CA2704383A1 - Rationally-designed single-chain meganucleases with non-palindromic recognition sequences - Google Patents
Rationally-designed single-chain meganucleases with non-palindromic recognition sequences Download PDFInfo
- Publication number
- CA2704383A1 CA2704383A1 CA2704383A CA2704383A CA2704383A1 CA 2704383 A1 CA2704383 A1 CA 2704383A1 CA 2704383 A CA2704383 A CA 2704383A CA 2704383 A CA2704383 A CA 2704383A CA 2704383 A1 CA2704383 A1 CA 2704383A1
- Authority
- CA
- Canada
- Prior art keywords
- meganuclease
- sequence
- laglidadg
- chain
- cleavage site
- 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
- 238000000034 method Methods 0.000 claims abstract description 103
- 150000007523 nucleic acids Chemical class 0.000 claims abstract description 94
- 102000039446 nucleic acids Human genes 0.000 claims abstract description 65
- 108020004707 nucleic acids Proteins 0.000 claims abstract description 65
- 108090000765 processed proteins & peptides Proteins 0.000 claims abstract description 46
- 229920001184 polypeptide Polymers 0.000 claims abstract description 23
- 102000004196 processed proteins & peptides Human genes 0.000 claims abstract description 23
- 108010050663 endodeoxyribonuclease CreI Proteins 0.000 claims description 117
- 108090000623 proteins and genes Proteins 0.000 claims description 108
- 238000003776 cleavage reaction Methods 0.000 claims description 85
- 230000007017 scission Effects 0.000 claims description 85
- 210000004027 cell Anatomy 0.000 claims description 79
- 210000004899 c-terminal region Anatomy 0.000 claims description 77
- 150000001413 amino acids Chemical class 0.000 claims description 72
- 102000004169 proteins and genes Human genes 0.000 claims description 62
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 49
- 210000000349 chromosome Anatomy 0.000 claims description 36
- 230000004048 modification Effects 0.000 claims description 29
- 238000012986 modification Methods 0.000 claims description 29
- 230000006780 non-homologous end joining Effects 0.000 claims description 27
- 230000006801 homologous recombination Effects 0.000 claims description 26
- 238000002744 homologous recombination Methods 0.000 claims description 26
- 238000004519 manufacturing process Methods 0.000 claims description 23
- 210000003527 eukaryotic cell Anatomy 0.000 claims description 22
- 230000001717 pathogenic effect Effects 0.000 claims description 22
- 238000001415 gene therapy Methods 0.000 claims description 21
- 244000052769 pathogen Species 0.000 claims description 21
- 208000015181 infectious disease Diseases 0.000 claims description 17
- 201000010099 disease Diseases 0.000 claims description 16
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 16
- 238000003780 insertion Methods 0.000 claims description 15
- 230000037431 insertion Effects 0.000 claims description 15
- 241000206602 Eukaryota Species 0.000 claims description 13
- 238000011282 treatment Methods 0.000 claims description 12
- 230000003612 virological effect Effects 0.000 claims description 8
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 6
- 241000894007 species Species 0.000 claims description 6
- 235000003869 genetically modified organism Nutrition 0.000 claims description 5
- 244000052613 viral pathogen Species 0.000 claims description 5
- 210000001938 protoplast Anatomy 0.000 claims description 3
- 210000002459 blastocyst Anatomy 0.000 claims description 2
- 210000001671 embryonic stem cell Anatomy 0.000 claims description 2
- 101100162704 Emericella nidulans I-AniI gene Proteins 0.000 claims 5
- 102000004190 Enzymes Human genes 0.000 abstract description 23
- 108090000790 Enzymes Proteins 0.000 abstract description 23
- 239000000833 heterodimer Substances 0.000 abstract description 23
- 230000004927 fusion Effects 0.000 description 94
- 235000001014 amino acid Nutrition 0.000 description 78
- 108020004414 DNA Proteins 0.000 description 59
- 235000018102 proteins Nutrition 0.000 description 57
- 230000000694 effects Effects 0.000 description 50
- 101100545004 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) YSP2 gene Proteins 0.000 description 40
- 101001018097 Homo sapiens L-selectin Proteins 0.000 description 37
- 102100033467 L-selectin Human genes 0.000 description 37
- 239000000178 monomer Substances 0.000 description 30
- CXOFVDLJLONNDW-UHFFFAOYSA-N Phenytoin Chemical compound N1C(=O)NC(=O)C1(C=1C=CC=CC=1)C1=CC=CC=C1 CXOFVDLJLONNDW-UHFFFAOYSA-N 0.000 description 22
- 108010042407 Endonucleases Proteins 0.000 description 17
- 239000013598 vector Substances 0.000 description 17
- 241000196324 Embryophyta Species 0.000 description 16
- 230000014509 gene expression Effects 0.000 description 15
- 230000004568 DNA-binding Effects 0.000 description 13
- 125000003275 alpha amino acid group Chemical group 0.000 description 12
- 230000001105 regulatory effect Effects 0.000 description 12
- 241000588724 Escherichia coli Species 0.000 description 11
- 230000002209 hydrophobic effect Effects 0.000 description 11
- 230000035772 mutation Effects 0.000 description 11
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 description 10
- 241000700605 Viruses Species 0.000 description 10
- 102100031780 Endonuclease Human genes 0.000 description 9
- 230000000875 corresponding effect Effects 0.000 description 9
- 239000000710 homodimer Substances 0.000 description 9
- RAXXELZNTBOGNW-UHFFFAOYSA-N imidazole Natural products C1=CNC=N1 RAXXELZNTBOGNW-UHFFFAOYSA-N 0.000 description 9
- 239000013612 plasmid Substances 0.000 description 9
- 101100289894 Caenorhabditis elegans lys-7 gene Proteins 0.000 description 8
- 102000004533 Endonucleases Human genes 0.000 description 8
- 101710163270 Nuclease Proteins 0.000 description 8
- 108091081021 Sense strand Proteins 0.000 description 8
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 8
- 239000002502 liposome Substances 0.000 description 8
- 102100031151 C-C chemokine receptor type 2 Human genes 0.000 description 7
- 101710149815 C-C chemokine receptor type 2 Proteins 0.000 description 7
- 108091081548 Palindromic sequence Proteins 0.000 description 7
- 239000002253 acid Substances 0.000 description 7
- 150000007513 acids Chemical class 0.000 description 7
- 230000001580 bacterial effect Effects 0.000 description 7
- 238000013461 design Methods 0.000 description 7
- 239000000203 mixture Substances 0.000 description 7
- 238000000746 purification Methods 0.000 description 7
- 238000002415 sodium dodecyl sulfate polyacrylamide gel electrophoresis Methods 0.000 description 7
- 239000000758 substrate Substances 0.000 description 7
- 230000007018 DNA scission Effects 0.000 description 6
- 206010028980 Neoplasm Diseases 0.000 description 6
- 230000000692 anti-sense effect Effects 0.000 description 6
- 238000013459 approach Methods 0.000 description 6
- 238000004422 calculation algorithm Methods 0.000 description 6
- 238000012217 deletion Methods 0.000 description 6
- 230000037430 deletion Effects 0.000 description 6
- 230000002068 genetic effect Effects 0.000 description 6
- 238000010353 genetic engineering Methods 0.000 description 6
- 238000000338 in vitro Methods 0.000 description 6
- 230000007246 mechanism Effects 0.000 description 6
- 239000000047 product Substances 0.000 description 6
- -1 proline amino acids Chemical class 0.000 description 6
- 230000009261 transgenic effect Effects 0.000 description 6
- 125000001433 C-terminal amino-acid group Chemical group 0.000 description 5
- 101100399480 Caenorhabditis elegans lmn-1 gene Proteins 0.000 description 5
- 102000053602 DNA Human genes 0.000 description 5
- 201000011510 cancer Diseases 0.000 description 5
- 230000003197 catalytic effect Effects 0.000 description 5
- 108020001507 fusion proteins Proteins 0.000 description 5
- 102000037865 fusion proteins Human genes 0.000 description 5
- 238000005304 joining Methods 0.000 description 5
- 239000002244 precipitate Substances 0.000 description 5
- 230000006798 recombination Effects 0.000 description 5
- 238000005215 recombination Methods 0.000 description 5
- 238000006467 substitution reaction Methods 0.000 description 5
- 210000001519 tissue Anatomy 0.000 description 5
- 241000894006 Bacteria Species 0.000 description 4
- 239000004471 Glycine Substances 0.000 description 4
- 241001465754 Metazoa Species 0.000 description 4
- 125000000729 N-terminal amino-acid group Chemical group 0.000 description 4
- ONIBWKKTOPOVIA-UHFFFAOYSA-N Proline Natural products OC(=O)C1CCCN1 ONIBWKKTOPOVIA-UHFFFAOYSA-N 0.000 description 4
- 241000220317 Rosa Species 0.000 description 4
- 239000005441 aurora Substances 0.000 description 4
- 230000027455 binding Effects 0.000 description 4
- 238000010367 cloning Methods 0.000 description 4
- 238000011161 development Methods 0.000 description 4
- 239000000539 dimer Substances 0.000 description 4
- 238000006471 dimerization reaction Methods 0.000 description 4
- 235000013601 eggs Nutrition 0.000 description 4
- 238000005516 engineering process Methods 0.000 description 4
- 238000011534 incubation Methods 0.000 description 4
- 230000001404 mediated effect Effects 0.000 description 4
- 239000002773 nucleotide Substances 0.000 description 4
- 125000003729 nucleotide group Chemical group 0.000 description 4
- 230000008439 repair process Effects 0.000 description 4
- 239000011780 sodium chloride Substances 0.000 description 4
- 238000013518 transcription Methods 0.000 description 4
- 230000035897 transcription Effects 0.000 description 4
- 238000001890 transfection Methods 0.000 description 4
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 3
- 108700028369 Alleles Proteins 0.000 description 3
- 230000033616 DNA repair Effects 0.000 description 3
- 241000233866 Fungi Species 0.000 description 3
- ONIBWKKTOPOVIA-BYPYZUCNSA-N L-Proline Chemical compound OC(=O)[C@@H]1CCCN1 ONIBWKKTOPOVIA-BYPYZUCNSA-N 0.000 description 3
- 241000713869 Moloney murine leukemia virus Species 0.000 description 3
- 108091005804 Peptidases Proteins 0.000 description 3
- 239000004365 Protease Substances 0.000 description 3
- 240000004808 Saccharomyces cerevisiae Species 0.000 description 3
- 125000000539 amino acid group Chemical group 0.000 description 3
- 238000004458 analytical method Methods 0.000 description 3
- 238000003556 assay Methods 0.000 description 3
- 239000000872 buffer Substances 0.000 description 3
- 238000006555 catalytic reaction Methods 0.000 description 3
- 230000002759 chromosomal effect Effects 0.000 description 3
- 239000003814 drug Substances 0.000 description 3
- 230000037433 frameshift Effects 0.000 description 3
- 238000012239 gene modification Methods 0.000 description 3
- 238000007429 general method Methods 0.000 description 3
- 238000010362 genome editing Methods 0.000 description 3
- 150000002333 glycines Chemical class 0.000 description 3
- 230000012010 growth Effects 0.000 description 3
- 230000004962 physiological condition Effects 0.000 description 3
- 230000017854 proteolysis Effects 0.000 description 3
- 238000011160 research Methods 0.000 description 3
- 108091008146 restriction endonucleases Proteins 0.000 description 3
- 102220232161 rs1085307160 Human genes 0.000 description 3
- 238000002864 sequence alignment Methods 0.000 description 3
- 230000003584 silencer Effects 0.000 description 3
- 230000003007 single stranded DNA break Effects 0.000 description 3
- 239000000126 substance Substances 0.000 description 3
- 238000012360 testing method Methods 0.000 description 3
- QKNYBSVHEMOAJP-UHFFFAOYSA-N 2-amino-2-(hydroxymethyl)propane-1,3-diol;hydron;chloride Chemical compound Cl.OCC(N)(CO)CO QKNYBSVHEMOAJP-UHFFFAOYSA-N 0.000 description 2
- 241000193738 Bacillus anthracis Species 0.000 description 2
- 241000222120 Candida <Saccharomycetales> Species 0.000 description 2
- 108020004705 Codon Proteins 0.000 description 2
- 241000255581 Drosophila <fruit fly, genus> Species 0.000 description 2
- 108091029865 Exogenous DNA Proteins 0.000 description 2
- 241000701044 Human gammaherpesvirus 4 Species 0.000 description 2
- 241000701806 Human papillomavirus Species 0.000 description 2
- 241000699670 Mus sp. Species 0.000 description 2
- 108010077850 Nuclear Localization Signals Proteins 0.000 description 2
- 102000035195 Peptidases Human genes 0.000 description 2
- 241000191967 Staphylococcus aureus Species 0.000 description 2
- 240000008042 Zea mays Species 0.000 description 2
- 235000002017 Zea mays subsp mays Nutrition 0.000 description 2
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 description 2
- 238000013019 agitation Methods 0.000 description 2
- 230000006907 apoptotic process Effects 0.000 description 2
- 239000012148 binding buffer Substances 0.000 description 2
- 230000033077 cellular process Effects 0.000 description 2
- 238000005119 centrifugation Methods 0.000 description 2
- 230000008859 change Effects 0.000 description 2
- 210000003763 chloroplast Anatomy 0.000 description 2
- 230000004186 co-expression Effects 0.000 description 2
- 239000013078 crystal Substances 0.000 description 2
- 230000003247 decreasing effect Effects 0.000 description 2
- 230000002950 deficient Effects 0.000 description 2
- 239000005546 dideoxynucleotide Substances 0.000 description 2
- 239000012636 effector Substances 0.000 description 2
- 238000004520 electroporation Methods 0.000 description 2
- 239000012149 elution buffer Substances 0.000 description 2
- 239000003623 enhancer Substances 0.000 description 2
- 238000002474 experimental method Methods 0.000 description 2
- 238000009472 formulation Methods 0.000 description 2
- 239000012634 fragment Substances 0.000 description 2
- 238000013467 fragmentation Methods 0.000 description 2
- 238000006062 fragmentation reaction Methods 0.000 description 2
- 238000012248 genetic selection Methods 0.000 description 2
- 210000004602 germ cell Anatomy 0.000 description 2
- 125000003630 glycyl group Chemical group [H]N([H])C([H])([H])C(*)=O 0.000 description 2
- 238000005734 heterodimerization reaction Methods 0.000 description 2
- 230000005661 hydrophobic surface Effects 0.000 description 2
- 238000002347 injection Methods 0.000 description 2
- 239000007924 injection Substances 0.000 description 2
- 238000004949 mass spectrometry Methods 0.000 description 2
- 238000000520 microinjection Methods 0.000 description 2
- 230000008520 organization Effects 0.000 description 2
- 239000008188 pellet Substances 0.000 description 2
- 230000023603 positive regulation of transcription initiation, DNA-dependent Effects 0.000 description 2
- 230000008569 process Effects 0.000 description 2
- 108020001580 protein domains Proteins 0.000 description 2
- 230000006337 proteolytic cleavage Effects 0.000 description 2
- 230000033458 reproduction Effects 0.000 description 2
- 230000001177 retroviral effect Effects 0.000 description 2
- 102220232162 rs1085307161 Human genes 0.000 description 2
- 238000012216 screening Methods 0.000 description 2
- 238000012163 sequencing technique Methods 0.000 description 2
- 239000002904 solvent Substances 0.000 description 2
- 230000004936 stimulating effect Effects 0.000 description 2
- 238000003860 storage Methods 0.000 description 2
- 230000001225 therapeutic effect Effects 0.000 description 2
- 230000001988 toxicity Effects 0.000 description 2
- 231100000419 toxicity Toxicity 0.000 description 2
- 230000009466 transformation Effects 0.000 description 2
- 238000013519 translation Methods 0.000 description 2
- 239000011701 zinc Substances 0.000 description 2
- 229910052725 zinc Inorganic materials 0.000 description 2
- OSJPPGNTCRNQQC-UWTATZPHSA-N 3-phospho-D-glyceric acid Chemical compound OC(=O)[C@H](O)COP(O)(O)=O OSJPPGNTCRNQQC-UWTATZPHSA-N 0.000 description 1
- 241000251468 Actinopterygii Species 0.000 description 1
- 229920000936 Agarose Polymers 0.000 description 1
- 241000589158 Agrobacterium Species 0.000 description 1
- 102100027211 Albumin Human genes 0.000 description 1
- 108010088751 Albumins Proteins 0.000 description 1
- 241000024188 Andala Species 0.000 description 1
- 241000219194 Arabidopsis Species 0.000 description 1
- 244000105624 Arachis hypogaea Species 0.000 description 1
- 241000182988 Assa Species 0.000 description 1
- 102000052609 BRCA2 Human genes 0.000 description 1
- 108700020462 BRCA2 Proteins 0.000 description 1
- 241000335423 Blastomyces Species 0.000 description 1
- 241000283690 Bos taurus Species 0.000 description 1
- 101150008921 Brca2 gene Proteins 0.000 description 1
- 101100335897 Caenorhabditis elegans gly-9 gene Proteins 0.000 description 1
- 240000001432 Calendula officinalis Species 0.000 description 1
- 235000005881 Calendula officinalis Nutrition 0.000 description 1
- 241000282472 Canis lupus familiaris Species 0.000 description 1
- 241000283707 Capra Species 0.000 description 1
- 241000700198 Cavia Species 0.000 description 1
- 206010008342 Cervix carcinoma Diseases 0.000 description 1
- 108091026890 Coding region Proteins 0.000 description 1
- 206010010356 Congenital anomaly Diseases 0.000 description 1
- 241001337994 Cryptococcus <scale insect> Species 0.000 description 1
- 108010063593 DNA modification methylase SssI Proteins 0.000 description 1
- 102000052510 DNA-Binding Proteins Human genes 0.000 description 1
- 108700020911 DNA-Binding Proteins Proteins 0.000 description 1
- 241000702421 Dependoparvovirus Species 0.000 description 1
- KCXVZYZYPLLWCC-UHFFFAOYSA-N EDTA Chemical compound OC(=O)CN(CC(O)=O)CCN(CC(O)=O)CC(O)=O KCXVZYZYPLLWCC-UHFFFAOYSA-N 0.000 description 1
- 108010067770 Endopeptidase K Proteins 0.000 description 1
- 241000991587 Enterovirus C Species 0.000 description 1
- 241000283086 Equidae Species 0.000 description 1
- 108700039887 Essential Genes Proteins 0.000 description 1
- 241000282326 Felis catus Species 0.000 description 1
- 102100039556 Galectin-4 Human genes 0.000 description 1
- WCORRBXVISTKQL-WHFBIAKZSA-N Gly-Ser-Ser Chemical group NCC(=O)N[C@@H](CO)C(=O)N[C@@H](CO)C(O)=O WCORRBXVISTKQL-WHFBIAKZSA-N 0.000 description 1
- 244000068988 Glycine max Species 0.000 description 1
- 235000010469 Glycine max Nutrition 0.000 description 1
- 102000009465 Growth Factor Receptors Human genes 0.000 description 1
- 108010009202 Growth Factor Receptors Proteins 0.000 description 1
- 241000288105 Grus Species 0.000 description 1
- 241000606790 Haemophilus Species 0.000 description 1
- 108010068250 Herpes Simplex Virus Protein Vmw65 Proteins 0.000 description 1
- 241000238631 Hexapoda Species 0.000 description 1
- MAJYPBAJPNUFPV-BQBZGAKWSA-N His-Cys Chemical compound SC[C@@H](C(O)=O)NC(=O)[C@@H](N)CC1=CN=CN1 MAJYPBAJPNUFPV-BQBZGAKWSA-N 0.000 description 1
- 102000003893 Histone acetyltransferases Human genes 0.000 description 1
- 108090000246 Histone acetyltransferases Proteins 0.000 description 1
- 102000003964 Histone deacetylase Human genes 0.000 description 1
- 108090000353 Histone deacetylase Proteins 0.000 description 1
- 102100039999 Histone deacetylase 2 Human genes 0.000 description 1
- 241000228402 Histoplasma Species 0.000 description 1
- 241000282412 Homo Species 0.000 description 1
- 101100166894 Homo sapiens CFTR gene Proteins 0.000 description 1
- 101000608765 Homo sapiens Galectin-4 Proteins 0.000 description 1
- 101001035011 Homo sapiens Histone deacetylase 2 Proteins 0.000 description 1
- 101000904868 Homo sapiens Transcriptional regulator ATRX Proteins 0.000 description 1
- 108010070875 Human Immunodeficiency Virus tat Gene Products Proteins 0.000 description 1
- 241000700588 Human alphaherpesvirus 1 Species 0.000 description 1
- 241000701074 Human alphaherpesvirus 2 Species 0.000 description 1
- 241000713772 Human immunodeficiency virus 1 Species 0.000 description 1
- 241000713340 Human immunodeficiency virus 2 Species 0.000 description 1
- 108060003951 Immunoglobulin Proteins 0.000 description 1
- 102100034353 Integrase Human genes 0.000 description 1
- 108091092195 Intron Proteins 0.000 description 1
- QNAYBMKLOCPYGJ-REOHCLBHSA-N L-alanine Chemical compound C[C@H](N)C(O)=O QNAYBMKLOCPYGJ-REOHCLBHSA-N 0.000 description 1
- 235000014647 Lens culinaris subsp culinaris Nutrition 0.000 description 1
- 244000043158 Lens esculenta Species 0.000 description 1
- 241000270322 Lepidosauria Species 0.000 description 1
- 241000209510 Liliopsida Species 0.000 description 1
- 239000000232 Lipid Bilayer Substances 0.000 description 1
- 240000004658 Medicago sativa Species 0.000 description 1
- 235000017587 Medicago sativa ssp. sativa Nutrition 0.000 description 1
- RJQXTJLFIWVMTO-TYNCELHUSA-N Methicillin Chemical compound COC1=CC=CC(OC)=C1C(=O)N[C@@H]1C(=O)N2[C@@H](C(O)=O)C(C)(C)S[C@@H]21 RJQXTJLFIWVMTO-TYNCELHUSA-N 0.000 description 1
- 241001529936 Murinae Species 0.000 description 1
- 101710135898 Myc proto-oncogene protein Proteins 0.000 description 1
- 102100038895 Myc proto-oncogene protein Human genes 0.000 description 1
- 241000204031 Mycoplasma Species 0.000 description 1
- 108091061960 Naked DNA Proteins 0.000 description 1
- 208000002454 Nasopharyngeal Carcinoma Diseases 0.000 description 1
- 208000009869 Neu-Laxova syndrome Diseases 0.000 description 1
- 102000008763 Neurofilament Proteins Human genes 0.000 description 1
- 108010088373 Neurofilament Proteins Proteins 0.000 description 1
- 241000221960 Neurospora Species 0.000 description 1
- 101100355599 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) mus-11 gene Proteins 0.000 description 1
- 244000061176 Nicotiana tabacum Species 0.000 description 1
- 235000002637 Nicotiana tabacum Nutrition 0.000 description 1
- 108010066154 Nuclear Export Signals Proteins 0.000 description 1
- 108091005461 Nucleic proteins Proteins 0.000 description 1
- 108091034117 Oligonucleotide Proteins 0.000 description 1
- 102000043276 Oncogene Human genes 0.000 description 1
- 108700020796 Oncogene Proteins 0.000 description 1
- 240000007594 Oryza sativa Species 0.000 description 1
- 235000007164 Oryza sativa Nutrition 0.000 description 1
- 241001494479 Pecora Species 0.000 description 1
- 244000046052 Phaseolus vulgaris Species 0.000 description 1
- 108091000080 Phosphotransferase Proteins 0.000 description 1
- 240000004713 Pisum sativum Species 0.000 description 1
- 235000010582 Pisum sativum Nutrition 0.000 description 1
- 208000001873 Pseudoaminopterin syndrome Diseases 0.000 description 1
- 101150006234 RAD52 gene Proteins 0.000 description 1
- 102000002490 Rad51 Recombinase Human genes 0.000 description 1
- 108010068097 Rad51 Recombinase Proteins 0.000 description 1
- 102000053062 Rad52 DNA Repair and Recombination Human genes 0.000 description 1
- 108700031762 Rad52 DNA Repair and Recombination Proteins 0.000 description 1
- 241000700159 Rattus Species 0.000 description 1
- 108010008281 Recombinant Fusion Proteins Proteins 0.000 description 1
- 102000007056 Recombinant Fusion Proteins Human genes 0.000 description 1
- 102100037486 Reverse transcriptase/ribonuclease H Human genes 0.000 description 1
- 241000235070 Saccharomyces Species 0.000 description 1
- 101100523532 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) RAD57 gene Proteins 0.000 description 1
- 108091061939 Selfish DNA Proteins 0.000 description 1
- 229920002684 Sepharose Polymers 0.000 description 1
- 241000194017 Streptococcus Species 0.000 description 1
- 241000282887 Suidae Species 0.000 description 1
- 108091008874 T cell receptors Proteins 0.000 description 1
- 102000016266 T-Cell Antigen Receptors Human genes 0.000 description 1
- 102100023931 Transcriptional regulator ATRX Human genes 0.000 description 1
- 101710150448 Transcriptional regulator Myc Proteins 0.000 description 1
- 241000219793 Trifolium Species 0.000 description 1
- 235000021307 Triticum Nutrition 0.000 description 1
- 244000098338 Triticum aestivum Species 0.000 description 1
- 208000006105 Uterine Cervical Neoplasms Diseases 0.000 description 1
- 241000700647 Variola virus Species 0.000 description 1
- 108700005077 Viral Genes Proteins 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
- 235000005824 Zea mays ssp. parviglumis Nutrition 0.000 description 1
- 235000016383 Zea mays subsp huehuetenangensis Nutrition 0.000 description 1
- 108010017070 Zinc Finger Nucleases Proteins 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
- 230000001594 aberrant effect Effects 0.000 description 1
- 230000002159 abnormal effect Effects 0.000 description 1
- 238000002835 absorbance Methods 0.000 description 1
- 239000012190 activator Substances 0.000 description 1
- 230000002411 adverse Effects 0.000 description 1
- 235000004279 alanine Nutrition 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
- 229940065181 bacillus anthracis Drugs 0.000 description 1
- 244000052616 bacterial pathogen Species 0.000 description 1
- 230000003115 biocidal effect Effects 0.000 description 1
- 230000008033 biological extinction Effects 0.000 description 1
- 230000033228 biological regulation Effects 0.000 description 1
- 230000015572 biosynthetic process Effects 0.000 description 1
- 239000001678 brown HT Substances 0.000 description 1
- 238000005251 capillar electrophoresis Methods 0.000 description 1
- 230000015556 catabolic process Effects 0.000 description 1
- 230000001413 cellular effect Effects 0.000 description 1
- 230000004700 cellular uptake Effects 0.000 description 1
- 201000010881 cervical cancer Diseases 0.000 description 1
- 238000006243 chemical reaction Methods 0.000 description 1
- 230000011088 chloroplast localization Effects 0.000 description 1
- 238000004590 computer program Methods 0.000 description 1
- 238000010276 construction Methods 0.000 description 1
- 235000005822 corn Nutrition 0.000 description 1
- 230000002596 correlated effect Effects 0.000 description 1
- 210000000805 cytoplasm Anatomy 0.000 description 1
- 230000006378 damage Effects 0.000 description 1
- 239000007857 degradation product Substances 0.000 description 1
- 238000006731 degradation reaction Methods 0.000 description 1
- 230000002939 deleterious effect Effects 0.000 description 1
- 238000011033 desalting Methods 0.000 description 1
- 230000001687 destabilization Effects 0.000 description 1
- 238000010586 diagram Methods 0.000 description 1
- 238000009510 drug design Methods 0.000 description 1
- 238000001962 electrophoresis Methods 0.000 description 1
- 238000004836 empirical method Methods 0.000 description 1
- 108010047072 endodeoxyribonuclease I-CeuI Proteins 0.000 description 1
- 108010078428 env Gene Products Proteins 0.000 description 1
- 230000002255 enzymatic effect Effects 0.000 description 1
- ZMMJGEGLRURXTF-UHFFFAOYSA-N ethidium bromide Chemical compound [Br-].C12=CC(N)=CC=C2C2=CC=C(N)C=C2[N+](CC)=C1C1=CC=CC=C1 ZMMJGEGLRURXTF-UHFFFAOYSA-N 0.000 description 1
- 229960005542 ethidium bromide Drugs 0.000 description 1
- 241001233957 eudicotyledons Species 0.000 description 1
- 238000011156 evaluation Methods 0.000 description 1
- 239000013604 expression vector Substances 0.000 description 1
- 239000000835 fiber Substances 0.000 description 1
- 231100000221 frame shift mutation induction Toxicity 0.000 description 1
- 230000002538 fungal effect Effects 0.000 description 1
- 238000001502 gel electrophoresis Methods 0.000 description 1
- 238000001476 gene delivery Methods 0.000 description 1
- 238000003205 genotyping method Methods 0.000 description 1
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 description 1
- 125000001165 hydrophobic group Chemical group 0.000 description 1
- 125000002887 hydroxy group Chemical group [H]O* 0.000 description 1
- 102000018358 immunoglobulin Human genes 0.000 description 1
- 229940072221 immunoglobulins Drugs 0.000 description 1
- 230000006872 improvement Effects 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 230000002779 inactivation Effects 0.000 description 1
- 230000002458 infectious effect Effects 0.000 description 1
- 230000005764 inhibitory process Effects 0.000 description 1
- 238000007689 inspection Methods 0.000 description 1
- 230000017730 intein-mediated protein splicing Effects 0.000 description 1
- 230000003993 interaction Effects 0.000 description 1
- 238000002955 isolation Methods 0.000 description 1
- BPHPUYQFMNQIOC-NXRLNHOXSA-N isopropyl beta-D-thiogalactopyranoside Chemical compound CC(C)S[C@@H]1O[C@H](CO)[C@H](O)[C@H](O)[C@H]1O BPHPUYQFMNQIOC-NXRLNHOXSA-N 0.000 description 1
- 235000021332 kidney beans Nutrition 0.000 description 1
- 235000021374 legumes Nutrition 0.000 description 1
- 231100001231 less toxic Toxicity 0.000 description 1
- 231100000518 lethal Toxicity 0.000 description 1
- 230000001665 lethal effect Effects 0.000 description 1
- 239000003446 ligand Substances 0.000 description 1
- 238000001638 lipofection Methods 0.000 description 1
- 210000004185 liver Anatomy 0.000 description 1
- 230000004807 localization Effects 0.000 description 1
- 230000007774 longterm Effects 0.000 description 1
- 238000012423 maintenance Methods 0.000 description 1
- 235000009973 maize Nutrition 0.000 description 1
- 210000004962 mammalian cell Anatomy 0.000 description 1
- 210000005075 mammary gland Anatomy 0.000 description 1
- 239000011159 matrix material Substances 0.000 description 1
- 238000001840 matrix-assisted laser desorption--ionisation time-of-flight mass spectrometry Methods 0.000 description 1
- MYWUZJCMWCOHBA-VIFPVBQESA-N methamphetamine Chemical compound CN[C@@H](C)CC1=CC=CC=C1 MYWUZJCMWCOHBA-VIFPVBQESA-N 0.000 description 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 description 1
- 229960003085 meticillin Drugs 0.000 description 1
- 235000013336 milk Nutrition 0.000 description 1
- 239000008267 milk Substances 0.000 description 1
- 210000004080 milk Anatomy 0.000 description 1
- 230000025608 mitochondrion localization Effects 0.000 description 1
- 238000002156 mixing Methods 0.000 description 1
- 231100000219 mutagenic Toxicity 0.000 description 1
- 230000003505 mutagenic effect Effects 0.000 description 1
- 210000005044 neurofilament Anatomy 0.000 description 1
- 210000002569 neuron Anatomy 0.000 description 1
- 230000003287 optical effect Effects 0.000 description 1
- 210000003101 oviduct Anatomy 0.000 description 1
- 210000000496 pancreas Anatomy 0.000 description 1
- 230000037361 pathway Effects 0.000 description 1
- 235000020232 peanut Nutrition 0.000 description 1
- 102000020233 phosphotransferase Human genes 0.000 description 1
- 238000000053 physical method Methods 0.000 description 1
- 108010089520 pol Gene Products Proteins 0.000 description 1
- 239000004175 ponceau 4R Substances 0.000 description 1
- 230000002028 premature Effects 0.000 description 1
- 235000019419 proteases Nutrition 0.000 description 1
- 230000012846 protein folding Effects 0.000 description 1
- 238000001273 protein sequence alignment Methods 0.000 description 1
- 235000021251 pulses Nutrition 0.000 description 1
- 108700022487 rRNA Genes Proteins 0.000 description 1
- 101150079601 recA gene Proteins 0.000 description 1
- 102000005962 receptors Human genes 0.000 description 1
- 108020003175 receptors Proteins 0.000 description 1
- 239000004180 red 2G Substances 0.000 description 1
- 230000009467 reduction Effects 0.000 description 1
- 230000010076 replication Effects 0.000 description 1
- 238000012827 research and development Methods 0.000 description 1
- 230000028617 response to DNA damage stimulus Effects 0.000 description 1
- 235000009566 rice Nutrition 0.000 description 1
- 102220232200 rs1085307201 Human genes 0.000 description 1
- 102220232205 rs1085307204 Human genes 0.000 description 1
- 102200155456 rs35947557 Human genes 0.000 description 1
- 102200118234 rs36038739 Human genes 0.000 description 1
- HBMJWWWQQXIZIP-UHFFFAOYSA-N silicon carbide Chemical compound [Si+]#[C-] HBMJWWWQQXIZIP-UHFFFAOYSA-N 0.000 description 1
- 229910010271 silicon carbide Inorganic materials 0.000 description 1
- 238000002741 site-directed mutagenesis Methods 0.000 description 1
- 238000000527 sonication Methods 0.000 description 1
- 125000006850 spacer group Chemical group 0.000 description 1
- 238000010186 staining Methods 0.000 description 1
- 210000000130 stem cell Anatomy 0.000 description 1
- 230000004960 subcellular localization Effects 0.000 description 1
- 230000006918 subunit interaction Effects 0.000 description 1
- 239000006228 supernatant Substances 0.000 description 1
- 230000004083 survival effect Effects 0.000 description 1
- 230000009897 systematic effect Effects 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
- 231100000331 toxic Toxicity 0.000 description 1
- 230000002588 toxic effect Effects 0.000 description 1
- 108700012359 toxins Proteins 0.000 description 1
- 230000002103 transcriptional effect Effects 0.000 description 1
- 238000012546 transfer Methods 0.000 description 1
- 201000008827 tuberculosis Diseases 0.000 description 1
- 210000004881 tumor cell Anatomy 0.000 description 1
- 230000005740 tumor formation Effects 0.000 description 1
- 241000701161 unidentified adenovirus Species 0.000 description 1
- 241001515965 unidentified phage Species 0.000 description 1
- 239000013603 viral vector Substances 0.000 description 1
- 238000003260 vortexing Methods 0.000 description 1
- 239000011534 wash buffer Substances 0.000 description 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/04—Antibacterial agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P33/00—Antiparasitic agents
- A61P33/02—Antiprotozoals, e.g. for leishmaniasis, trichomoniasis, toxoplasmosis
-
- 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
- C12N15/63—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
-
- 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
-
- 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)
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/04—Phosphoric diester hydrolases (3.1.4)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Communicable Diseases (AREA)
- Oncology (AREA)
- Mycology (AREA)
- Cell Biology (AREA)
- Tropical Medicine & Parasitology (AREA)
- Virology (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US124707P | 2007-10-31 | 2007-10-31 | |
| US61/001,247 | 2007-10-31 | ||
| PCT/US2008/082072 WO2009059195A2 (en) | 2007-10-31 | 2008-10-31 | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2704383A1 true CA2704383A1 (en) | 2009-05-07 |
Family
ID=40591777
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2704383A Abandoned CA2704383A1 (en) | 2007-10-31 | 2008-10-31 | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
Country Status (8)
| Country | Link |
|---|---|
| US (10) | US8445251B2 (enExample) |
| EP (4) | EP3578646A3 (enExample) |
| JP (7) | JP5761996B2 (enExample) |
| AU (1) | AU2008318430A1 (enExample) |
| CA (1) | CA2704383A1 (enExample) |
| DK (3) | DK2215223T3 (enExample) |
| ES (3) | ES2422291T3 (enExample) |
| WO (1) | WO2009059195A2 (enExample) |
Families Citing this family (131)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| EP3578646A3 (en) | 2007-10-31 | 2020-03-18 | Precision Biosciences, Inc. | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| CA2781693C (en) | 2009-11-27 | 2018-12-18 | Basf Plant Science Company Gmbh | Chimeric endonucleases and uses thereof |
| AU2010325564A1 (en) | 2009-11-27 | 2012-07-12 | Basf Plant Science Company Gmbh | Chimeric endonucleases and uses thereof |
| DE112010004582T5 (de) | 2009-11-27 | 2012-11-29 | Basf Plant Science Company Gmbh | Optimierte Endonukleasen und Anwendungen davon |
| US20130198897A1 (en) | 2010-03-22 | 2013-08-01 | Philip Morris Products S.A. | Modifying enzyme activity in plants |
| WO2012138901A1 (en) * | 2011-04-05 | 2012-10-11 | Cellectis Sa | Method for enhancing rare-cutting endonuclease efficiency and uses thereof |
| DK2714936T3 (en) | 2011-06-01 | 2019-03-25 | Prec Biosciences Inc | METHODS AND PRODUCTS FOR PRODUCING MANIPULATED MAMMAL CELL LINES WITH AMPLIFIED TRANSGENES |
| EP2612918A1 (en) | 2012-01-06 | 2013-07-10 | BASF Plant Science Company GmbH | In planta recombination |
| WO2013166113A1 (en) | 2012-05-04 | 2013-11-07 | E. I. Du Pont De Nemours And Company | Compositions and methods comprising sequences having meganuclease activity |
| EP3116533B1 (en) | 2014-03-12 | 2020-08-12 | Precision Biosciences, Inc. | Dystrophin gene exon deletion using engineered nucleases |
| CN106714826A (zh) | 2014-09-07 | 2017-05-24 | 西莱克塔生物科技公司 | 用于减弱基因表达调节抗病毒转移载体免疫应答的方法和组合物 |
| ES2905181T3 (es) | 2015-05-01 | 2022-04-07 | Prec Biosciences Inc | Deleción precisa de secuencias cromosómicas in vivo |
| CA2985816A1 (en) | 2015-05-15 | 2016-11-24 | The General Hospital Corporation | Antagonistic anti-tumor necrosis factor receptor superfamily antibodies |
| EP4115894A1 (en) | 2015-06-19 | 2023-01-11 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| JP6980534B2 (ja) | 2015-06-25 | 2021-12-15 | ザ チルドレンズ メディカル センター コーポレーション | 造血幹細胞の増大、富化、および維持に関する方法および組成物 |
| WO2017024407A1 (en) | 2015-08-11 | 2017-02-16 | The Royal Institution For The Advancement Of Learning/Mcgill University | Peptidic tgf-beta antagonists |
| EP4530354A3 (en) | 2015-09-08 | 2025-09-10 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
| ES2768984T3 (es) | 2015-10-05 | 2020-06-24 | Prec Biosciences Inc | Meganucleasas diseñadas con secuencias de reconocimiento encontradas en el gen de la región constante alfa del receptor de células T humanas |
| AU2016333898B2 (en) | 2015-10-05 | 2020-11-12 | Precision Biosciences, Inc. | Genetically-modified cells comprising a modified human T cell receptor alpha constant region gene |
| WO2017112859A1 (en) | 2015-12-23 | 2017-06-29 | Precision Biosciences, Inc. | Engineered meganucleases with recognition sequences found in the human beta-2 microglobulin gene |
| US11274288B2 (en) | 2016-02-16 | 2022-03-15 | Emendobio Inc. | Compositions and methods for promoting homology directed repair mediated gene editing |
| DK3429603T3 (da) | 2016-03-15 | 2022-03-14 | Childrens Medical Center | Fremgangsmåder og sammensætninger vedrørende ekspansion af hæmatopoietiske stamceller |
| CA3017645C (en) | 2016-04-27 | 2021-07-13 | University Of Puerto Rico | 1,5-disubstituted 1,2,3-triazoles are inhibitors of rac/cdc42 gtpases |
| JP2019514397A (ja) | 2016-05-03 | 2019-06-06 | プレシジョン バイオサイエンシズ，インク． | 血友病ａの治療に有用な操作されたヌクレアーゼ |
| MX2018015798A (es) | 2016-06-22 | 2019-07-12 | Univ North Carolina State | Método para incrementar la eficiencia de uso de nitrógeno y/o la eficiencia de utilización de nitrógeno en plantas. |
| EP3487994A4 (en) * | 2016-07-25 | 2020-01-29 | Bluebird Bio, Inc. | HOMING BCL11A ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE |
| WO2018039333A1 (en) | 2016-08-23 | 2018-03-01 | Bluebird Bio, Inc. | Tim3 homing endonuclease variants, compositions, and methods of use |
| DK3510157T5 (da) | 2016-09-08 | 2024-09-02 | 2Seventy Bio Inc | Pd-1-homing-endonucleasevarianter, sammensætninger og fremgangsmåder til anvendelse |
| AU2017338846B2 (en) | 2016-10-04 | 2020-09-24 | Precision Biosciences, Inc. | Co-stimulatory domains for use in genetically-modified cells |
| US10662416B2 (en) * | 2016-10-14 | 2020-05-26 | Precision Biosciences, Inc. | Engineered meganucleases specific for recognition sequences in the hepatitis B virus genome |
| AU2017346683B2 (en) * | 2016-10-17 | 2024-04-18 | Regeneron Pharmaceuticals, Inc. | TGFβR2 endonuclease variants, compositions, and methods of use |
| WO2018073393A2 (en) | 2016-10-19 | 2018-04-26 | Cellectis | Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy |
| JP7352807B2 (ja) | 2017-04-12 | 2023-09-29 | マジェンタ セラピューティクス インコーポレイテッド | アリール炭化水素受容体アンタゴニスト及びその使用 |
| KR20230170125A (ko) * | 2017-04-21 | 2023-12-18 | 프리시젼 바이오사이언시스 인코포레이티드 | Pcsk9 유전자 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
| WO2018201144A1 (en) | 2017-04-28 | 2018-11-01 | Precision Biosciences, Inc. | Methods for reducing dna-induced cytotoxicity and enhancing gene editing in primary cells |
| AU2018266698A1 (en) | 2017-05-08 | 2019-11-28 | Precision Biosciences, Inc. | Nucleic acid molecules encoding an engineered antigen receptor and an inhibitory nucleic acid molecule and methods of use thereof |
| KR102590466B1 (ko) | 2017-05-25 | 2023-10-19 | 2세븐티 바이오, 인코포레이티드 | Cblb 엔도뉴클레아제 변이체, 조성물 및 사용 방법 |
| CA3067842C (en) | 2017-06-23 | 2025-11-04 | Univ Kentucky Res Found | Methods of modulating the alkaloid content of a plant or part thereof |
| CA3068465A1 (en) | 2017-06-30 | 2019-01-03 | Precision Biosciences, Inc. | Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene |
| JP2020529206A (ja) | 2017-07-31 | 2020-10-08 | アール・ジエイ・レイノルズ・タバコ・カンパニー | 植物におけるウイルスベースの遺伝子編集のための方法及び組成物 |
| US20200239544A1 (en) | 2017-10-03 | 2020-07-30 | Precision Biosciences, Inc. | Modified epidermal growth factor receptor peptides for use in genetically-modified cells |
| WO2019070974A1 (en) * | 2017-10-04 | 2019-04-11 | Bluebird Bio, Inc. | PCSK9 ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE |
| AU2018347583A1 (en) | 2017-10-13 | 2020-05-21 | Selecta Biosciences, Inc. | Methods and compositions for attenuating anti-viral transfer vector IgM responses |
| EP3703715A1 (en) | 2017-10-31 | 2020-09-09 | Magenta Therapeutics, Inc. | Compositions and methods for hematopoietic stem and progenitor cell transplant therapy |
| US20190314407A1 (en) | 2017-10-31 | 2019-10-17 | Magenta Therapeutics Inc. | Compositions and methods for the expansion of hematopoietic stem and progenitor cells |
| WO2019089913A1 (en) | 2017-11-01 | 2019-05-09 | Precision Biosciences, Inc. | Engineered nucleases that target human and canine factor viii genes as a treatment for hemophilia a |
| JP2021505172A (ja) | 2017-12-06 | 2021-02-18 | マジェンタ セラピューティクス インコーポレイテッドＭａｇｅｎｔａ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． | 造血幹細胞及び前駆細胞を動員させるための投薬レジメン |
| US20210002621A1 (en) * | 2017-12-20 | 2021-01-07 | Bluebird Bio, Inc. | Ctla4 homing endonuclease variants, compositions, and methods of use |
| JP2021510150A (ja) | 2018-01-03 | 2021-04-15 | マジェンタ セラピューティクス インコーポレイテッドＭａｇｅｎｔａ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． | 造血幹細胞および前駆細胞の増幅ならびに遺伝性代謝障害の処置のための組成物および方法 |
| CN111819285B (zh) | 2018-01-09 | 2024-08-09 | 希博斯美国有限公司 | 防碎基因和突变 |
| CA3092372A1 (en) | 2018-02-26 | 2019-08-29 | AnTolRx, Inc. | Tolerogenic liposomes and methods of use thereof |
| EA202092382A1 (ru) | 2018-04-04 | 2021-02-02 | САЙБАС ЮЭс ЛЛС | Гены fad2 и мутации |
| IT201800004253A1 (it) | 2018-04-05 | 2019-10-05 | Composizioni e metodi per il trattamento della tachicardia ventricolare polimorfa catecolaminergica dominante ereditaria. | |
| KR102617818B1 (ko) | 2018-04-12 | 2023-12-27 | 프리시젼 바이오사이언시스 인코포레이티드 | 인간 t 세포 수용체 알파 불변 영역 유전자에 대한 특이성을 갖는 최적화된 조작된 뉴클레아제 |
| TW201945388A (zh) | 2018-04-12 | 2019-12-01 | 美商精密生物科學公司 | 對b型肝炎病毒基因體中之識別序列具有特異性之最佳化之經工程化巨核酸酶 |
| US20210230565A1 (en) * | 2018-04-27 | 2021-07-29 | Seattle Children's Hospital D/B/A Seattle Children's Research Institute | Bruton's tyrosine kinase homing endonuclease variants, compositions, and methods of use |
| BR112020025439A2 (pt) | 2018-06-14 | 2021-03-16 | Bluebird Bio, Inc. | Receptores de antígenos quiméricos cd79a |
| GB201812603D0 (en) | 2018-08-02 | 2018-09-19 | British American Tobacco Investments Ltd | Method |
| KR20210071976A (ko) | 2018-09-04 | 2021-06-16 | 마젠타 테라퓨틱스 인코포레이티드 | 아릴 하이드로카본 수용체 길항제 및 사용 방법 |
| WO2020072059A1 (en) * | 2018-10-04 | 2020-04-09 | Bluebird Bio, Inc. | Cblb endonuclease variants, compositions, and methods of use |
| GB201817971D0 (en) | 2018-11-02 | 2018-12-19 | British American Tobacco Investments Ltd | Method |
| GB201818715D0 (en) | 2018-11-16 | 2019-01-02 | British American Tobacco Investments Ltd | Method |
| CA3122278A1 (en) | 2018-12-10 | 2020-06-18 | Bluebird Bio, Inc. | Homing endonuclease variants |
| US20220090047A1 (en) | 2018-12-21 | 2022-03-24 | Precision Biosciences, Inc. | Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria |
| GB201900940D0 (en) | 2019-01-23 | 2019-03-13 | British American Tobacco Investments Ltd | Method |
| AU2020253538A1 (en) | 2019-04-03 | 2021-11-18 | Precision Biosciences, Inc. | Genetically-modified immune cells comprising a microRNA-adapted shRNA (shRNAmiR) |
| US20220204994A1 (en) | 2019-04-05 | 2022-06-30 | Precision Biosciences, Inc. | Methods of preparing populations of genetically-modified immune cells |
| CA3137975A1 (en) | 2019-05-07 | 2020-11-12 | Precision Biosciences, Inc. | Optimization of engineered meganucleases for recognition sequences |
| GB201906768D0 (en) | 2019-05-14 | 2019-06-26 | British American Tobacco Investments Ltd | Method |
| CN114206396A (zh) | 2019-05-28 | 2022-03-18 | 西莱克塔生物科技公司 | 用于减弱抗病毒转移载体免疫应答的方法和组合物 |
| GB201909562D0 (en) | 2019-07-03 | 2019-08-14 | British American Tobacco Investments Ltd | Method |
| GB201909563D0 (en) | 2019-07-03 | 2019-08-14 | British American Tobacco Investments Ltd | Method |
| JP2022544825A (ja) | 2019-08-20 | 2022-10-21 | プレシジョン バイオサイエンシズ，インク． | 細胞免疫療法のためのリンパ球枯渇投与レジメン |
| WO2021087305A1 (en) | 2019-10-30 | 2021-05-06 | Precision Biosciences, Inc. | Cd20 chimeric antigen receptors and methods of use for immunotherapy |
| US20220401481A1 (en) | 2019-11-01 | 2022-12-22 | Magenta Therapeutics, Inc. | Dosing regimens for the mobilization of hematopoietic stem and progenitor cells |
| FI4069729T3 (fi) | 2019-12-06 | 2025-04-01 | Prec Biosciences Inc | Optimoidut muokatut meganukleaasit, joilla on spesifisyys tunnistussekvenssiä varten hepatiitti b -virusgenomissa |
| EP3835309A1 (en) | 2019-12-13 | 2021-06-16 | KWS SAAT SE & Co. KGaA | Method for increasing cold or frost tolerance in a plant |
| WO2021158915A1 (en) | 2020-02-06 | 2021-08-12 | Precision Biosciences, Inc. | Recombinant adeno-associated virus compositions and methods for producing and using the same |
| WO2021205000A2 (en) | 2020-04-09 | 2021-10-14 | R.J. Reynolds Tobacco Company | Method |
| US20230330185A1 (en) | 2020-04-27 | 2023-10-19 | Magenta Therapeutics, Inc. | Methods and compositions for transducing hematopoietic stem and progenitor cells in vivo |
| WO2021231259A1 (en) | 2020-05-11 | 2021-11-18 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| JP2023526310A (ja) | 2020-05-12 | 2023-06-21 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 導入遺伝子発現のｄｒｇ特異的低減のための組成物 |
| US20230193230A1 (en) | 2020-05-12 | 2023-06-22 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using improved engineered meganucleases |
| EP4192875A1 (en) | 2020-08-10 | 2023-06-14 | Precision BioSciences, Inc. | Antibodies and fragments specific for b-cell maturation antigen and uses thereof |
| CA3172292A1 (en) | 2020-08-21 | 2022-02-24 | Cassandra GORSUCH | Engineered meganucleases having specificity for a recognition sequence in the transthyretin gene |
| US20230365995A1 (en) | 2020-10-07 | 2023-11-16 | Precision Biosciences, Inc. | Lipid nanoparticle compositions |
| WO2022094180A1 (en) | 2020-10-29 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Aav capsids and compositions containing same |
| EP4244342A1 (en) | 2020-11-12 | 2023-09-20 | Precision BioSciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
| CA3172161A1 (en) | 2021-01-08 | 2022-07-14 | James Jefferson Smith | Engineered meganucleases having specificity for a recognition sequence in the hydroxyacid oxidase 1 gene |
| EP4284823A1 (en) | 2021-01-28 | 2023-12-06 | Precision BioSciences, Inc. | Modulation of tgf beta signaling in genetically-modified eukaryotic cells |
| WO2022197776A1 (en) | 2021-03-16 | 2022-09-22 | Magenta Therapeutics, Inc. | Dosing regimens for hematopoietic stem cell mobilization for stem cell transplants in multiple myeloma patients |
| AU2022262778A1 (en) | 2021-04-22 | 2023-11-30 | Precision Biosciences, Inc. | Engineered meganucleases that target human mitochondrial genomes |
| EP4326861B1 (en) | 2021-04-22 | 2025-12-17 | Precision Biosciences, Inc. | Engineered meganucleases that target human mitochondrial genomes |
| WO2022226316A1 (en) | 2021-04-22 | 2022-10-27 | Precision Biosciences, Inc. | Compositions and methods for generating male sterile plants |
| AR125467A1 (es) | 2021-04-27 | 2023-07-19 | Univ Pennsylvania | Cápsides de virus adenoasociados derivados de porcinos y usos de los estos |
| WO2023064367A1 (en) | 2021-10-12 | 2023-04-20 | Selecta Biosciences, Inc. | Methods and compositions for attenuating anti-viral transfer vector igm responses |
| WO2023070003A1 (en) | 2021-10-19 | 2023-04-27 | Precision Biosciences, Inc. | Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency |
| AU2022368911A1 (en) | 2021-10-19 | 2024-05-30 | Precision Biosciences, Inc. | Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| MX2024009603A (es) | 2022-02-03 | 2024-08-14 | Nicoventures Trading Ltd | Metodo. |
| WO2023148476A1 (en) | 2022-02-03 | 2023-08-10 | Nicoventures Trading Limited | Method of modulating alkaloid content in tobacco plants |
| KR20240132133A (ko) | 2022-02-04 | 2024-09-02 | 니코벤처스 트레이딩 리미티드 | 담배 식물들에서 알칼로이드 함량을 조절하는 방법 |
| US20230357437A1 (en) | 2022-03-09 | 2023-11-09 | Selecta Biosciences, Inc. | Immunosuppressants in combination with anti-igm agents and related dosing |
| GB202205148D0 (en) | 2022-04-07 | 2022-05-25 | Nicoventures Trading Ltd | Method |
| GB202205149D0 (en) | 2022-04-07 | 2022-05-25 | Nicoventures Trading Ltd | Method |
| GB202205561D0 (en) | 2022-04-14 | 2022-06-01 | Nicoventures Trading Ltd | Method |
| GB202205562D0 (en) | 2022-04-14 | 2022-06-01 | Nicoventures Trading Ltd | Method |
| GB202206107D0 (en) | 2022-04-27 | 2022-06-08 | Nicoventures Trading Ltd | Method |
| GB202206109D0 (en) | 2022-04-27 | 2022-06-08 | Nicoventures Trading Ltd | Method |
| WO2023215498A2 (en) | 2022-05-05 | 2023-11-09 | Modernatx, Inc. | Compositions and methods for cd28 antagonism |
| WO2024015966A2 (en) | 2022-07-15 | 2024-01-18 | The Trustees Of The University Of Pennsylvania | Recombinant aav having aav clade d and clade e capsids and compositions containing same |
| WO2024056902A2 (en) | 2022-09-16 | 2024-03-21 | Christopher Shaw | Compositions and methods for treating neurological diseases |
| WO2024097639A1 (en) | 2022-10-31 | 2024-05-10 | Modernatx, Inc. | Hsa-binding antibodies and binding proteins and uses thereof |
| WO2024118866A1 (en) | 2022-12-01 | 2024-06-06 | Modernatx, Inc. | Gpc3-specific antibodies, binding domains, and related proteins and uses thereof |
| AU2023392154A1 (en) | 2022-12-14 | 2025-06-26 | King's College London | Compositions and methods for treating neurological diseases |
| AU2024206812A1 (en) | 2023-01-05 | 2025-08-14 | Precision Biosciences, Inc. | Optimized engineered meganucleases having specificity for the human t cell receptor alpha constant region gene |
| GB202300905D0 (en) | 2023-01-20 | 2023-03-08 | Nicoventures Trading Ltd | Method |
| WO2024161022A2 (en) | 2023-02-03 | 2024-08-08 | King's College London | Compositions and methods for treating neurological diseases |
| KR20250141243A (ko) | 2023-02-06 | 2025-09-26 | 블루록 테라퓨틱스 엘피 | 데그론 융합 단백질 및 그의 생산 및 사용 방법 |
| TW202448928A (zh) | 2023-03-02 | 2024-12-16 | 美商艾羅伊治療公司 | 抗cd22抗體及其用途 |
| GB202303077D0 (en) | 2023-03-02 | 2023-04-19 | Univ Oslo | Brassica plants with improved seed retention |
| AU2024255934A1 (en) | 2023-04-14 | 2025-11-20 | Precision Biosciences, Inc. | Muscle-specific expression cassettes |
| WO2024216116A1 (en) | 2023-04-14 | 2024-10-17 | Precision Biosciences, Inc. | Muscle-specific expression cassettes |
| 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 |
| 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 |
| WO2025083169A1 (en) | 2023-10-17 | 2025-04-24 | Tenpoint Therapeutics Limited | Combination of a vegf inhibitor and a complement pathway inhibitor for treating ocular disorders |
| WO2025101946A1 (en) | 2023-11-08 | 2025-05-15 | Precision Biosciences, Inc. | Polypeptide linkers for use in engineered meganucleases |
| WO2025126153A2 (en) | 2023-12-14 | 2025-06-19 | Aviadobio Ltd. | Compositions and methods for treating sod1-mediated neurological diseases |
| WO2025233894A1 (en) | 2024-05-09 | 2025-11-13 | University Of Miami | Engineered meganucleases that target human mitochondrial genomes |
Family Cites Families (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE122007000007I1 (de) | 1986-04-09 | 2007-05-16 | Genzyme Corp | Genetisch transformierte Tiere, die ein gewünschtes Protein in Milch absondern |
| US4873316A (en) | 1987-06-23 | 1989-10-10 | Biogen, Inc. | Isolation of exogenous recombinant proteins from the milk of transgenic mammals |
| US6156304A (en) | 1990-12-20 | 2000-12-05 | University Of Pittsburgh Of The Commonwealth System Of Higher Education | Gene transfer for studying and treating a connective tissue of a mammalian host |
| US5384253A (en) | 1990-12-28 | 1995-01-24 | Dekalb Genetics Corporation | Genetic transformation of maize cells by electroporation of cells pretreated with pectin degrading enzymes |
| US5997861A (en) | 1994-10-31 | 1999-12-07 | Burstein Laboratories, Inc. | Antiviral supramolecules containing target-binding molecules and therapeutic molecules bound to spectrin |
| US6056973A (en) | 1996-10-11 | 2000-05-02 | Sequus Pharmaceuticals, Inc. | Therapeutic liposome composition and method of preparation |
| US6060082A (en) | 1997-04-18 | 2000-05-09 | Massachusetts Institute Of Technology | Polymerized liposomes targeted to M cells and useful for oral or mucosal drug delivery |
| US6511676B1 (en) | 1999-11-05 | 2003-01-28 | Teni Boulikas | Therapy for human cancers using cisplatin and other drugs or genes encapsulated into liposomes |
| US6593308B2 (en) | 1999-12-03 | 2003-07-15 | The Regents Of The University Of California | Targeted drug delivery with a hyaluronan ligand |
| AU2002317771A1 (en) * | 2001-06-05 | 2002-12-16 | Cellectis | Methods for modifying the cpg content of polynucleotides |
| AU2003210996A1 (en) * | 2002-02-11 | 2003-09-04 | Zymogenetics, Inc. | Materials and methods for preparing dimeric growth factors |
| WO2003078619A1 (en) * | 2002-03-15 | 2003-09-25 | Cellectis | Hybrid and single chain meganucleases and use thereof |
| WO2009095742A1 (en) | 2008-01-31 | 2009-08-06 | Cellectis | New i-crei derived single-chain meganuclease and uses thereof |
| JP2006518372A (ja) | 2003-01-28 | 2006-08-10 | セレクティス | 脊椎動物の体組織においてエクスビボかつイントトで相同的組換えを誘発するためのメガヌクレアーゼの使用およびその応用 |
| JP4095622B2 (ja) * | 2004-03-31 | 2008-06-04 | キヤノン株式会社 | 金結合性複合タンパク質 |
| EP1591521A1 (en) * | 2004-04-30 | 2005-11-02 | Cellectis | I-Dmo I derivatives with enhanced activity at 37 degrees C and use thereof |
| DK1863909T3 (da) * | 2005-03-15 | 2010-08-30 | Cellectis | I-CreI-meganukleasevarianter med modificeret specificitet, fremgangsmåde til fremstilling og anvendelser deraf |
| WO2006097784A1 (en) | 2005-03-15 | 2006-09-21 | Cellectis | I-crei meganuclease variants with modified specificity, method of preparation and uses thereof |
| WO2007034262A1 (en) | 2005-09-19 | 2007-03-29 | Cellectis | Heterodimeric meganucleases and use thereof |
| EP1764414A1 (en) * | 2005-09-17 | 2007-03-21 | Icon Genetics AG | Plant viral particles comprising a plurality of fusion proteins consisting of a plant viral coat protein, a peptide linker and a recombinant protein and use of such plant viral particles for protein purification |
| US8021867B2 (en) * | 2005-10-18 | 2011-09-20 | Duke University | Rationally-designed meganucleases with altered sequence specificity and DNA-binding affinity |
| WO2007049095A1 (en) | 2005-10-25 | 2007-05-03 | Cellectis | Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof |
| WO2007060495A1 (en) | 2005-10-25 | 2007-05-31 | Cellectis | I-crei homing endonuclease variants having novel cleavage specificity and use thereof |
| EP1957520A4 (en) * | 2005-11-29 | 2009-05-27 | Univ Virginia Commonwealth | POLYVALENTIC CHIMERIC OSPC VACCINOGEN AND DIAGNOSTIC ANTIGEN |
| WO2007093836A1 (en) | 2006-02-13 | 2007-08-23 | Cellectis | Meganuclease variants cleaving a dna target sequence from a xp gene and uses thereof |
| GB0603099D0 (en) * | 2006-02-16 | 2006-03-29 | Lontra Environmental Technolog | Rotary piston and cylinder devices |
| WO2008010009A1 (en) | 2006-07-18 | 2008-01-24 | Cellectis | Meganuclease variants cleaving a dna target sequence from a rag gene and uses thereof |
| SG176487A1 (en) | 2006-11-14 | 2011-12-29 | Cellectis | Meganuclease variants cleaving a dna target sequence from the hprt gene and uses thereof |
| WO2008059317A1 (en) | 2006-11-14 | 2008-05-22 | Cellectis | Meganuclease variants cleaving a dna target sequence from the hprt gene and uses thereof |
| WO2008093152A1 (en) | 2007-02-01 | 2008-08-07 | Cellectis | Obligate heterodimer meganucleases and uses thereof |
| AU2007347328B2 (en) | 2007-02-19 | 2013-03-07 | Cellectis | LAGLIDADG homing endonuclease variants having novel substrate specificity and use thereof |
| WO2008102199A1 (en) * | 2007-02-20 | 2008-08-28 | Cellectis | Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof |
| WO2008149176A1 (en) * | 2007-06-06 | 2008-12-11 | Cellectis | Meganuclease variants cleaving a dna target sequence from the mouse rosa26 locus and uses thereof |
| EP2167666A2 (en) | 2007-06-29 | 2010-03-31 | Pioneer Hi-Bred International Inc. | Methods for altering the genome of a monocot plant cell |
| EP3578646A3 (en) | 2007-10-31 | 2020-03-18 | Precision Biosciences, Inc. | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| WO2009074842A1 (en) | 2007-12-13 | 2009-06-18 | Cellectis | Improved chimeric meganuclease enzymes and uses thereof |
-
2008
- 2008-10-31 EP EP19167904.2A patent/EP3578646A3/en active Pending
- 2008-10-31 WO PCT/US2008/082072 patent/WO2009059195A2/en not_active Ceased
- 2008-10-31 CA CA2704383A patent/CA2704383A1/en not_active Abandoned
- 2008-10-31 EP EP16163925.7A patent/EP3098309B1/en active Active
- 2008-10-31 ES ES08845549T patent/ES2422291T3/es active Active
- 2008-10-31 EP EP08845549.8A patent/EP2215223B1/en active Active
- 2008-10-31 AU AU2008318430A patent/AU2008318430A1/en not_active Abandoned
- 2008-10-31 JP JP2010532293A patent/JP5761996B2/ja active Active
- 2008-10-31 EP EP13165733.0A patent/EP2660317B1/en active Active
- 2008-10-31 DK DK08845549.8T patent/DK2215223T3/da active
- 2008-10-31 DK DK13165733.0T patent/DK2660317T3/en active
- 2008-10-31 ES ES13165733.0T patent/ES2575412T3/es active Active
- 2008-10-31 DK DK16163925.7T patent/DK3098309T3/da active
- 2008-10-31 ES ES16163925T patent/ES2732735T3/es active Active
-
2010
- 2010-04-30 US US12/771,163 patent/US8445251B2/en active Active
-
2013
- 2013-05-20 US US13/897,923 patent/US20130267009A1/en not_active Abandoned
-
2015
- 2015-05-28 US US14/723,840 patent/US20150337335A1/en not_active Abandoned
- 2015-06-08 JP JP2015115646A patent/JP6050861B2/ja active Active
- 2015-09-18 US US14/858,989 patent/US9434931B2/en active Active
- 2015-09-18 US US14/858,986 patent/US9340777B2/en active Active
-
2016
- 2016-04-19 US US15/132,941 patent/US10041053B2/en active Active
- 2016-09-30 JP JP2016193485A patent/JP2017051190A/ja active Pending
-
2018
- 2018-07-02 US US16/025,747 patent/US20180340160A1/en not_active Abandoned
- 2018-11-22 JP JP2018219205A patent/JP2019062898A/ja active Pending
-
2020
- 2020-10-23 US US17/079,377 patent/US20210054354A1/en not_active Abandoned
- 2020-11-27 JP JP2020196770A patent/JP2021052770A/ja active Pending
-
2022
- 2022-11-25 JP JP2022187981A patent/JP2023029894A/ja active Pending
-
2023
- 2023-03-17 US US18/185,726 patent/US12351839B2/en active Active
-
2024
- 2024-10-24 JP JP2024187133A patent/JP2025028832A/ja active Pending
-
2025
- 2025-01-17 US US19/029,758 patent/US20250154485A1/en active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US12351839B2 (en) | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences | |
| EP2484758B1 (en) | Rationally-designed meganucleases with altered sequence specificity and DNA-binding affinity | |
| US20130224863A1 (en) | Rationally-designed meganucleases with recognition sequences found in dnase hypersensitive regions of the human genome | |
| AU2024203387A1 (en) | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FZDE | Discontinued |
Effective date: 20141031 |