JP2013544077A5 - - Google Patents
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- JP2013544077A5 JP2013544077A5 JP2013533982A JP2013533982A JP2013544077A5 JP 2013544077 A5 JP2013544077 A5 JP 2013544077A5 JP 2013533982 A JP2013533982 A JP 2013533982A JP 2013533982 A JP2013533982 A JP 2013533982A JP 2013544077 A5 JP2013544077 A5 JP 2013544077A5
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- 210000003754 fetus Anatomy 0.000 claims description 2
- 210000001161 mammalian embryo Anatomy 0.000 claims description 2
- 210000004027 cell Anatomy 0.000 claims 12
- 239000008194 pharmaceutical composition Substances 0.000 claims 11
- 108090000623 proteins and genes Proteins 0.000 claims 10
- 102000004169 proteins and genes Human genes 0.000 claims 7
- 230000004568 DNA-binding Effects 0.000 claims 4
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims 4
- 108091033319 polynucleotide Proteins 0.000 claims 4
- 239000002157 polynucleotide Substances 0.000 claims 4
- 102000040430 polynucleotide Human genes 0.000 claims 4
- 102100022641 Coagulation factor IX Human genes 0.000 claims 3
- 108010076282 Factor IX Proteins 0.000 claims 3
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 claims 3
- 239000003795 chemical substances by application Substances 0.000 claims 3
- 210000003494 hepatocyte Anatomy 0.000 claims 3
- 239000011701 zinc Substances 0.000 claims 3
- 229910052725 zinc Inorganic materials 0.000 claims 3
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 238000003776 cleavage reaction Methods 0.000 claims 2
- 229960004222 factor ix Drugs 0.000 claims 2
- 229930182830 galactose Natural products 0.000 claims 2
- 238000001802 infusion Methods 0.000 claims 2
- 210000004185 liver Anatomy 0.000 claims 2
- 230000007017 scission Effects 0.000 claims 2
- VSNHCAURESNICA-NJFSPNSNSA-N 1-oxidanylurea Chemical compound N[14C](=O)NO VSNHCAURESNICA-NJFSPNSNSA-N 0.000 claims 1
- 101710132601 Capsid protein Proteins 0.000 claims 1
- 101710094648 Coat protein Proteins 0.000 claims 1
- 102100021181 Golgi phosphoprotein 3 Human genes 0.000 claims 1
- 101710125418 Major capsid protein Proteins 0.000 claims 1
- 241001465754 Metazoa Species 0.000 claims 1
- 101710141454 Nucleoprotein Proteins 0.000 claims 1
- 241000283973 Oryctolagus cuniculus Species 0.000 claims 1
- 101710083689 Probable capsid protein Proteins 0.000 claims 1
- 241000283984 Rodentia Species 0.000 claims 1
- 241001493546 Suina Species 0.000 claims 1
- YZCKVEUIGOORGS-NJFSPNSNSA-N Tritium Chemical compound [3H] YZCKVEUIGOORGS-NJFSPNSNSA-N 0.000 claims 1
- 101710185494 Zinc finger protein Proteins 0.000 claims 1
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims 1
- NOFOAYPPHIUXJR-APNQCZIXSA-N aphidicolin Chemical compound C1[C@@]23[C@@]4(C)CC[C@@H](O)[C@@](C)(CO)[C@@H]4CC[C@H]3C[C@H]1[C@](CO)(O)CC2 NOFOAYPPHIUXJR-APNQCZIXSA-N 0.000 claims 1
- SEKZNWAQALMJNH-YZUCACDQSA-N aphidicolin Natural products C[C@]1(CO)CC[C@]23C[C@H]1C[C@@H]2CC[C@H]4[C@](C)(CO)[C@H](O)CC[C@]34C SEKZNWAQALMJNH-YZUCACDQSA-N 0.000 claims 1
- 210000000013 bile duct Anatomy 0.000 claims 1
- DQLATGHUWYMOKM-UHFFFAOYSA-L cisplatin Chemical compound N[Pt](N)(Cl)Cl DQLATGHUWYMOKM-UHFFFAOYSA-L 0.000 claims 1
- 229960004316 cisplatin Drugs 0.000 claims 1
- 210000001671 embryonic stem cell Anatomy 0.000 claims 1
- VJJPUSNTGOMMGY-MRVIYFEKSA-N etoposide Chemical compound COC1=C(O)C(OC)=CC([C@@H]2C3=CC=4OCOC=4C=C3[C@@H](O[C@H]3[C@@H]([C@@H](O)[C@@H]4O[C@H](C)OC[C@H]4O3)O)[C@@H]3[C@@H]2C(OC3)=O)=C1 VJJPUSNTGOMMGY-MRVIYFEKSA-N 0.000 claims 1
- 229960005420 etoposide Drugs 0.000 claims 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 1
- 208000009429 hemophilia B Diseases 0.000 claims 1
- 210000002767 hepatic artery Anatomy 0.000 claims 1
- 210000003897 hepatic stem cell Anatomy 0.000 claims 1
- 210000005260 human cell Anatomy 0.000 claims 1
- 210000004263 induced pluripotent stem cell Anatomy 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 238000007912 intraperitoneal administration Methods 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 238000012753 partial hepatectomy Methods 0.000 claims 1
- OIGNJSKKLXVSLS-VWUMJDOOSA-N prednisolone Chemical compound O=C1C=C[C@]2(C)[C@H]3[C@@H](O)C[C@](C)([C@@](CC4)(O)C(=O)CO)[C@@H]4[C@@H]3CCC2=C1 OIGNJSKKLXVSLS-VWUMJDOOSA-N 0.000 claims 1
- 229960005205 prednisolone Drugs 0.000 claims 1
- 210000000130 stem cell Anatomy 0.000 claims 1
- 229910052722 tritium Inorganic materials 0.000 claims 1
- 238000009281 ultraviolet germicidal irradiation Methods 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 150000003751 zinc Chemical class 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US39233310P | 2010-10-12 | 2010-10-12 | |
| US61/392,333 | 2010-10-12 | ||
| PCT/US2011/056020 WO2012051343A1 (en) | 2010-10-12 | 2011-10-12 | Methods and compositions for treating hemophilia b |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013544077A JP2013544077A (ja) | 2013-12-12 |
| JP2013544077A5 true JP2013544077A5 (OSRAM) | 2016-01-07 |
| JP6018069B2 JP6018069B2 (ja) | 2016-11-02 |
Family
ID=45938707
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013533982A Active JP6018069B2 (ja) | 2010-10-12 | 2011-10-12 | 血友病ｂを治療する方法及び組成物 |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US9175280B2 (OSRAM) |
| EP (1) | EP2627665B1 (OSRAM) |
| JP (1) | JP6018069B2 (OSRAM) |
| KR (1) | KR101930711B1 (OSRAM) |
| CN (1) | CN103270046B (OSRAM) |
| AU (1) | AU2011316575B2 (OSRAM) |
| BR (1) | BR112013008881B1 (OSRAM) |
| CA (1) | CA2814143C (OSRAM) |
| ES (1) | ES2562421T3 (OSRAM) |
| RU (1) | RU2608643C2 (OSRAM) |
| WO (1) | WO2012051343A1 (OSRAM) |
| ZA (1) | ZA201302625B (OSRAM) |
Families Citing this family (63)
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| US20100256062A1 (en) | 2004-12-06 | 2010-10-07 | Howard Tommy E | Allelic Variants of Human Factor VIII |
| US9517257B2 (en) | 2010-08-10 | 2016-12-13 | Ecole Polytechnique Federale De Lausanne (Epfl) | Erythrocyte-binding therapeutics |
| CN108117586A (zh) | 2010-08-10 | 2018-06-05 | 洛桑聚合联合学院 | 红细胞结合性治疗剂 |
| US9850296B2 (en) | 2010-08-10 | 2017-12-26 | Ecole Polytechnique Federale De Lausanne (Epfl) | Erythrocyte-binding therapeutics |
| EP3498833B1 (en) | 2011-09-21 | 2023-08-16 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of transgene expression |
| CN104520428B (zh) * | 2012-02-17 | 2018-09-21 | 费城儿童医院 | 将基因转移到细胞、器官和组织的aav载体组合物和方法 |
| US10648001B2 (en) | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| CA2875618C (en) | 2012-07-11 | 2021-04-27 | Sangamo Biosciences, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
| EP2929017A4 (en) * | 2012-12-05 | 2016-09-28 | Sangamo Biosciences Inc | METHOD AND COMPOSITIONS FOR REGULATING METABOLISM DISEASES |
| US20160045575A1 (en) * | 2012-12-07 | 2016-02-18 | Tom E. HOWARD | FACTOR VIII MUTATION REPAIR AND TOLERANCE INDUCTION AND RELATED cDNAs, COMPOSITIONS, METHODS AND SYSTEMS |
| US10272163B2 (en) | 2012-12-07 | 2019-04-30 | The Regents Of The University Of California | Factor VIII mutation repair and tolerance induction |
| WO2014093701A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
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| EP3011030B1 (en) | 2013-06-17 | 2023-11-08 | The Broad Institute, Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
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| CN110713995B (zh) * | 2013-10-17 | 2023-08-01 | 桑格摩生物科学股份有限公司 | 用于核酸酶介导的基因组工程改造的递送方法和组合物 |
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| WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
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