CN102959087B - 用于连接锌指模块的组合物 - Google Patents
用于连接锌指模块的组合物 Download PDFInfo
- Publication number
- CN102959087B CN102959087B CN201180022329.2A CN201180022329A CN102959087B CN 102959087 B CN102959087 B CN 102959087B CN 201180022329 A CN201180022329 A CN 201180022329A CN 102959087 B CN102959087 B CN 102959087B
- Authority
- CN
- China
- Prior art keywords
- joint
- zinc finger
- gene
- zfp
- seq
- 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.)
- Active
Links
- 239000000203 mixture Substances 0.000 title claims abstract description 30
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 title claims description 50
- 239000011701 zinc Substances 0.000 title claims description 50
- 229910052725 zinc Inorganic materials 0.000 title claims description 50
- 238000000034 method Methods 0.000 claims abstract description 44
- 108090000623 proteins and genes Proteins 0.000 claims description 106
- 238000005520 cutting process Methods 0.000 claims description 76
- 230000004927 fusion Effects 0.000 claims description 56
- 210000004027 cell Anatomy 0.000 claims description 42
- 101710185494 Zinc finger protein Proteins 0.000 claims description 31
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 31
- 230000001105 regulatory effect Effects 0.000 claims description 27
- 102000040430 polynucleotide Human genes 0.000 claims description 16
- 108091033319 polynucleotide Proteins 0.000 claims description 16
- 239000002157 polynucleotide Substances 0.000 claims description 16
- 125000000539 amino acid group Chemical group 0.000 claims description 14
- 230000004913 activation Effects 0.000 claims description 10
- 230000002103 transcriptional effect Effects 0.000 claims description 9
- 125000001500 prolyl group Chemical group [H]N1C([H])(C(=O)[*])C([H])([H])C([H])([H])C1([H])[H] 0.000 claims description 8
- 108700005075 Regulator Genes Proteins 0.000 claims description 6
- 230000001413 cellular effect Effects 0.000 claims description 6
- 125000000729 N-terminal amino-acid group Chemical group 0.000 claims description 4
- 125000001433 C-terminal amino-acid group Chemical group 0.000 claims description 3
- 125000000637 arginyl group Chemical group N[C@@H](CCCNC(N)=N)C(=O)* 0.000 claims description 3
- 125000000487 histidyl group Chemical group [H]N([H])C(C(=O)O*)C([H])([H])C1=C([H])N([H])C([H])=N1 0.000 claims description 2
- 125000003588 lysine group Chemical group [H]N([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])(N([H])[H])C(*)=O 0.000 claims description 2
- 230000027455 binding Effects 0.000 abstract description 42
- 230000004568 DNA-binding Effects 0.000 abstract description 20
- 102000004169 proteins and genes Human genes 0.000 description 63
- 235000018102 proteins Nutrition 0.000 description 62
- 108010017070 Zinc Finger Nucleases Proteins 0.000 description 59
- 238000002965 ELISA Methods 0.000 description 47
- 150000007523 nucleic acids Chemical class 0.000 description 40
- 108020004414 DNA Proteins 0.000 description 38
- 102000039446 nucleic acids Human genes 0.000 description 38
- 108020004707 nucleic acids Proteins 0.000 description 38
- 108090000765 processed proteins & peptides Proteins 0.000 description 30
- 238000012360 testing method Methods 0.000 description 30
- 239000002773 nucleotide Substances 0.000 description 27
- 125000003729 nucleotide group Chemical group 0.000 description 27
- 238000011160 research Methods 0.000 description 27
- 235000001014 amino acid Nutrition 0.000 description 25
- 102000004196 processed proteins & peptides Human genes 0.000 description 25
- 229920001184 polypeptide Polymers 0.000 description 24
- 238000013459 approach Methods 0.000 description 22
- 150000001413 amino acids Chemical class 0.000 description 21
- 230000008859 change Effects 0.000 description 21
- 101710163270 Nuclease Proteins 0.000 description 20
- 230000000694 effects Effects 0.000 description 17
- 125000003275 alpha amino acid group Chemical group 0.000 description 15
- 238000013461 design Methods 0.000 description 13
- 230000014509 gene expression Effects 0.000 description 13
- 210000003483 chromatin Anatomy 0.000 description 11
- 108010077544 Chromatin Proteins 0.000 description 10
- 108020004705 Codon Proteins 0.000 description 10
- 102000004190 Enzymes Human genes 0.000 description 10
- 108090000790 Enzymes Proteins 0.000 description 10
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 description 10
- 230000006870 function Effects 0.000 description 10
- 108091008146 restriction endonucleases Proteins 0.000 description 10
- 108010042407 Endonucleases Proteins 0.000 description 9
- 240000004808 Saccharomyces cerevisiae Species 0.000 description 9
- 238000003780 insertion Methods 0.000 description 9
- 230000037431 insertion Effects 0.000 description 9
- -1 intergrase Proteins 0.000 description 9
- 241000196324 Embryophyta Species 0.000 description 8
- 102100031780 Endonuclease Human genes 0.000 description 8
- ONIBWKKTOPOVIA-BYPYZUCNSA-N L-Proline Chemical compound OC(=O)[C@@H]1CCCN1 ONIBWKKTOPOVIA-BYPYZUCNSA-N 0.000 description 8
- ONIBWKKTOPOVIA-UHFFFAOYSA-N Proline Natural products OC(=O)C1CCCN1 ONIBWKKTOPOVIA-UHFFFAOYSA-N 0.000 description 8
- 238000003556 assay Methods 0.000 description 8
- 239000013612 plasmid Substances 0.000 description 8
- 239000000523 sample Substances 0.000 description 8
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 7
- 108091007916 Zinc finger transcription factors Proteins 0.000 description 6
- 102000038627 Zinc finger transcription factors Human genes 0.000 description 6
- 230000009471 action Effects 0.000 description 6
- 238000006471 dimerization reaction Methods 0.000 description 6
- 239000012634 fragment Substances 0.000 description 6
- 238000007423 screening assay Methods 0.000 description 6
- 239000000126 substance Substances 0.000 description 6
- 125000003178 carboxy group Chemical group [H]OC(*)=O 0.000 description 5
- 230000002068 genetic effect Effects 0.000 description 5
- 230000003993 interaction Effects 0.000 description 5
- 230000004048 modification Effects 0.000 description 5
- 238000012986 modification Methods 0.000 description 5
- 238000004153 renaturation Methods 0.000 description 5
- 108091026890 Coding region Proteins 0.000 description 4
- 108010033040 Histones Proteins 0.000 description 4
- 108091034117 Oligonucleotide Proteins 0.000 description 4
- NBIIXXVUZAFLBC-UHFFFAOYSA-N Phosphoric acid Chemical compound OP(O)(O)=O NBIIXXVUZAFLBC-UHFFFAOYSA-N 0.000 description 4
- 102000040945 Transcription factor Human genes 0.000 description 4
- 108091023040 Transcription factor Proteins 0.000 description 4
- 241000700605 Viruses Species 0.000 description 4
- 230000011559 double-strand break repair via nonhomologous end joining Effects 0.000 description 4
- 229930182817 methionine Natural products 0.000 description 4
- 238000002823 phage display Methods 0.000 description 4
- 239000013641 positive control Substances 0.000 description 4
- 238000013518 transcription Methods 0.000 description 4
- 230000035897 transcription Effects 0.000 description 4
- 239000004475 Arginine Substances 0.000 description 3
- 108010068250 Herpes Simplex Virus Protein Vmw65 Proteins 0.000 description 3
- 101000687346 Homo sapiens PR domain zinc finger protein 2 Proteins 0.000 description 3
- 101000915539 Homo sapiens Zinc finger protein 1 homolog Proteins 0.000 description 3
- 101000915531 Homo sapiens Zinc finger protein ZFP2 Proteins 0.000 description 3
- 102100021872 NADPH oxidase 4 Human genes 0.000 description 3
- 102000011931 Nucleoproteins Human genes 0.000 description 3
- 108010061100 Nucleoproteins Proteins 0.000 description 3
- 108010047956 Nucleosomes Proteins 0.000 description 3
- 102100024885 PR domain zinc finger protein 2 Human genes 0.000 description 3
- 102000001253 Protein Kinase Human genes 0.000 description 3
- 102100028610 Zinc finger protein 1 homolog Human genes 0.000 description 3
- 102100028612 Zinc finger protein ZFP2 Human genes 0.000 description 3
- 239000002253 acid Substances 0.000 description 3
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 description 3
- 230000000295 complement effect Effects 0.000 description 3
- 238000005516 engineering process Methods 0.000 description 3
- 108020001507 fusion proteins Proteins 0.000 description 3
- 102000037865 fusion proteins Human genes 0.000 description 3
- 239000000833 heterodimer Substances 0.000 description 3
- 238000002744 homologous recombination Methods 0.000 description 3
- 230000006801 homologous recombination Effects 0.000 description 3
- 230000002779 inactivation Effects 0.000 description 3
- 239000013642 negative control Substances 0.000 description 3
- 210000001623 nucleosome Anatomy 0.000 description 3
- 239000003755 preservative agent Substances 0.000 description 3
- 230000002335 preservative effect Effects 0.000 description 3
- 108060006633 protein kinase Proteins 0.000 description 3
- 238000006467 substitution reaction Methods 0.000 description 3
- 230000009182 swimming Effects 0.000 description 3
- 238000001890 transfection Methods 0.000 description 3
- 238000012546 transfer Methods 0.000 description 3
- 238000013519 translation Methods 0.000 description 3
- YBJHBAHKTGYVGT-ZKWXMUAHSA-N (+)-Biotin Chemical compound N1C(=O)N[C@@H]2[C@H](CCCCC(=O)O)SC[C@@H]21 YBJHBAHKTGYVGT-ZKWXMUAHSA-N 0.000 description 2
- 108010013043 Acetylesterase Proteins 0.000 description 2
- 101001030716 Arabidopsis thaliana Histone deacetylase HDT1 Proteins 0.000 description 2
- 101100438066 Arabidopsis thaliana ZFN3 gene Proteins 0.000 description 2
- 102100036279 DNA (cytosine-5)-methyltransferase 1 Human genes 0.000 description 2
- 101100049549 Enterobacteria phage P4 sid gene Proteins 0.000 description 2
- 102000006947 Histones Human genes 0.000 description 2
- 101001055144 Homo sapiens Interleukin-2 receptor subunit alpha Proteins 0.000 description 2
- 101000615488 Homo sapiens Methyl-CpG-binding domain protein 2 Proteins 0.000 description 2
- 101000915543 Homo sapiens Zinc finger protein 14 homolog Proteins 0.000 description 2
- 101000744929 Homo sapiens Zinc finger protein 205 Proteins 0.000 description 2
- 102100026878 Interleukin-2 receptor subunit alpha Human genes 0.000 description 2
- FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 description 2
- COLNVLDHVKWLRT-QMMMGPOBSA-N L-phenylalanine Chemical compound OC(=O)[C@@H](N)CC1=CC=CC=C1 COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 description 2
- 108090000364 Ligases Proteins 0.000 description 2
- 102000003960 Ligases Human genes 0.000 description 2
- 108091036060 Linker DNA Proteins 0.000 description 2
- 241001465754 Metazoa Species 0.000 description 2
- 102100021299 Methyl-CpG-binding domain protein 2 Human genes 0.000 description 2
- 108060004795 Methyltransferase Proteins 0.000 description 2
- XUYPXLNMDZIRQH-LURJTMIESA-N N-acetyl-L-methionine Chemical compound CSCC[C@@H](C(O)=O)NC(C)=O XUYPXLNMDZIRQH-LURJTMIESA-N 0.000 description 2
- 108010057466 NF-kappa B Proteins 0.000 description 2
- 102000003945 NF-kappa B Human genes 0.000 description 2
- 108020005497 Nuclear hormone receptor Proteins 0.000 description 2
- 102000007399 Nuclear hormone receptor Human genes 0.000 description 2
- 102000018120 Recombinases Human genes 0.000 description 2
- 108010091086 Recombinases Proteins 0.000 description 2
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 description 2
- 101710183280 Topoisomerase Proteins 0.000 description 2
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 description 2
- 229930003756 Vitamin B7 Natural products 0.000 description 2
- 241000589634 Xanthomonas Species 0.000 description 2
- 102100028616 Zinc finger protein 14 homolog Human genes 0.000 description 2
- 102100039959 Zinc finger protein 205 Human genes 0.000 description 2
- 108020002494 acetyltransferase Proteins 0.000 description 2
- 102000005421 acetyltransferase Human genes 0.000 description 2
- 239000012190 activator Substances 0.000 description 2
- 229910000147 aluminium phosphate Inorganic materials 0.000 description 2
- 230000000692 anti-sense effect Effects 0.000 description 2
- 125000003118 aryl group Chemical group 0.000 description 2
- 238000002306 biochemical method Methods 0.000 description 2
- 230000003197 catalytic effect Effects 0.000 description 2
- 210000003763 chloroplast Anatomy 0.000 description 2
- 230000002759 chromosomal effect Effects 0.000 description 2
- 210000000349 chromosome Anatomy 0.000 description 2
- 238000010276 construction Methods 0.000 description 2
- 238000013016 damping Methods 0.000 description 2
- 230000029087 digestion Effects 0.000 description 2
- 238000006073 displacement reaction Methods 0.000 description 2
- 230000002255 enzymatic effect Effects 0.000 description 2
- 210000003527 eukaryotic cell Anatomy 0.000 description 2
- 238000002474 experimental method Methods 0.000 description 2
- 239000012530 fluid Substances 0.000 description 2
- 238000000338 in vitro Methods 0.000 description 2
- 238000001727 in vivo Methods 0.000 description 2
- 230000006698 induction Effects 0.000 description 2
- 208000015181 infectious disease Diseases 0.000 description 2
- 230000002458 infectious effect Effects 0.000 description 2
- 238000002898 library design Methods 0.000 description 2
- 150000002632 lipids Chemical class 0.000 description 2
- 210000004962 mammalian cell Anatomy 0.000 description 2
- 239000000463 material Substances 0.000 description 2
- 108020004999 messenger RNA Proteins 0.000 description 2
- 238000010369 molecular cloning Methods 0.000 description 2
- 230000009871 nonspecific binding Effects 0.000 description 2
- 229920000642 polymer Polymers 0.000 description 2
- 238000002360 preparation method Methods 0.000 description 2
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 description 2
- 102000021127 protein binding proteins Human genes 0.000 description 2
- 108091011138 protein binding proteins Proteins 0.000 description 2
- 230000004044 response Effects 0.000 description 2
- 230000003584 silencer Effects 0.000 description 2
- 238000005728 strengthening Methods 0.000 description 2
- 239000011735 vitamin B7 Substances 0.000 description 2
- 235000011912 vitamin B7 Nutrition 0.000 description 2
- 230000005730 ADP ribosylation Effects 0.000 description 1
- 101710159080 Aconitate hydratase A Proteins 0.000 description 1
- 101710159078 Aconitate hydratase B Proteins 0.000 description 1
- BHELIUBJHYAEDK-OAIUPTLZSA-N Aspoxicillin Chemical compound C1([C@H](C(=O)N[C@@H]2C(N3[C@H](C(C)(C)S[C@@H]32)C(O)=O)=O)NC(=O)[C@H](N)CC(=O)NC)=CC=C(O)C=C1 BHELIUBJHYAEDK-OAIUPTLZSA-N 0.000 description 1
- 239000002126 C01EB10 - Adenosine Substances 0.000 description 1
- 101100290380 Caenorhabditis elegans cel-1 gene Proteins 0.000 description 1
- 102000053642 Catalytic RNA Human genes 0.000 description 1
- 108090000994 Catalytic RNA Proteins 0.000 description 1
- 235000005979 Citrus limon Nutrition 0.000 description 1
- 244000131522 Citrus pyriformis Species 0.000 description 1
- 101710155335 DELLA protein SLR1 Proteins 0.000 description 1
- 102000053602 DNA Human genes 0.000 description 1
- 108010009540 DNA (Cytosine-5-)-Methyltransferase 1 Proteins 0.000 description 1
- 102100024812 DNA (cytosine-5)-methyltransferase 3A Human genes 0.000 description 1
- 102100024810 DNA (cytosine-5)-methyltransferase 3B Human genes 0.000 description 1
- 101710123222 DNA (cytosine-5)-methyltransferase 3B Proteins 0.000 description 1
- 108010024491 DNA Methyltransferase 3A Proteins 0.000 description 1
- 108010076525 DNA Repair Enzymes Proteins 0.000 description 1
- 102100033195 DNA ligase 4 Human genes 0.000 description 1
- 230000008836 DNA modification Effects 0.000 description 1
- 102000016928 DNA-directed DNA polymerase Human genes 0.000 description 1
- 108010014303 DNA-directed DNA polymerase Proteins 0.000 description 1
- 108010054576 Deoxyribonuclease EcoRI Proteins 0.000 description 1
- 229920002307 Dextran Polymers 0.000 description 1
- 102000004533 Endonucleases Human genes 0.000 description 1
- 101000889905 Enterobacteria phage RB3 Intron-associated endonuclease 3 Proteins 0.000 description 1
- 101000889904 Enterobacteria phage T4 Defective intron-associated endonuclease 3 Proteins 0.000 description 1
- 101000889900 Enterobacteria phage T4 Intron-associated endonuclease 1 Proteins 0.000 description 1
- 101000889899 Enterobacteria phage T4 Intron-associated endonuclease 2 Proteins 0.000 description 1
- 108060002716 Exonuclease Proteins 0.000 description 1
- 241000233866 Fungi Species 0.000 description 1
- 108700007698 Genetic Terminator Regions Proteins 0.000 description 1
- 102000003886 Glycoproteins Human genes 0.000 description 1
- 108090000288 Glycoproteins Proteins 0.000 description 1
- 101710154606 Hemagglutinin Proteins 0.000 description 1
- 102100022130 High mobility group protein B3 Human genes 0.000 description 1
- 108050005231 Histone H2A Proteins 0.000 description 1
- 102000017286 Histone H2A Human genes 0.000 description 1
- 101710103773 Histone H2B Proteins 0.000 description 1
- 102100021639 Histone H2B type 1-K Human genes 0.000 description 1
- 102100039869 Histone H2B type F-S Human genes 0.000 description 1
- 102100022846 Histone acetyltransferase KAT2B Human genes 0.000 description 1
- 108090000246 Histone acetyltransferases Proteins 0.000 description 1
- 102000003893 Histone acetyltransferases Human genes 0.000 description 1
- 102000003964 Histone deacetylase Human genes 0.000 description 1
- 108090000353 Histone deacetylase Proteins 0.000 description 1
- 101000931098 Homo sapiens DNA (cytosine-5)-methyltransferase 1 Proteins 0.000 description 1
- 101001006375 Homo sapiens High mobility group nucleosome-binding domain-containing protein 4 Proteins 0.000 description 1
- 101001045794 Homo sapiens High mobility group protein B3 Proteins 0.000 description 1
- 101001035372 Homo sapiens Histone H2B type F-S Proteins 0.000 description 1
- 101001047006 Homo sapiens Histone acetyltransferase KAT2B Proteins 0.000 description 1
- 101000615495 Homo sapiens Methyl-CpG-binding domain protein 3 Proteins 0.000 description 1
- 101000802101 Homo sapiens mRNA decay activator protein ZFP36L2 Proteins 0.000 description 1
- 208000026350 Inborn Genetic disease Diseases 0.000 description 1
- 108020004684 Internal Ribosome Entry Sites Proteins 0.000 description 1
- OUYCCCASQSFEME-QMMMGPOBSA-N L-tyrosine Chemical compound OC(=O)[C@@H](N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-QMMMGPOBSA-N 0.000 description 1
- 108090001030 Lipoproteins Proteins 0.000 description 1
- 102000004895 Lipoproteins Human genes 0.000 description 1
- 241000124008 Mammalia Species 0.000 description 1
- 102000006890 Methyl-CpG-Binding Protein 2 Human genes 0.000 description 1
- 108010072388 Methyl-CpG-Binding Protein 2 Proteins 0.000 description 1
- 102100021291 Methyl-CpG-binding domain protein 3 Human genes 0.000 description 1
- 108010059724 Micrococcal Nuclease Proteins 0.000 description 1
- 108010086093 Mung Bean Nuclease Proteins 0.000 description 1
- 101000744913 Mus musculus Zinc finger protein 11 Proteins 0.000 description 1
- 108010082699 NADPH Oxidase 4 Proteins 0.000 description 1
- 206010028980 Neoplasm Diseases 0.000 description 1
- 108700019961 Neoplasm Genes Proteins 0.000 description 1
- 102000048850 Neoplasm Genes Human genes 0.000 description 1
- 108091092724 Noncoding DNA Proteins 0.000 description 1
- 108010077850 Nuclear Localization Signals Proteins 0.000 description 1
- 108091028043 Nucleic acid sequence Proteins 0.000 description 1
- 108091007494 Nucleic acid- binding domains Proteins 0.000 description 1
- XDMCWZFLLGVIID-SXPRBRBTSA-N O-(3-O-D-galactosyl-N-acetyl-beta-D-galactosaminyl)-L-serine Chemical compound CC(=O)N[C@H]1[C@H](OC[C@H]([NH3+])C([O-])=O)O[C@H](CO)[C@H](O)[C@@H]1OC1[C@H](O)[C@@H](O)[C@@H](O)[C@@H](CO)O1 XDMCWZFLLGVIID-SXPRBRBTSA-N 0.000 description 1
- 108700020796 Oncogene Proteins 0.000 description 1
- 108700026244 Open Reading Frames Proteins 0.000 description 1
- 101100046877 Oryza sativa subsp. japonica TRAB1 gene Proteins 0.000 description 1
- 101710093908 Outer capsid protein VP4 Proteins 0.000 description 1
- 101710135467 Outer capsid protein sigma-1 Proteins 0.000 description 1
- 238000012408 PCR amplification Methods 0.000 description 1
- 101100440941 Petroselinum crispum CPRF1 gene Proteins 0.000 description 1
- 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 1
- ABLZXFCXXLZCGV-UHFFFAOYSA-N Phosphorous acid Chemical group OP(O)=O ABLZXFCXXLZCGV-UHFFFAOYSA-N 0.000 description 1
- 101710176177 Protein A56 Proteins 0.000 description 1
- 108700040121 Protein Methyltransferases Proteins 0.000 description 1
- 102000055027 Protein Methyltransferases Human genes 0.000 description 1
- 102000044126 RNA-Binding Proteins Human genes 0.000 description 1
- 101710105008 RNA-binding protein Proteins 0.000 description 1
- 101150065817 ROM2 gene Proteins 0.000 description 1
- 108020004511 Recombinant DNA Proteins 0.000 description 1
- 108091028664 Ribonucleotide Proteins 0.000 description 1
- 101001025539 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) Homothallic switching endonuclease Proteins 0.000 description 1
- 108700025695 Suppressor Genes Proteins 0.000 description 1
- 210000001744 T-lymphocyte Anatomy 0.000 description 1
- 101001081186 Tetrahymena pyriformis High mobility group protein Proteins 0.000 description 1
- AYFVYJQAPQTCCC-UHFFFAOYSA-N Threonine Natural products CC(O)C(N)C(O)=O AYFVYJQAPQTCCC-UHFFFAOYSA-N 0.000 description 1
- 239000004473 Threonine Substances 0.000 description 1
- PTFCDOFLOPIGGS-UHFFFAOYSA-N Zinc dication Chemical compound [Zn+2] PTFCDOFLOPIGGS-UHFFFAOYSA-N 0.000 description 1
- 230000002159 abnormal effect Effects 0.000 description 1
- 150000007513 acids Chemical class 0.000 description 1
- 239000000654 additive Substances 0.000 description 1
- 230000000996 additive effect Effects 0.000 description 1
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 description 1
- 229960005305 adenosine Drugs 0.000 description 1
- 239000000956 alloy Substances 0.000 description 1
- 229910045601 alloy Inorganic materials 0.000 description 1
- 238000004458 analytical method Methods 0.000 description 1
- 238000003149 assay kit Methods 0.000 description 1
- 239000011230 binding agent Substances 0.000 description 1
- 230000033228 biological regulation Effects 0.000 description 1
- 238000007413 biotinylation Methods 0.000 description 1
- 230000006287 biotinylation Effects 0.000 description 1
- 239000001506 calcium phosphate Substances 0.000 description 1
- 229910000389 calcium phosphate Inorganic materials 0.000 description 1
- 235000011010 calcium phosphates Nutrition 0.000 description 1
- 201000011510 cancer Diseases 0.000 description 1
- 150000001720 carbohydrates Chemical class 0.000 description 1
- 235000014633 carbohydrates Nutrition 0.000 description 1
- 238000004113 cell culture Methods 0.000 description 1
- 239000006143 cell culture medium Substances 0.000 description 1
- 238000012512 characterization method Methods 0.000 description 1
- 230000007073 chemical hydrolysis Effects 0.000 description 1
- 108091006090 chromatin-associated proteins Proteins 0.000 description 1
- 239000013611 chromosomal DNA Substances 0.000 description 1
- 238000003776 cleavage reaction Methods 0.000 description 1
- 238000000749 co-immunoprecipitation Methods 0.000 description 1
- 230000001276 controlling effect Effects 0.000 description 1
- 238000010168 coupling process Methods 0.000 description 1
- 125000004122 cyclic group Chemical group 0.000 description 1
- 230000024835 cytogamy Effects 0.000 description 1
- 239000005547 deoxyribonucleotide Substances 0.000 description 1
- 125000002637 deoxyribonucleotide group Chemical group 0.000 description 1
- 230000004069 differentiation Effects 0.000 description 1
- 239000000539 dimer Substances 0.000 description 1
- 230000008034 disappearance Effects 0.000 description 1
- 238000010494 dissociation reaction Methods 0.000 description 1
- 230000005593 dissociations Effects 0.000 description 1
- 238000009826 distribution Methods 0.000 description 1
- 239000003937 drug carrier Substances 0.000 description 1
- 238000004043 dyeing Methods 0.000 description 1
- 230000004064 dysfunction Effects 0.000 description 1
- 238000001962 electrophoresis Methods 0.000 description 1
- 238000004520 electroporation Methods 0.000 description 1
- 238000004836 empirical method Methods 0.000 description 1
- 108010050663 endodeoxyribonuclease CreI Proteins 0.000 description 1
- 238000012407 engineering method Methods 0.000 description 1
- 230000007613 environmental effect Effects 0.000 description 1
- 102000013165 exonuclease Human genes 0.000 description 1
- 230000008175 fetal development Effects 0.000 description 1
- 230000002538 fungal effect Effects 0.000 description 1
- 238000001502 gel electrophoresis Methods 0.000 description 1
- 238000001415 gene therapy Methods 0.000 description 1
- 208000016361 genetic disease Diseases 0.000 description 1
- 238000010362 genome editing Methods 0.000 description 1
- 150000004676 glycans Chemical class 0.000 description 1
- 230000013595 glycosylation Effects 0.000 description 1
- 238000006206 glycosylation reaction Methods 0.000 description 1
- 125000003630 glycyl group Chemical group [H]N([H])C([H])([H])C(*)=O 0.000 description 1
- 230000012010 growth Effects 0.000 description 1
- 230000012447 hatching Effects 0.000 description 1
- 239000000185 hemagglutinin Substances 0.000 description 1
- 239000000710 homodimer Substances 0.000 description 1
- 210000005260 human cell Anatomy 0.000 description 1
- 238000006460 hydrolysis reaction Methods 0.000 description 1
- 238000001114 immunoprecipitation Methods 0.000 description 1
- 230000008676 import Effects 0.000 description 1
- 238000002347 injection Methods 0.000 description 1
- 239000007924 injection Substances 0.000 description 1
- 239000012212 insulator Substances 0.000 description 1
- 230000010354 integration Effects 0.000 description 1
- 238000002955 isolation Methods 0.000 description 1
- 239000002502 liposome Substances 0.000 description 1
- 102100034703 mRNA decay activator protein ZFP36L2 Human genes 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
- 239000011159 matrix material Substances 0.000 description 1
- 239000002609 medium Substances 0.000 description 1
- 239000012528 membrane Substances 0.000 description 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 description 1
- 230000001035 methylating effect Effects 0.000 description 1
- 210000003205 muscle Anatomy 0.000 description 1
- 210000000663 muscle cell Anatomy 0.000 description 1
- 230000007498 myristoylation Effects 0.000 description 1
- 238000007899 nucleic acid hybridization Methods 0.000 description 1
- 210000003463 organelle Anatomy 0.000 description 1
- 210000002220 organoid Anatomy 0.000 description 1
- 239000005022 packaging material Substances 0.000 description 1
- 238000012856 packing Methods 0.000 description 1
- 210000000496 pancreas Anatomy 0.000 description 1
- 239000002245 particle Substances 0.000 description 1
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Natural products OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 description 1
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 description 1
- 230000026731 phosphorylation Effects 0.000 description 1
- 238000006366 phosphorylation reaction Methods 0.000 description 1
- 229920001282 polysaccharide Polymers 0.000 description 1
- 239000005017 polysaccharide Substances 0.000 description 1
- 230000029279 positive regulation of transcription, DNA-dependent Effects 0.000 description 1
- 238000001556 precipitation Methods 0.000 description 1
- 230000008569 process Effects 0.000 description 1
- 230000001737 promoting effect Effects 0.000 description 1
- 230000004952 protein activity Effects 0.000 description 1
- 108020001580 protein domains Proteins 0.000 description 1
- 230000004853 protein function Effects 0.000 description 1
- 230000006916 protein interaction Effects 0.000 description 1
- 230000009145 protein modification Effects 0.000 description 1
- 238000007634 remodeling Methods 0.000 description 1
- 230000010076 replication Effects 0.000 description 1
- 239000002336 ribonucleotide Substances 0.000 description 1
- 125000002652 ribonucleotide group Chemical group 0.000 description 1
- 108091092562 ribozyme Proteins 0.000 description 1
- 229920006395 saturated elastomer Polymers 0.000 description 1
- 230000007017 scission Effects 0.000 description 1
- 230000028327 secretion Effects 0.000 description 1
- 238000010187 selection method Methods 0.000 description 1
- 238000012163 sequencing technique Methods 0.000 description 1
- 230000035939 shock Effects 0.000 description 1
- 238000002741 site-directed mutagenesis Methods 0.000 description 1
- 150000003384 small molecules Chemical class 0.000 description 1
- 241000894007 species Species 0.000 description 1
- 150000003431 steroids Chemical class 0.000 description 1
- 238000003860 storage Methods 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical group [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 description 1
- 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 1
- 238000003160 two-hybrid assay Methods 0.000 description 1
- 238000010396 two-hybrid screening Methods 0.000 description 1
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 description 1
- 238000010798 ubiquitination Methods 0.000 description 1
- 230000034512 ubiquitination Effects 0.000 description 1
- 238000011144 upstream manufacturing Methods 0.000 description 1
- 235000013311 vegetables Nutrition 0.000 description 1
- 230000003612 virological effect Effects 0.000 description 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
-
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/52—Genes encoding for enzymes or proenzymes
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Communicable Diseases (AREA)
- Oncology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Virology (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Contacts (AREA)
- Solid-Sorbent Or Filter-Aiding Compositions (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US34372910P | 2010-05-03 | 2010-05-03 | |
| US61/343,729 | 2010-05-03 | ||
| PCT/US2011/000758 WO2011139349A1 (en) | 2010-05-03 | 2011-05-02 | Compositions for linking zinc finger modules |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN102959087A CN102959087A (zh) | 2013-03-06 |
| CN102959087B true CN102959087B (zh) | 2015-04-29 |
Family
ID=44903931
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201180022329.2A Active CN102959087B (zh) | 2010-05-03 | 2011-05-02 | 用于连接锌指模块的组合物 |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US8772453B2 (enExample) |
| EP (2) | EP2566972B1 (enExample) |
| JP (1) | JP5898179B2 (enExample) |
| KR (2) | KR101974036B1 (enExample) |
| CN (1) | CN102959087B (enExample) |
| AU (1) | AU2011249019B2 (enExample) |
| CA (1) | CA2797189C (enExample) |
| CY (1) | CY1122636T1 (enExample) |
| DK (1) | DK2566972T3 (enExample) |
| ES (1) | ES2773052T3 (enExample) |
| HR (1) | HRP20200254T1 (enExample) |
| HU (1) | HUE048072T2 (enExample) |
| IL (1) | IL222632B (enExample) |
| LT (1) | LT2566972T (enExample) |
| PL (1) | PL2566972T3 (enExample) |
| PT (1) | PT2566972T (enExample) |
| SI (1) | SI2566972T1 (enExample) |
| WO (1) | WO2011139349A1 (enExample) |
Families Citing this family (76)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20120196370A1 (en) | 2010-12-03 | 2012-08-02 | Fyodor Urnov | Methods and compositions for targeted genomic deletion |
| CA2805442C (en) | 2010-07-21 | 2020-05-12 | Sangamo Biosciences, Inc. | Methods and compositions for modification of an hla locus |
| WO2012094132A1 (en) | 2011-01-05 | 2012-07-12 | Sangamo Biosciences, Inc. | Methods and compositions for gene correction |
| WO2013016446A2 (en) | 2011-07-25 | 2013-01-31 | Sangamo Biosciences, Inc. | Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (cftr) gene |
| CA2852955C (en) | 2011-10-27 | 2021-02-16 | Sangamo Biosciences, Inc. | Methods and compositions for modification of the hprt locus |
| BR112014021104B1 (pt) | 2012-02-29 | 2023-03-28 | Sangamo Biosciences, Inc | Proteína de fusão de ocorrência não natural compreendendo um domínio de ligação de dna de dedo de zinco manipulado que se liga a um gene htt, seu uso, método in vitro de modificação da expressão de um gene htt em uma célula, e método de geração de um sistema modelo para o estudo da doença de huntington |
| WO2014011901A2 (en) | 2012-07-11 | 2014-01-16 | Sangamo Biosciences, Inc. | Methods and compositions for delivery of biologics |
| US10648001B2 (en) | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| AU2013308770B2 (en) | 2012-08-29 | 2019-01-17 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| WO2014071070A1 (en) * | 2012-11-01 | 2014-05-08 | Pacific Biosciences Of California, Inc. | Compositions and methods for selection of nucleic acids |
| US9290800B2 (en) | 2013-03-15 | 2016-03-22 | Pacific Biosciences Of California, Inc. | Targeted rolling circle amplification |
| WO2014165612A2 (en) | 2013-04-05 | 2014-10-09 | Dow Agrosciences Llc | Methods and compositions for integration of an exogenous sequence within the genome of plants |
| WO2015031619A1 (en) | 2013-08-28 | 2015-03-05 | Sangamo Biosciences, Inc. | Compositions for linking dna-binding domains and cleavage domains |
| US9957526B2 (en) | 2013-10-17 | 2018-05-01 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| WO2015057976A1 (en) | 2013-10-17 | 2015-04-23 | Sangamo Biosciences, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells |
| CN105934524A (zh) | 2013-11-11 | 2016-09-07 | 桑格摩生物科学股份有限公司 | 用于治疗亨廷顿氏病的方法和组合物 |
| DK3492593T3 (da) | 2013-11-13 | 2021-11-08 | Childrens Medical Center | Nukleasemedieret regulering af genekspression |
| US10072066B2 (en) | 2014-02-03 | 2018-09-11 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a beta thalessemia |
| AU2015231353B2 (en) | 2014-03-18 | 2020-11-05 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of zinc finger protein expression |
| US10435685B2 (en) | 2014-08-19 | 2019-10-08 | Pacific Biosciences Of California, Inc. | Compositions and methods for enrichment of nucleic acids |
| KR20170141217A (ko) | 2015-05-12 | 2017-12-22 | 상가모 테라퓨틱스, 인코포레이티드 | 유전자 발현의 뉴클레아제-매개된 조절 |
| US9957501B2 (en) | 2015-06-18 | 2018-05-01 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
| WO2017011519A1 (en) | 2015-07-13 | 2017-01-19 | Sangamo Biosciences, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| ES2983043T3 (es) | 2015-12-18 | 2024-10-21 | Sangamo Therapeutics Inc | Alteración dirigida del receptor de células T |
| CN108699132B (zh) | 2015-12-18 | 2023-08-11 | 桑格摩生物治疗股份有限公司 | Mhc细胞受体的靶向破坏 |
| EP3402533B1 (en) | 2016-01-15 | 2021-08-04 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of neurologic disease |
| US10724020B2 (en) | 2016-02-02 | 2020-07-28 | Sangamo Therapeutics, Inc. | Compositions for linking DNA-binding domains and cleavage domains |
| SG11201901531TA (en) | 2016-08-24 | 2019-03-28 | Sangamo Therapeutics Inc | Regulation of gene expression using engineered nucleases |
| US10975393B2 (en) | 2016-08-24 | 2021-04-13 | Sangamo Therapeutics, Inc. | Engineered target specific nucleases |
| AU2017345430B2 (en) | 2016-10-20 | 2024-09-05 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of Fabry disease |
| JP7108608B2 (ja) | 2016-10-31 | 2022-07-28 | サンガモ セラピューティクス, インコーポレイテッド | 造血幹細胞および前駆細胞におけるscid関連遺伝子の遺伝子修正 |
| MX2019006426A (es) | 2016-12-01 | 2019-08-14 | Sangamo Therapeutics Inc | Reguladores de tau, y composiciones y metodos para su administracion. |
| MA71412A (fr) | 2017-04-03 | 2025-04-30 | Encoded Therapeutics, Inc. | Expression de transgène sélective d'un tissu |
| US11820728B2 (en) | 2017-04-28 | 2023-11-21 | Acuitas Therapeutics, Inc. | Carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| CN110869497A (zh) | 2017-05-03 | 2020-03-06 | 桑格摩生物治疗股份有限公司 | 修饰囊性纤维化跨膜传导调节蛋白(cftr)基因的方法和组合物 |
| US11512287B2 (en) | 2017-06-16 | 2022-11-29 | Sangamo Therapeutics, Inc. | Targeted disruption of T cell and/or HLA receptors |
| CN111565730B (zh) | 2017-11-09 | 2024-09-17 | 桑格摩生物治疗股份有限公司 | 细胞因子诱导型含sh2蛋白(cish)基因的遗传修饰 |
| CN111727259B (zh) | 2017-12-01 | 2024-04-19 | 编码治疗公司 | 工程化的dna结合蛋白 |
| JP7275152B2 (ja) | 2018-02-08 | 2023-05-17 | サンガモ セラピューティクス, インコーポレイテッド | 操作された標的特異的なヌクレアーゼ |
| WO2019204457A1 (en) * | 2018-04-18 | 2019-10-24 | Sangamo Therapeutics, Inc. | Zinc finger protein compositions for modulation of huntingtin (htt) |
| US11690921B2 (en) | 2018-05-18 | 2023-07-04 | Sangamo Therapeutics, Inc. | Delivery of target specific nucleases |
| JP7541508B2 (ja) | 2018-08-23 | 2024-08-28 | サンガモ セラピューティクス, インコーポレイテッド | 操作された標的特異的な塩基エディター |
| WO2020061161A1 (en) | 2018-09-18 | 2020-03-26 | Sangamo Therapeutics, Inc. | Programmed cell death 1 (pd1) specific nucleases |
| EP3852911B1 (en) | 2018-09-21 | 2025-01-22 | Acuitas Therapeutics, Inc. | Systems and methods for manufacturing lipid nanoparticles and liposomes |
| CN113226333A (zh) | 2018-10-02 | 2021-08-06 | 桑格摩生物治疗股份有限公司 | 用于调控Tau蛋白的方法和组合物 |
| EP3861130A4 (en) * | 2018-10-02 | 2022-08-03 | Sangamo Therapeutics, Inc. | GENETIC MODULATORS |
| US11453639B2 (en) | 2019-01-11 | 2022-09-27 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| JP2022519410A (ja) | 2019-02-06 | 2022-03-24 | サンガモ セラピューティクス, インコーポレイテッド | ムコ多糖症i型の処置のための方法 |
| CA3132167A1 (en) | 2019-04-02 | 2020-10-08 | Weston P. MILLER IV | Methods for the treatment of beta-thalassemia |
| PE20212332A1 (es) | 2019-04-23 | 2021-12-14 | Sangamo Therapeutics Inc | Moduladores de la expresioon del gen de marco de lectura abierto 72 del cromosoma 9 y usos de los mismos |
| WO2021028359A1 (en) | 2019-08-09 | 2021-02-18 | Sangamo Therapeutics France | Controlled expression of chimeric antigen receptors in t cells |
| CN114728037A (zh) | 2019-10-02 | 2022-07-08 | 桑格摩生物治疗股份有限公司 | 用于朊病毒病治疗的锌指蛋白转录因子 |
| AU2020356962A1 (en) | 2019-10-02 | 2022-04-14 | Sangamo Therapeutics, Inc. | Zinc finger protein transcription factors for repressing alpha-synuclein expression |
| AU2020376048A1 (en) | 2019-11-01 | 2022-06-02 | Sangamo Therapeutics, Inc. | Compositions and methods for genome engineering |
| CN115210251A (zh) | 2020-01-22 | 2022-10-18 | 桑格摩生物治疗股份有限公司 | 用于阻遏tau表达的锌指蛋白转录因子 |
| US20230174612A1 (en) * | 2020-04-09 | 2023-06-08 | The Regents Of The University Of California | Notch receptors with zinc finger-containing transcriptional effector |
| MX2023000614A (es) | 2020-07-16 | 2023-02-13 | Acuitas Therapeutics Inc | Lipidos cationicos para usarse en nanoparticulas lipidicas. |
| US12152251B2 (en) | 2020-08-25 | 2024-11-26 | Kite Pharma, Inc. | T cells with improved functionality |
| EP4217479A1 (en) | 2020-09-25 | 2023-08-02 | Sangamo Therapeutics, Inc. | Zinc finger fusion proteins for nucleobase editing |
| EP4221837A1 (en) | 2020-10-02 | 2023-08-09 | Sangamo Therapeutics, Inc. | Novel zinc finger protein transcription factors for repressing alpha-synuclein expression |
| WO2022140577A2 (en) | 2020-12-22 | 2022-06-30 | Chroma Medicine, Inc. | Compositions and methods for epigenetic editing |
| WO2023081756A1 (en) | 2021-11-03 | 2023-05-11 | The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone | Precise genome editing using retrons |
| IL313486A (en) | 2021-12-16 | 2024-08-01 | Acuitas Therapeutics Inc | Lipids for use in lipid nanoparticle formulations |
| IL313758A (en) | 2021-12-22 | 2024-08-01 | Sangamo Therapeutics Inc | Novel zinc finger fusion proteins for nuclear base editing |
| WO2023141602A2 (en) | 2022-01-21 | 2023-07-27 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
| JP2025516294A (ja) | 2022-05-01 | 2025-05-27 | エヌクロマ・バイオ,インコーポレーテッド | Pcsk9発現のエピジェネティック調節のための組成物及び方法 |
| CA3260474A1 (en) | 2022-06-23 | 2023-12-28 | Chroma Medicine Inc | Compositions and methods for epigenetic regulation of b2m expression |
| WO2023250490A1 (en) | 2022-06-23 | 2023-12-28 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of trac expression |
| JP2025525389A (ja) | 2022-06-23 | 2025-08-05 | エヌクロマ・バイオ,インコーポレーテッド | Ciita発現のエピジェネティック調節のための組成物及び方法 |
| WO2024044723A1 (en) | 2022-08-25 | 2024-02-29 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
| AU2023347320A1 (en) | 2022-09-23 | 2025-04-10 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| WO2024081879A1 (en) | 2022-10-14 | 2024-04-18 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of cd247 expression |
| AU2024270764A1 (en) | 2023-05-15 | 2025-12-04 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| WO2024238700A1 (en) | 2023-05-15 | 2024-11-21 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| WO2025049959A2 (en) | 2023-09-01 | 2025-03-06 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6479626B1 (en) * | 1998-03-02 | 2002-11-12 | Massachusetts Institute Of Technology | Poly zinc finger proteins with improved linkers |
Family Cites Families (58)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4512110A (en) | 1981-12-10 | 1985-04-23 | Timesavers, Inc. | Endless abrasive belts |
| US5422251A (en) | 1986-11-26 | 1995-06-06 | Princeton University | Triple-stranded nucleic acids |
| US5176996A (en) | 1988-12-20 | 1993-01-05 | Baylor College Of Medicine | Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use |
| US5420032A (en) | 1991-12-23 | 1995-05-30 | Universitge Laval | Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence |
| US5356802A (en) | 1992-04-03 | 1994-10-18 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease |
| US5436150A (en) | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease |
| US5487994A (en) | 1992-04-03 | 1996-01-30 | The Johns Hopkins University | Insertion and deletion mutants of FokI restriction endonuclease |
| US5792632A (en) | 1992-05-05 | 1998-08-11 | Institut Pasteur | Nucleotide sequence encoding the enzyme I-SceI and the uses thereof |
| US6242568B1 (en) | 1994-01-18 | 2001-06-05 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| US6140466A (en) | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| AU704601B2 (en) | 1994-01-18 | 1999-04-29 | Scripps Research Institute, The | Zinc finger protein derivatives and methods therefor |
| US5585245A (en) | 1994-04-22 | 1996-12-17 | California Institute Of Technology | Ubiquitin-based split protein sensor |
| GB9824544D0 (en) | 1998-11-09 | 1999-01-06 | Medical Res Council | Screening system |
| US7285416B2 (en) * | 2000-01-24 | 2007-10-23 | Gendaq Limited | Regulated gene expression in plants |
| EP0781331B1 (en) | 1994-08-20 | 2008-09-03 | Gendaq Limited | Improvements in or relating to binding proteins for recognition of dna |
| US5789538A (en) | 1995-02-03 | 1998-08-04 | Massachusetts Institute Of Technology | Zinc finger proteins with high affinity new DNA binding specificities |
| US5925523A (en) | 1996-08-23 | 1999-07-20 | President & Fellows Of Harvard College | Intraction trap assay, reagents and uses thereof |
| GB2338237B (en) | 1997-02-18 | 2001-02-28 | Actinova Ltd | In vitro peptide or protein expression library |
| GB9703369D0 (en) | 1997-02-18 | 1997-04-09 | Lindqvist Bjorn H | Process |
| US6342345B1 (en) | 1997-04-02 | 2002-01-29 | The Board Of Trustees Of The Leland Stanford Junior University | Detection of molecular interactions by reporter subunit complementation |
| GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| US6410248B1 (en) | 1998-01-30 | 2002-06-25 | Massachusetts Institute Of Technology | General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites |
| US6140081A (en) | 1998-10-16 | 2000-10-31 | The Scripps Research Institute | Zinc finger binding domains for GNN |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
| US7151201B2 (en) | 2000-01-21 | 2006-12-19 | The Scripps Research Institute | Methods and compositions to modulate expression in plants |
| ATE355368T1 (de) * | 2000-01-24 | 2006-03-15 | Gendaq Ltd | Nucleinsäure bindende polypeptide gekennzeichnet durch flexible linker verbundene nucleinsäuredomäne |
| JP5047437B2 (ja) | 2000-02-08 | 2012-10-10 | サンガモ バイオサイエンシーズ, インコーポレイテッド | 薬物の発見のための細胞 |
| US20020061512A1 (en) | 2000-02-18 | 2002-05-23 | Kim Jin-Soo | Zinc finger domains and methods of identifying same |
| WO2001088197A2 (en) | 2000-05-16 | 2001-11-22 | Massachusetts Institute Of Technology | Methods and compositions for interaction trap assays |
| US20060268407A1 (en) | 2000-07-07 | 2006-11-30 | Fergason James L | Display system using two displays and polarization direction rotation for showing high-resolution and three-dimensional images and method and use of a DBEF beam splitter |
| JP2002060786A (ja) | 2000-08-23 | 2002-02-26 | Kao Corp | 硬質表面用殺菌防汚剤 |
| US7067317B2 (en) | 2000-12-07 | 2006-06-27 | Sangamo Biosciences, Inc. | Regulation of angiogenesis with zinc finger proteins |
| US20040197892A1 (en) * | 2001-04-04 | 2004-10-07 | Michael Moore | Composition binding polypeptides |
| GB0108491D0 (en) | 2001-04-04 | 2001-05-23 | Gendaq Ltd | Engineering zinc fingers |
| JP2005500061A (ja) | 2001-08-20 | 2005-01-06 | ザ スクリップス リサーチ インスティテュート | Cnnについての亜鉛フィンガー結合ドメイン |
| US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
| WO2003104414A2 (en) * | 2002-06-11 | 2003-12-18 | The Scripps Research Institute | Artificial transcription factors |
| US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
| US7407776B2 (en) * | 2003-09-19 | 2008-08-05 | Sangamo Biosciences, Inc. | Engineered zinc finger proteins for regulation of gene expression |
| WO2005061705A1 (en) | 2003-12-23 | 2005-07-07 | Toolgen, Inc. | Regulation of prokaryotic gene expression with zinc finger proteins |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| CA2562193A1 (en) | 2004-04-08 | 2005-10-27 | Sangamo Biosciences, Inc. | Treatment of neuropathic pain with zinc finger proteins |
| AU2005287278B2 (en) | 2004-09-16 | 2011-08-04 | Sangamo Biosciences, Inc. | Compositions and methods for protein production |
| EP1877583A2 (en) | 2005-05-05 | 2008-01-16 | Arizona Board of Regents on behalf of the Unversity of Arizona | Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences |
| WO2007014275A2 (en) | 2005-07-26 | 2007-02-01 | Sangamo Biosciences, Inc. | Targeted integration and expression of exogenous nucleic acid sequences |
| ES2626025T3 (es) | 2005-10-18 | 2017-07-21 | Precision Biosciences | Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas |
| WO2007136685A2 (en) | 2006-05-19 | 2007-11-29 | Sangamo Biosciences, Inc. | Methods and compositions for inactivation of dihydrofolate reductase |
| EP2213731B1 (en) | 2006-05-25 | 2013-12-04 | Sangamo BioSciences, Inc. | Variant foki cleavage half-domains |
| EP2447279B1 (en) | 2006-05-25 | 2014-04-09 | Sangamo BioSciences, Inc. | Methods and compositions for gene inactivation |
| CA2667414C (en) | 2006-11-13 | 2015-12-29 | Sangamo Biosciences, Inc. | Methods and compositions for modification of the human glucocorticoid receptor locus |
| US8110379B2 (en) | 2007-04-26 | 2012-02-07 | Sangamo Biosciences, Inc. | Targeted integration into the PPP1R12C locus |
| EP2188384B1 (en) | 2007-09-27 | 2015-07-15 | Sangamo BioSciences, Inc. | Rapid in vivo identification of biologically active nucleases |
| JP2013500018A (ja) * | 2009-07-24 | 2013-01-07 | シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニー | ゲノム編集のための方法 |
-
2011
- 2011-05-02 PL PL11777690T patent/PL2566972T3/pl unknown
- 2011-05-02 EP EP11777690.6A patent/EP2566972B1/en active Active
- 2011-05-02 AU AU2011249019A patent/AU2011249019B2/en active Active
- 2011-05-02 US US13/068,102 patent/US8772453B2/en active Active
- 2011-05-02 KR KR1020187017770A patent/KR101974036B1/ko active Active
- 2011-05-02 CN CN201180022329.2A patent/CN102959087B/zh active Active
- 2011-05-02 WO PCT/US2011/000758 patent/WO2011139349A1/en not_active Ceased
- 2011-05-02 CA CA2797189A patent/CA2797189C/en active Active
- 2011-05-02 HR HRP20200254TT patent/HRP20200254T1/hr unknown
- 2011-05-02 KR KR1020127028770A patent/KR20130073887A/ko not_active Ceased
- 2011-05-02 EP EP19211724.0A patent/EP3636766A1/en active Pending
- 2011-05-02 HU HUE11777690A patent/HUE048072T2/hu unknown
- 2011-05-02 JP JP2013509041A patent/JP5898179B2/ja not_active Expired - Fee Related
- 2011-05-02 SI SI201131844T patent/SI2566972T1/sl unknown
- 2011-05-02 DK DK11777690.6T patent/DK2566972T3/da active
- 2011-05-02 PT PT117776906T patent/PT2566972T/pt unknown
- 2011-05-02 ES ES11777690T patent/ES2773052T3/es active Active
- 2011-05-02 LT LTEP11777690.6T patent/LT2566972T/lt unknown
-
2012
- 2012-10-23 IL IL222632A patent/IL222632B/en active IP Right Grant
-
2014
- 2014-06-04 US US14/296,159 patent/US9163245B2/en active Active
-
2020
- 2020-02-14 CY CY20201100140T patent/CY1122636T1/el unknown
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6479626B1 (en) * | 1998-03-02 | 2002-11-12 | Massachusetts Institute Of Technology | Poly zinc finger proteins with improved linkers |
Non-Patent Citations (1)
| Title |
|---|
| Structure-based design of transcription factors;Pomerantz JL等;《Science》;19950131;第267卷(第5194期);摘要、图1-5 * |
Also Published As
| Publication number | Publication date |
|---|---|
| JP2013527765A (ja) | 2013-07-04 |
| AU2011249019B2 (en) | 2015-01-22 |
| CY1122636T1 (el) | 2021-03-12 |
| US20110287512A1 (en) | 2011-11-24 |
| CA2797189C (en) | 2020-05-26 |
| SI2566972T1 (sl) | 2020-04-30 |
| JP5898179B2 (ja) | 2016-04-06 |
| EP3636766A1 (en) | 2020-04-15 |
| KR20180077284A (ko) | 2018-07-06 |
| US8772453B2 (en) | 2014-07-08 |
| IL222632A0 (en) | 2012-12-31 |
| CA2797189A1 (en) | 2011-11-10 |
| DK2566972T3 (da) | 2020-02-17 |
| HUE048072T2 (hu) | 2020-05-28 |
| IL222632B (en) | 2018-12-31 |
| PL2566972T3 (pl) | 2020-06-29 |
| EP2566972A4 (en) | 2014-01-01 |
| US9163245B2 (en) | 2015-10-20 |
| KR101974036B1 (ko) | 2019-04-30 |
| ES2773052T3 (es) | 2020-07-09 |
| US20140287500A1 (en) | 2014-09-25 |
| PT2566972T (pt) | 2020-03-02 |
| LT2566972T (lt) | 2020-03-10 |
| WO2011139349A1 (en) | 2011-11-10 |
| CN102959087A (zh) | 2013-03-06 |
| HRP20200254T1 (hr) | 2020-05-29 |
| KR20130073887A (ko) | 2013-07-03 |
| EP2566972A1 (en) | 2013-03-13 |
| EP2566972B1 (en) | 2020-01-15 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CN102959087B (zh) | 用于连接锌指模块的组合物 | |
| AU2011249019A1 (en) | Compositions for linking zinc finger modules | |
| US9522936B2 (en) | Engineered transcription activator like effector (TALE) proteins | |
| US11661612B2 (en) | DNA-binding proteins and uses thereof | |
| US10646543B2 (en) | Methods and compositions for treating trinucleotide repeat disorders | |
| AU2012340213B2 (en) | Modified DNA-binding proteins and uses thereof | |
| KR102084539B1 (ko) | 헌팅턴병을 치료하기 위한 방법 및 조성물 | |
| EP2877488B1 (en) | New modular base-specific nucleic acid binding domains from burkholderia rhizoxinica proteins | |
| KR20190039955A (ko) | 가공된 표적 특이적 뉴클레아제 | |
| HK40026462A (en) | Compositions for linking zinc finger modules | |
| HK1182417B (en) | Compositions for linking zinc finger modules | |
| HK1182417A (en) | Compositions for linking zinc finger modules |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| C06 | Publication | ||
| PB01 | Publication | ||
| C10 | Entry into substantive examination | ||
| SE01 | Entry into force of request for substantive examination | ||
| C14 | Grant of patent or utility model | ||
| GR01 | Patent grant |