic deletion |
| US11311574B2 (en) | 2003-08-08 | 2022-04-26 | Sangamo Therapeutics, Inc. | Methods and compositions for targeted cleavage and recombination |
| AU2007201649B2 (en) * | 2003-08-08 | 2010-11-04 | Sangamo Therapeutics, Inc. | Methods and Compositions for Targeted Cleavage and Recombination |
| US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| AU2004291692B2 (en) * | 2003-11-18 | 2010-06-24 | BASF Agricultural Solutions Seed US LLC | Improved targeted DNA insertion in plants |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| WO2006074956A1 (en) * | 2005-01-14 | 2006-07-20 | Bayer Bioscience N.V. | Improved plant transformation methods |
| CA2603177C (en) | 2005-04-04 | 2016-01-26 | Bayer Bioscience N.V. | Methods and means for removal of a selected dna sequence |
| ES2626025T3 (es) | 2005-10-18 | 2017-07-21 | Precision Biosciences | Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas |
| WO2008027558A2 (en) | 2006-08-31 | 2008-03-06 | Codon Devices, Inc. | Iterative nucleic acid assembly using activation of vector-encoded traits |
| CA2664414C (en) | 2006-09-28 | 2016-07-12 | Bayer Bioscience N.V. | Methods and means for removal of a selected dna sequence |
| CA2667414C (en) * | 2006-11-13 | 2015-12-29 | Sangamo Biosciences, Inc. | Methods and compositions for modification of the human glucocorticoid receptor locus |
| WO2008130629A2 (en) * | 2007-04-19 | 2008-10-30 | Codon Devices, Inc. | Engineered nucleases and their uses for nucleic acid assembly |
| CA2689345C (en) | 2007-06-05 | 2017-07-11 | Bayer Bioscience N.V. | Methods and means for exact replacement of target dna in eukaryotic organisms |
| WO2009006297A2 (en) | 2007-06-29 | 2009-01-08 | Pioneer Hi-Bred International, Inc. | Methods for altering the genome of a monocot plant cell |
| AU2008335324A1 (en) * | 2007-12-07 | 2009-06-18 | Precision Biosciences, Inc. | Rationally-designed meganucleases with recognition sequences found in DNase hypersensitive regions of the human genome |
| WO2009114321A2 (en) | 2008-03-11 | 2009-09-17 | Precision Biosciencs, Inc. | Rationally-designed meganucleases for maize genome engineering |
| US20100071083A1 (en) * | 2008-03-12 | 2010-03-18 | Smith James J | Temperature-dependent meganuclease activity |
| JP2011519558A (ja) * | 2008-04-28 | 2011-07-14 | プレシジョン バイオサイエンシズ,インク. | 合理的に設計されたdna結合タンパク質とエフェクタードメインとの融合分子 |
| DK2313498T3 (en) | 2008-07-14 | 2017-05-22 | Prec Biosciences Inc | RECOGNITION SEQUENCES FOR I-CREI-DERIVED MECHANUCLEASES AND APPLICATIONS THEREOF |
| CN102625655B (zh) * | 2008-12-04 | 2016-07-06 | 桑格摩生物科学股份有限公司 | 使用锌指核酸酶在大鼠中进行基因组编辑 |
| US20110016543A1 (en) * | 2008-12-04 | 2011-01-20 | Sigma-Aldrich Co. | Genomic editing of genes involved in inflammation |
| US20110023151A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genome editing of abc transporters |
| US20110023156A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Feline genome editing with zinc finger nucleases |
| US20110023139A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in cardiovascular disease |
| US20110023141A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved with parkinson's disease |
| US20110023158A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Bovine genome editing with zinc finger nucleases |
| US20110030072A1 (en) * | 2008-12-04 | 2011-02-03 | Sigma-Aldrich Co. | Genome editing of immunodeficiency genes in animals |
| US20110016541A1 (en) * | 2008-12-04 | 2011-01-20 | Sigma-Aldrich Co. | Genome editing of sensory-related genes in animals |
| US20110023153A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in alzheimer's disease |
| US20110023146A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in secretase-associated disorders |
| US20110016540A1 (en) * | 2008-12-04 | 2011-01-20 | Sigma-Aldrich Co. | Genome editing of genes associated with trinucleotide repeat expansion disorders in animals |
| US20110023154A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Silkworm genome editing with zinc finger nucleases |
| US20110023145A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in autism spectrum disorders |
| US20110023148A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genome editing of addiction-related genes in animals |
| US20110023150A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genome editing of genes associated with schizophrenia in animals |
| US20110023140A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Rabbit genome editing with zinc finger nucleases |
| US20110023152A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genome editing of cognition related genes in animals |
| US20110023143A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of neurodevelopmental genes in animals |
| US20110023144A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in amyotrophyic lateral sclerosis disease |
| US20110016546A1 (en) * | 2008-12-04 | 2011-01-20 | Sigma-Aldrich Co. | Porcine genome editing with zinc finger nucleases |
| US20110023149A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in tumor suppression in animals |
| US20120159654A1 (en) * | 2008-12-04 | 2012-06-21 | Sigma-Aldrich Co. | Genome editing of genes involved in adme and toxicology in animals |
| US20110016539A1 (en) * | 2008-12-04 | 2011-01-20 | Sigma-Aldrich Co. | Genome editing of neurotransmission-related genes in animals |
| US20110023147A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of prion disorder-related genes in animals |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| US20110239315A1 (en) | 2009-01-12 | 2011-09-29 | Ulla Bonas | Modular dna-binding domains and methods of use |
| EP4442820A3 (en) | 2009-02-26 | 2025-01-29 | Poseida Therapeutics, Inc. | Hyperactive piggybac transposases |
| CN102639695B (zh) | 2009-10-26 | 2015-01-21 | 独立行政法人农业生物资源研究所 | 转基因植物细胞的制造方法 |
| WO2011056872A2 (en) | 2009-11-03 | 2011-05-12 | Gen9, Inc. | Methods and microfluidic devices for the manipulation of droplets in high fidelity polynucleotide assembly |
| US8956828B2 (en) | 2009-11-10 | 2015-02-17 | Sangamo Biosciences, Inc. | Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases |
| WO2011066185A1 (en) | 2009-11-25 | 2011-06-03 | Gen9, Inc. | Microfluidic devices and methods for gene synthesis |
| CA2783351C (en) | 2009-12-10 | 2021-09-07 | Regents Of The University Of Minnesota | Tal effector-mediated dna modification |
| US9217144B2 (en) | 2010-01-07 | 2015-12-22 | Gen9, Inc. | Assembly of high fidelity polynucleotides |
| WO2011095475A1 (en) * | 2010-02-02 | 2011-08-11 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for the diagnosis and therapy of retinitis pigmentosa |
| CA2798988C (en) | 2010-05-17 | 2020-03-10 | Sangamo Biosciences, Inc. | Tal-effector (tale) dna-binding polypeptides and uses thereof |
| SG185668A1 (en) | 2010-06-09 | 2012-12-28 | Bayer Cropscience Nv | Methods and means to modify a plant genome at a nucleotide sequence commonly used in plant genome engineering |
| EP2580336B1 (en) | 2010-06-09 | 2017-05-10 | Bayer CropScience NV | Methods and means to modify a plant genome at a nucleotide sequence commonly used in plant genome engineering |
| EP3489359A1 (en) | 2010-07-23 | 2019-05-29 | Sigma Aldrich Co. LLC | Genome editing using targeting endonucleases and single-stranded nucleic acids |
| EP2637780B1 (en) | 2010-11-12 | 2022-02-09 | Gen9, Inc. | Protein arrays and methods of using and making the same |
| AU2011338841B2 (en) | 2010-11-12 | 2017-02-16 | Gen9, Inc. | Methods and devices for nucleic acids synthesis |
| AU2012266597B2 (en) | 2011-06-06 | 2016-09-22 | Bayer Cropscience Nv | Methods and means to modify a plant genome at a preselected site |
| CN103620027B (zh) | 2011-06-10 | 2017-11-21 | 巴斯夫植物科学有限公司 | 核酸酶融合蛋白及其用途 |
| MX346439B (es) | 2011-08-22 | 2017-03-17 | Bayer Cropscience Nv | Métodos y medios para modificar un genoma vegetal. |
| AU2012300401B2 (en) | 2011-08-26 | 2018-02-08 | Ginkgo Bioworks, Inc. | Compositions and methods for high fidelity assembly of nucleic acids |
| WO2013050318A1 (en) | 2011-10-07 | 2013-04-11 | Basf Plant Science Company Gmbh | Method of producing plants having increased resistance to pathogens |
| WO2013050593A1 (en) | 2011-10-07 | 2013-04-11 | Basf Plant Science Company Gmbh | Method of producing plants having increased resistance to pathogens |
| WO2013050611A1 (en) | 2011-10-07 | 2013-04-11 | Basf Plant Science Company Gmbh | Method of producing plants having increased resistance to pathogens |
| WO2013053711A1 (en) | 2011-10-10 | 2013-04-18 | Basf Plant Science Company Gmbh | Method of producing plants having increased resistance to pathogens |
| WO2013053686A1 (en) | 2011-10-10 | 2013-04-18 | Basf Plant Science Company Gmbh | Method of producing plants having increased resistance to pathogens |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| US9150853B2 (en) | 2012-03-21 | 2015-10-06 | Gen9, Inc. | Methods for screening proteins using DNA encoded chemical libraries as templates for enzyme catalysis |
| EP2841601B1 (en) | 2012-04-24 | 2019-03-06 | Gen9, Inc. | Methods for sorting nucleic acids and multiplexed preparative in vitro cloning |
| JP6275120B2 (ja) | 2012-04-25 | 2018-02-07 | リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. | 大きい標的化ベクターによるヌクレアーゼ媒介標的化 |
| FI3597749T3 (fi) | 2012-05-25 | 2023-10-09 | Univ California | Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten |
| US20150225734A1 (en) | 2012-06-19 | 2015-08-13 | Regents Of The University Of Minnesota | Gene targeting in plants using dna viruses |
| US20150191719A1 (en) | 2012-06-25 | 2015-07-09 | Gen9, Inc. | Methods for Nucleic Acid Assembly and High Throughput Sequencing |
| AU2013308770B2 (en) | 2012-08-29 | 2019-01-17 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| MX367031B (es) | 2012-11-01 | 2019-08-02 | Cellectis | Plantas para la produccion de proteinas terapeuticas. |
| PT3363902T (pt) | 2012-12-06 | 2019-12-19 | Sigma Aldrich Co Llc | Modificação e regulação de genoma baseadas em crispr |
| US20140178561A1 (en) | 2012-12-21 | 2014-06-26 | Cellectis | Potatoes with reduced cold-induced sweetening |
| AU2014235794A1 (en) | 2013-03-14 | 2015-10-22 | Caribou Biosciences, Inc. | Compositions and methods of nucleic acid-targeting nucleic acids |
| US10113162B2 (en) | 2013-03-15 | 2018-10-30 | Cellectis | Modifying soybean oil composition through targeted knockout of the FAD2-1A/1B genes |
| EP3456831B1 (en) | 2013-04-16 | 2021-07-14 | Regeneron Pharmaceuticals, Inc. | Targeted modification of rat genome |
| WO2015010114A1 (en) | 2013-07-19 | 2015-01-22 | Larix Bioscience, Llc | Methods and compositions for producing double allele knock outs |
| US11060083B2 (en) | 2013-07-19 | 2021-07-13 | Larix Bioscience Llc | Methods and compositions for producing double allele knock outs |
| DK3492593T3 (da) | 2013-11-13 | 2021-11-08 | Childrens Medical Center | Nukleasemedieret regulering af genekspression |
| MX388127B (es) | 2013-12-11 | 2025-03-19 | Regeneron Pharma | Metodos y composiciones para la modificacion dirigida de un genoma. |
| US10072066B2 (en) | 2014-02-03 | 2018-09-11 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a beta thalessemia |
| CA2952906A1 (en) | 2014-06-20 | 2015-12-23 | Cellectis | Potatoes with reduced granule-bound starch synthase |
| CA3176380A1 (en) | 2014-11-21 | 2016-05-26 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modification using paired guide rnas |
| AU2015360502A1 (en) | 2014-12-10 | 2017-06-29 | Regents Of The University Of Minnesota | Genetically modified cells, tissues, and organs for treating disease |
| WO2016166268A1 (en) | 2015-04-17 | 2016-10-20 | Cellectis | Engineering animal or plant genome using dna-guided argonaute interference systems (dais) from mesophilic prokaryotes |
| CA2990699A1 (en) | 2015-06-29 | 2017-01-05 | Ionis Pharmaceuticals, Inc. | Modified crispr rna and modified single crispr rna and uses thereof |
| GB2557123B (en) | 2015-07-31 | 2021-11-03 | Univ Minnesota | Modified cells and methods of therapy |
| US10837024B2 (en) | 2015-09-17 | 2020-11-17 | Cellectis | Modifying messenger RNA stability in plant transformations |
| JP6888906B2 (ja) * | 2015-12-11 | 2021-06-18 | 株式会社豊田中央研究所 | 生物体のゲノムの改変方法及びその利用 |
| EP4646936A3 (en) | 2016-02-02 | 2025-11-19 | Cellectis | Modifying soybean oil composition through targeted knockout of the fad3a/b/c genes |
| CN105647968B (zh) * | 2016-02-02 | 2019-07-23 | 浙江大学 | 一种CRISPR/Cas9工作效率快速测试系统及其应用 |
| AU2017291851B2 (en) | 2016-07-06 | 2022-10-13 | Cellectis | Sequential gene editing in primary immune cells |
| 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 |
| WO2018073391A1 (en) | 2016-10-19 | 2018-04-26 | Cellectis | Targeted gene insertion for improved immune cells therapy |
| UY37482A (es) | 2016-11-16 | 2018-05-31 | Cellectis | Métodos para alterar el contenido de aminoácidos en plantas mediante mutaciones de desplazamiento de marco |
| CA3059643A1 (en) | 2017-04-13 | 2018-10-18 | Cellectis | New sequence specific reagents targeting ccr5 in primary hematopoietic cells |
| EP3615668B1 (en) | 2017-04-25 | 2024-02-28 | Cellectis | Alfalfa with reduced lignin composition |
| WO2018206791A1 (en) | 2017-05-12 | 2018-11-15 | Cellectis | Protease based switch chimeric antigen receptors for safer cell immunotherapy |
| 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 |
| KR102424850B1 (ko) | 2017-06-30 | 2022-07-22 | 인스크립타 인코포레이티드 | 자동 세포 처리 방법, 모듈, 기기 및 시스템 |
| WO2019006418A2 (en) | 2017-06-30 | 2019-01-03 | Intima Bioscience, Inc. | ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY |
| US10738327B2 (en) | 2017-08-28 | 2020-08-11 | Inscripta, Inc. | Electroporation cuvettes for automation |
| AU2018338990B2 (en) | 2017-09-30 | 2022-06-23 | Inscripta, Inc. | Flow through electroporation instrumentation |
| JP7621795B2 (ja) | 2017-10-19 | 2025-01-27 | セレクティス | 改善された免疫細胞療法のためのnk阻害物質遺伝子の標的指向遺伝子組み込み |
| EP3775159A4 (en) | 2018-03-29 | 2022-01-19 | Inscripta, Inc. | AUTOMATED REGULATION OF CELL GROWTH RATES FOR INDUCTION AND TRANSFORMATION |
| WO2019200004A1 (en) | 2018-04-13 | 2019-10-17 | Inscripta, Inc. | Automated cell processing instruments comprising reagent cartridges |
| WO2019209926A1 (en) | 2018-04-24 | 2019-10-31 | Inscripta, Inc. | Automated instrumentation for production of peptide libraries |
| US10858761B2 (en) | 2018-04-24 | 2020-12-08 | Inscripta, Inc. | Nucleic acid-guided editing of exogenous polynucleotides in heterologous cells |
| US10526598B2 (en) | 2018-04-24 | 2020-01-07 | Inscripta, Inc. | Methods for identifying T-cell receptor antigens |
| TWI831776B (zh) | 2018-04-27 | 2024-02-11 | 美商艾歐凡斯生物治療公司 | 腫瘤浸潤性淋巴細胞之基因編輯和彼於免疫治療之用途 |
| AU2019292919A1 (en) | 2018-06-30 | 2021-03-11 | Inscripta, Inc. | Instruments, modules, and methods for improved detection of edited sequences in live cells |
| US11142740B2 (en) | 2018-08-14 | 2021-10-12 | Inscripta, Inc. | Detection of nuclease edited sequences in automated modules and instruments |
| US10532324B1 (en) | 2018-08-14 | 2020-01-14 | Inscripta, Inc. | Instruments, modules, and methods for improved detection of edited sequences in live cells |
| WO2020081149A2 (en) | 2018-08-30 | 2020-04-23 | Inscripta, Inc. | Improved detection of nuclease edited sequences in automated modules and instruments |
| CN114008070A (zh) | 2019-06-21 | 2022-02-01 | 因思科瑞普特公司 | 导致大肠杆菌赖氨酸产量增加的全基因组合理设计的突变 |
| US10927385B2 (en) | 2019-06-25 | 2021-02-23 | Inscripta, Inc. | Increased nucleic-acid guided cell editing in yeast |
| WO2021081378A1 (en) | 2019-10-25 | 2021-04-29 | Iovance Biotherapeutics, Inc. | Gene editing of tumor infiltrating lymphocytes and uses of same in immunotherapy |
| US10689669B1 (en) | 2020-01-11 | 2020-06-23 | Inscripta, Inc. | Automated multi-module cell processing methods, instruments, and systems |
| US20210332388A1 (en) | 2020-04-24 | 2021-10-28 | Inscripta, Inc. | Compositions, methods, modules and instruments for automated nucleic acid-guided nuclease editing in mammalian cells |
| WO2021224395A1 (en) | 2020-05-06 | 2021-11-11 | Cellectis S.A. | Methods for targeted insertion of exogenous sequences in cellular genomes |
| US20230279440A1 (en) | 2020-05-06 | 2023-09-07 | Cellectis S.A. | Methods to genetically modify cells for delivery of therapeutic proteins |
| US11787841B2 (en) | 2020-05-19 | 2023-10-17 | Inscripta, Inc. | Rationally-designed mutations to the thrA gene for enhanced lysine production in E. coli |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPH0777556B2 (ja) * | 1990-08-21 | 1995-08-23 | 理化学研究所 | 部位特異的エンドヌクレアーゼSceIの小サブユニットの遺伝子 |
| US5792640A (en) | 1992-04-03 | 1998-08-11 | The Johns Hopkins University | General method to clone hybrid restriction endonucleases using lig gene |
| US5436150A (en) * | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease |
| US5474896A (en) * | 1992-05-05 | 1995-12-12 | Institut Pasteur | Nucleotide sequence encoding the enzyme I-SceI and the uses thereof |
| US5792632A (en) * | 1992-05-05 | 1998-08-11 | Institut Pasteur | Nucleotide sequence encoding the enzyme I-SceI and the uses thereof |
| ATE178654T1 (de) * | 1993-12-20 | 1999-04-15 | Akzo Nobel Nv | Impstoff zum schutze von pferden gegen pferdeherpesvirus-infektionen |
| US5830729A (en) * | 1996-04-18 | 1998-11-03 | Institut Pasteur | I Sce I-induced gene replacement and gene conversion in embryonic stem cells |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| CA2360878A1 (en) * | 1999-02-03 | 2000-08-10 | The Children's Medical Center Corporation | Gene repair involving excision of targeting dna |
| WO2000046386A2 (en) * | 1999-02-03 | 2000-08-10 | The Children's Medical Center Corporation | Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site |
| EP2180058A1 (en) * | 2008-10-23 | 2010-04-28 | Cellectis | Meganuclease recombination system |
-
2000
- 2000-02-03 WO PCT/US2000/003014 patent/WO2000046386A2/en not_active Ceased
- 2000-02-03 JP JP2000597445A patent/JP2002535995A/ja active Pending
- 2000-02-03 CA CA002361191A patent/CA2361191A1/en not_active Abandoned
- 2000-02-03 EP EP00908499A patent/EP1147209A2/en not_active Ceased
- 2000-02-03 AU AU29829/00A patent/AU2982900A/en not_active Abandoned
-
2001
- 2001-07-27 US US09/917,295 patent/US20020107214A1/en not_active Abandoned
-
2003
- 2003-01-06 US US10/337,229 patent/US20040019002A1/en not_active Abandoned
-
2006
- 2006-12-08 US US11/636,397 patent/US7629326B2/en not_active Expired - Fee Related
-
2009
- 2009-10-28 US US12/607,502 patent/US20100111924A1/en not_active Abandoned
-
2012
- 2012-03-12 US US13/417,969 patent/US8921332B2/en not_active Expired - Fee Related
-
2014
- 2014-11-21 US US14/550,463 patent/US9458439B2/en not_active Expired - Fee Related
-
2016
- 2016-09-30 US US15/281,242 patent/US20170037434A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| US20100111924A1 (en) | 2010-05-06 |
| US8921332B2 (en) | 2014-12-30 |
| EP1147209A2 (en) | 2001-10-24 |
| US20120230971A1 (en) | 2012-09-13 |
| US20020107214A1 (en) | 2002-08-08 |
| US20040019002A1 (en) | 2004-01-29 |
| AU2982900A (en) | 2000-08-25 |
| US20170037434A1 (en) | 2017-02-09 |
| WO2000046386A2 (en) | 2000-08-10 |
| WO2000046386A3 (en) | 2000-12-14 |
| JP2002535995A (ja) | 2002-10-29 |
| US9458439B2 (en) | 2016-10-04 |
| US7629326B2 (en) | 2009-12-08 |
| WO2000046386A9 (en) | 2001-09-27 |
| US20150259655A1 (en) | 2015-09-17 |
| US20070141038A1 (en) | 2007-06-21 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US7629326B2 (en) | Chromosomal modification involving the induction of double-stranded DNA cleavage and homologous recombination at the cleavage site | |
| US7960525B2 (en) | Gene repair involving in vivo excision of targeting DNA | |
| US10006053B2 (en) | Use of chimeric nucleases to stimulate gene targeting | |
| AU2002333832B9 (en) | Random integration of a polynucleotide after in vivo linearization | |
| KR100490188B1 (ko) | 진핵 세포에서의 서열 특이적 dna 재조합 | |
| AU2004202860B2 (en) | Gene repair involving in vivo excision of targeting DNA | |
| Grandchamp et al. | Hybrid Lentivirus-phiC31-int-NLS Vector Allows Site-Specific Recombination in |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request | ||
| FZDE | Dead |