JP2018531614A5 - - Google Patents
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- JP2018531614A5 JP2018531614A5 JP2018521965A JP2018521965A JP2018531614A5 JP 2018531614 A5 JP2018531614 A5 JP 2018531614A5 JP 2018521965 A JP2018521965 A JP 2018521965A JP 2018521965 A JP2018521965 A JP 2018521965A JP 2018531614 A5 JP2018531614 A5 JP 2018531614A5
- Authority
- JP
- Japan
- Prior art keywords
- polynucleotide expression
- expression construct
- sequence
- promoter
- transgene
- 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.)
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- 108091033319 polynucleotide Proteins 0.000 claims description 43
- 239000002157 polynucleotide Substances 0.000 claims description 43
- 102000040430 polynucleotide Human genes 0.000 claims description 43
- 108700019146 Transgenes Proteins 0.000 claims description 23
- 108010071690 Prealbumin Proteins 0.000 claims description 17
- 102000009190 Transthyretin Human genes 0.000 claims description 17
- 108090000623 proteins and genes Proteins 0.000 claims description 12
- 102000004169 proteins and genes Human genes 0.000 claims description 12
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims description 11
- 239000013607 AAV vector Substances 0.000 claims description 10
- 210000004185 liver Anatomy 0.000 claims description 9
- 101710163270 Nuclease Proteins 0.000 claims description 8
- 101710181917 Serine proteinase inhibitor 1 Proteins 0.000 claims description 8
- 239000003623 enhancer Substances 0.000 claims description 8
- 239000012212 insulator Substances 0.000 claims description 8
- 125000006850 spacer group Chemical group 0.000 claims description 8
- 208000031220 Hemophilia Diseases 0.000 claims description 6
- 208000009292 Hemophilia A Diseases 0.000 claims description 6
- 208000015439 Lysosomal storage disease Diseases 0.000 claims description 6
- 230000002950 deficient Effects 0.000 claims description 5
- 239000008194 pharmaceutical composition Substances 0.000 claims description 4
- 230000008488 polyadenylation Effects 0.000 claims description 4
- 241000702321 Microvirus Species 0.000 claims description 2
- 241001529936 Murinae Species 0.000 claims description 2
- 230000035772 mutation Effects 0.000 claims description 2
- 239000013598 vector Substances 0.000 claims description 2
- 108091033409 CRISPR Proteins 0.000 claims 3
- 238000010354 CRISPR gene editing Methods 0.000 claims 3
- 238000010459 TALEN Methods 0.000 claims 2
- 108010043645 Transcription Activator-Like Effector Nucleases Proteins 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 241000702623 Minute virus of mice Species 0.000 claims 1
- 238000000034 method Methods 0.000 description 7
- 241000124008 Mammalia Species 0.000 description 4
- 108010088751 Albumins Proteins 0.000 description 2
- 230000001225 therapeutic effect Effects 0.000 description 2
- 241001465754 Metazoa Species 0.000 description 1
- 210000003719 b-lymphocyte Anatomy 0.000 description 1
- 210000004027 cell Anatomy 0.000 description 1
- 210000003494 hepatocyte Anatomy 0.000 description 1
- 230000001939 inductive effect Effects 0.000 description 1
- 239000003112 inhibitor Substances 0.000 description 1
- 238000002493 microarray Methods 0.000 description 1
- 150000003431 steroids Chemical class 0.000 description 1
- 230000003612 virological effect Effects 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021110561A JP7324249B2 (ja) | 2015-10-28 | 2021-07-02 | 肝臓特異的コンストラクト、第ｖｉｉｉ因子発現カセット、およびその使用の方法 |
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562247469P | 2015-10-28 | 2015-10-28 | |
| US62/247,469 | 2015-10-28 | ||
| US201662307897P | 2016-03-14 | 2016-03-14 | |
| US62/307,897 | 2016-03-14 | ||
| US201662315438P | 2016-03-30 | 2016-03-30 | |
| US62/315,438 | 2016-03-30 | ||
| US201662326229P | 2016-04-22 | 2016-04-22 | |
| US62/326,229 | 2016-04-22 | ||
| US201662355106P | 2016-06-27 | 2016-06-27 | |
| US62/355,106 | 2016-06-27 | ||
| PCT/US2016/042099 WO2017074526A1 (en) | 2015-10-28 | 2016-07-13 | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021110561A Division JP7324249B2 (ja) | 2015-10-28 | 2021-07-02 | 肝臓特異的コンストラクト、第ｖｉｉｉ因子発現カセット、およびその使用の方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018531614A JP2018531614A (ja) | 2018-11-01 |
| JP2018531614A5 true JP2018531614A5 (enExample) | 2019-08-15 |
| JP6909212B2 JP6909212B2 (ja) | 2021-07-28 |
Family
ID=58631984
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018521965A Active JP6909212B2 (ja) | 2015-10-28 | 2016-07-13 | 肝臓特異的コンストラクト、第ｖｉｉｉ因子発現カセット、およびその使用の方法 |
| JP2021110561A Active JP7324249B2 (ja) | 2015-10-28 | 2021-07-02 | 肝臓特異的コンストラクト、第ｖｉｉｉ因子発現カセット、およびその使用の方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021110561A Active JP7324249B2 (ja) | 2015-10-28 | 2021-07-02 | 肝臓特異的コンストラクト、第ｖｉｉｉ因子発現カセット、およびその使用の方法 |
Country Status (17)
| Country | Link |
|---|---|
| US (4) | US10143760B2 (enExample) |
| EP (1) | EP3368664A4 (enExample) |
| JP (2) | JP6909212B2 (enExample) |
| KR (2) | KR20250022906A (enExample) |
| CN (2) | CN115141836B (enExample) |
| AU (1) | AU2016343887B2 (enExample) |
| CA (1) | CA3002524A1 (enExample) |
| CO (1) | CO2018005215A2 (enExample) |
| IL (1) | IL258826B2 (enExample) |
| MX (1) | MX2018005272A (enExample) |
| MY (1) | MY189674A (enExample) |
| PE (1) | PE20181206A1 (enExample) |
| PH (1) | PH12018500927B1 (enExample) |
| SA (1) | SA518391456B1 (enExample) |
| SG (1) | SG11201803547RA (enExample) |
| WO (1) | WO2017074526A1 (enExample) |
| ZA (1) | ZA201802723B (enExample) |
Families Citing this family (87)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6261500B2 (ja) | 2011-07-22 | 2018-01-17 | プレジデント アンド フェローズ オブ ハーバード カレッジ | ヌクレアーゼ切断特異性の評価および改善 |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9068179B1 (en) | 2013-12-12 | 2015-06-30 | President And Fellows Of Harvard College | Methods for correcting presenilin point mutations |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| AU2016291778B2 (en) | 2015-07-13 | 2021-05-06 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| EP3365356B1 (en) | 2015-10-23 | 2023-06-28 | President and Fellows of Harvard College | Nucleobase editors and uses thereof |
| EP3368664A4 (en) * | 2015-10-28 | 2019-08-28 | Sangamo Therapeutics, Inc. | LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF |
| PE20231949A1 (es) | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| US11584780B2 (en) | 2016-07-26 | 2023-02-21 | Biomarin Pharmaceutical Inc. | Adeno-associated virus capsid proteins |
| JP7231935B2 (ja) | 2016-08-03 | 2023-03-08 | プレジデント アンド フェローズ オブ ハーバード カレッジ | アデノシン核酸塩基編集因子およびそれらの使用 |
| EP3497214B1 (en) | 2016-08-09 | 2023-06-28 | President and Fellows of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| CA3035534A1 (en) | 2016-09-07 | 2018-03-15 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
| EP3526320A1 (en) | 2016-10-14 | 2019-08-21 | President and Fellows of Harvard College | Aav delivery of nucleobase editors |
| EP4667565A2 (en) | 2016-10-20 | 2025-12-24 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of fabry disease |
| IL266862B2 (en) | 2016-12-01 | 2024-01-01 | Sangamo Therapeutics Inc | Tau modulators and methods and preparations for their administration |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| WO2018165631A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Cancer vaccine |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE) |
| MY208862A (en) * | 2017-08-01 | 2025-06-04 | Spark Therapeutics Inc | Factor viii (fviii) gene therapy methods |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| CN111757937A (zh) | 2017-10-16 | 2020-10-09 | 布罗德研究所股份有限公司 | 腺苷碱基编辑器的用途 |
| EP3697907A1 (en) * | 2017-10-17 | 2020-08-26 | CRISPR Therapeutics AG | Compositions and methods for gene editing for hemophilia a |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| EP3737762A1 (en) | 2018-01-12 | 2020-11-18 | CRISPR Therapeutics AG | Compositions and methods for gene editing by targeting transferrin |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| BR112020015798A2 (pt) | 2018-02-01 | 2021-03-09 | Homology Medicines, Inc. | Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas |
| RU2020129448A (ru) | 2018-02-08 | 2022-03-09 | Сангамо Терапьютикс, Инк. | Сконструированные специфические для мишени нуклеазы |
| MA51869A (fr) * | 2018-02-16 | 2020-12-23 | Bayer Healthcare Llc | Compositions et méthodes pour l'édition génique par ciblage du fibrinogène-alpha |
| EP3755795A4 (en) | 2018-02-19 | 2022-07-20 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof |
| WO2019217513A2 (en) | 2018-05-09 | 2019-11-14 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| KR20210008491A (ko) * | 2018-05-14 | 2021-01-22 | 바이오마린 파머수티컬 인크. | 청소년 대상체에서 aav 벡터의 안정한 발현 |
| US11690921B2 (en) | 2018-05-18 | 2023-07-04 | Sangamo Therapeutics, Inc. | Delivery of target specific nucleases |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| CN112639110A (zh) * | 2018-06-22 | 2021-04-09 | 阿斯克肋匹奥生物制药公司 | 用于基因递送以在细胞内持续存在的载体 |
| MX2021001375A (es) * | 2018-08-03 | 2021-04-19 | Sangamo Therapeutics Inc | Parametros clinicos mejorados por la expresion del factor viii. |
| WO2020041773A1 (en) * | 2018-08-24 | 2020-02-27 | Spark Therapeutics, Inc. | Optimized promoter sequences, intron-free expression constructs and methods of use |
| US12329857B2 (en) | 2018-09-21 | 2025-06-17 | Acuitas Therapeutics, Inc. | Systems and methods for manufacturing lipid nanoparticles and liposomes |
| WO2020072451A1 (en) * | 2018-10-01 | 2020-04-09 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| US12318427B2 (en) | 2018-10-02 | 2025-06-03 | Sangamo Therapeutics, Inc. | Methods and compositions for modulation of tau proteins |
| KR20210091167A (ko) | 2018-10-16 | 2021-07-21 | 블루알렐, 엘엘씨 | 유전자에서 dna의 표적화 삽입을 위한 방법 |
| MX2021004277A (es) | 2018-10-18 | 2021-09-08 | Intellia Therapeutics Inc | Composiciones y metodos para expresar el factor ix. |
| WO2020084162A1 (en) * | 2018-10-26 | 2020-04-30 | Vrije Universiteit Brussel | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| AU2020205717B2 (en) | 2019-01-11 | 2025-09-11 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| GB201900741D0 (en) | 2019-01-18 | 2019-03-06 | Synpromics Ltd | Liver-specifc inducible prometers and methods of use thereof |
| WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| AU2019428629A1 (en) | 2019-02-06 | 2021-01-28 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type I |
| MA54951A (fr) | 2019-02-15 | 2021-12-22 | Bayer Healthcare Llc | Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée |
| MA55313A (fr) | 2019-03-13 | 2022-01-19 | Generation Bio Co | Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii |
| MX2021011426A (es) | 2019-03-19 | 2022-03-11 | Broad Inst Inc | Metodos y composiciones para editar secuencias de nucleótidos. |
| AU2020253532B2 (en) * | 2019-04-04 | 2024-06-20 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized coagulation factor 12 locus |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CN110229849B (zh) * | 2019-05-31 | 2021-09-17 | 中国科学院深圳先进技术研究院 | 时空调控型uPA基因表达非病毒载体及其制备方法和应用 |
| US11622547B2 (en) | 2019-06-07 | 2023-04-11 | Regeneran Pharmaceuticals, Inc. | Genetically modified mouse that expresses human albumin |
| CN110684798A (zh) * | 2019-09-03 | 2020-01-14 | 深圳新诺微环生物科技有限公司 | 肌肉靶向的微环dna基因治疗 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| CN113058041B (zh) * | 2020-08-27 | 2022-04-05 | 华东师范大学 | 一种用于治疗庞贝氏病的产品 |
| CN113088519B (zh) * | 2019-12-23 | 2024-02-02 | 苏州华毅乐健生物科技有限公司 | 一种分离的核酸分子及其应用 |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| CA3165015A1 (en) * | 2020-01-16 | 2021-07-22 | Matthias Klugmann | Adeno associated virus based gene therapy for phenylketonuria |
| MX2022014008A (es) | 2020-05-08 | 2023-02-09 | Broad Inst Inc | Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo. |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| AU2021308681A1 (en) | 2020-07-16 | 2023-03-09 | Acuitas Therapeutics, Inc. | Cationic lipids for use in lipid nanoparticles |
| MX2023005218A (es) | 2020-11-03 | 2023-05-16 | Pfizer | Metodos de purificacion de vectores de aav mediante cromatografia de intercambio anionico. |
| KR102536321B1 (ko) | 2020-12-09 | 2023-05-26 | 재단법인 아산사회복지재단 | 인체 유래 유전자 발현 유도 모듈에 기반한 간 특이 유전자 발현 시스템 |
| IL303737A (en) | 2020-12-18 | 2023-08-01 | Sangamo Therapeutics Inc | Improved pharmaceutical compositions containing adeno-associated viral vector |
| JP2024503234A (ja) | 2020-12-23 | 2024-01-25 | ファイザー・インク | 親和性クロマトグラフィーによるａａｖベクターの精製方法 |
| KR20240023638A (ko) | 2021-06-22 | 2024-02-22 | 화이자 인코포레이티드 | 곤충 세포에서의 아데노-연관 바이러스 벡터의 생산 |
| WO2023078220A1 (zh) * | 2021-11-02 | 2023-05-11 | 苏州华毅乐健生物科技有限公司 | 分离的核酸分子及其应用 |
| KR20240123832A (ko) | 2021-12-16 | 2024-08-14 | 아퀴타스 테라퓨틱스 인크. | 지질 나노입자 제형에 사용하기 위한 지질 |
| WO2024003687A1 (en) | 2022-06-28 | 2024-01-04 | Pfizer Inc. | Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy |
| CN115948408A (zh) * | 2022-09-23 | 2023-04-11 | 上海信致医药科技有限公司 | 改进的人凝血因子viii基因表达盒及其应用 |
| WO2025090427A1 (en) | 2023-10-23 | 2025-05-01 | University Of Rochester | Glial-targeted relief of hyperexcitability in neurodegenerative diseases |
| WO2025226943A1 (en) | 2024-04-25 | 2025-10-30 | Masonic Medical Research Laboratory | Polynucleotide for hepatocyte expression of vestigial like protein 4 and method of use thereof |
| WO2025235425A1 (en) * | 2024-05-06 | 2025-11-13 | Research Institute At Nationwide Children's Hospital | Improved proviral plasmids |
| WO2025250457A1 (en) | 2024-05-28 | 2025-12-04 | University Of Rochester | Enhanced brain transduction by gene therapeutics |
Family Cites Families (57)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU5588298A (en) | 1996-12-02 | 1998-06-29 | Cell Genesys, Inc. | Adeno-associated viral vector-mediated delivery of dna to cells of the liver |
| US6221349B1 (en) * | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US6719969B1 (en) * | 1999-08-09 | 2004-04-13 | The Regents Of The University Of Michigan | Treatment of liver disease and injury with CXC chemokines |
| AU776576B2 (en) | 1999-12-06 | 2004-09-16 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
| KR20020086508A (ko) | 2000-02-08 | 2002-11-18 | 상가모 바이오사이언스 인코포레이티드 | 약물 발견용 세포 |
| US7067317B2 (en) | 2000-12-07 | 2006-06-27 | Sangamo Biosciences, Inc. | Regulation of angiogenesis with zinc finger proteins |
| US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
| WO2003087341A2 (en) | 2002-01-23 | 2003-10-23 | The University Of Utah Research Foundation | Targeted chromosomal mutagenesis using zinc finger nucleases |
| EP1504092B2 (en) | 2002-03-21 | 2014-06-25 | Sangamo BioSciences, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
| CA2392863A1 (en) | 2002-07-08 | 2004-01-08 | Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw | A high capacity recombinant adenoviral vector for treatment of hemophilia a |
| US9447434B2 (en) | 2002-09-05 | 2016-09-20 | California Institute Of Technology | Use of chimeric nucleases to stimulate gene targeting |
| US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
| US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| EP1675956B1 (en) * | 2003-10-21 | 2010-10-13 | Merck Serono SA | Minimal dna sequence acting as a chromatin insulator and its use in protein expression |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| WO2005080581A2 (en) * | 2004-02-17 | 2005-09-01 | University Of Florida Research Foundation, Inc. | Insulated herpesvirus-derived gene expression cassettes for sustained and regulatable gene expression |
| CA2579677A1 (en) | 2004-09-16 | 2006-03-30 | Sangamo Biosciences, Inc. | Compositions and methods for protein production |
| CA2611465A1 (en) * | 2005-06-10 | 2007-02-22 | Genentech, Inc. | Improving antibody expression using vectors containing insulator elements |
| EP2027262B1 (en) | 2006-05-25 | 2010-03-31 | Sangamo Biosciences Inc. | Variant foki cleavage half-domains |
| JP5551432B2 (ja) | 2006-05-25 | 2014-07-16 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 遺伝子不活性化のための方法と組成物 |
| EP1985631A1 (en) * | 2007-04-20 | 2008-10-29 | LFB Biotechnologies | Demannosylated recombinant factor VIII for the treatment of patients with hemophiila A |
| DE602008003684D1 (de) | 2007-04-26 | 2011-01-05 | Sangamo Biosciences Inc | Gezielte integration in die ppp1r12c-position |
| AU2009238629C1 (en) | 2008-04-14 | 2015-04-30 | Sangamo Therapeutics, Inc. | Linear donor constructs for targeted integration |
| DK2282764T3 (da) | 2008-04-22 | 2019-10-14 | Vib Vzw | Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf |
| US8703489B2 (en) | 2008-08-22 | 2014-04-22 | Sangamo Biosciences, Inc. | Methods and compositions for targeted single-stranded cleavage and targeted integration |
| ES2627552T3 (es) | 2008-12-04 | 2017-07-28 | Sigma Aldrich Company | Edición de genoma en ratas usando nucleasas con dedos de cinc |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| US8956828B2 (en) | 2009-11-10 | 2015-02-17 | Sangamo Biosciences, Inc. | Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases |
| EP2534173B1 (en) | 2010-02-08 | 2019-09-11 | Sangamo Therapeutics, Inc. | Engineered cleavage half-domains |
| EP2534163B1 (en) | 2010-02-09 | 2015-11-04 | Sangamo BioSciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| CN102939377B (zh) | 2010-04-26 | 2016-06-08 | 桑格摩生物科学股份有限公司 | 使用锌指核酸酶对Rosa位点进行基因组编辑 |
| EP2571512B1 (en) | 2010-05-17 | 2017-08-23 | Sangamo BioSciences, Inc. | Novel dna-binding proteins and uses thereof |
| AU2011281062B2 (en) | 2010-07-21 | 2015-01-22 | Board Of Regents, The University Of Texas System | Methods and compositions for modification of a HLA locus |
| US9175280B2 (en) | 2010-10-12 | 2015-11-03 | Sangamo Biosciences, Inc. | Methods and compositions for treating hemophilia B |
| US20140235693A1 (en) * | 2011-06-23 | 2014-08-21 | Alnylam Pharmaceuticals, Inc. | Serpina1 sirnas: compositions of matter and methods of treatment |
| KR102061557B1 (ko) | 2011-09-21 | 2020-01-03 | 상가모 테라퓨틱스, 인코포레이티드 | 이식 유전자 발현의 조절을 위한 방법 및 조성물 |
| AU2012328682B2 (en) | 2011-10-27 | 2017-09-21 | Sangamo Therapeutics, Inc. | Methods and compositions for modification of the HPRT locus |
| CA2854819C (en) | 2011-11-16 | 2022-07-19 | Sangamo Biosciences, Inc. | Modified dna-binding proteins and uses thereof |
| RS59670B1 (sr) | 2012-01-12 | 2020-01-31 | Bioverativ Therapeutics Inc | Himerni polipeptidi faktora viii i njihove upotrebe |
| EP3816281A1 (en) | 2012-07-11 | 2021-05-05 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
| KR20220164090A (ko) | 2012-08-29 | 2022-12-12 | 상가모 테라퓨틱스, 인코포레이티드 | 유전적 병태를 치료하기 위한 방법 및 조성물 |
| WO2014043131A1 (en) * | 2012-09-14 | 2014-03-20 | The Regents Of The University Of California | Lentiviral vector for stem cell gene therapy of sickle cell disease |
| DK2911687T3 (da) * | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
| US10398787B2 (en) * | 2012-10-26 | 2019-09-03 | Vrije Universiteit Brussel | Vectors for liver-directed gene therapy of hemophilia and methods and use thereof |
| AU2013355327A1 (en) | 2012-12-05 | 2015-06-11 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of metabolic disorders |
| AU2014246731A1 (en) * | 2013-03-30 | 2015-11-19 | Usha Biotech Limited | Methods and constructs for expressing biologically active proteins in mammalian cells |
| AU2014265331B2 (en) | 2013-05-15 | 2019-12-05 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| SI3044231T1 (sl) * | 2013-09-12 | 2020-12-31 | Biomarin Pharmaceutical Inc. | Vektorji AAV, ki vsebujejo gen, ki kodira faktor VIII |
| CN105940013B (zh) * | 2013-12-09 | 2020-03-27 | 桑格摩生物科学股份有限公司 | 用于治疗血友病的方法和组合物 |
| ES3009565T3 (en) * | 2015-04-16 | 2025-03-27 | Univ Emory | Recombinant promoters and vectors for protein expression in liver and use thereof |
| AU2016291778B2 (en) | 2015-07-13 | 2021-05-06 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| RU2020126034A (ru) | 2015-09-24 | 2020-09-02 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| EP3368664A4 (en) * | 2015-10-28 | 2019-08-28 | Sangamo Therapeutics, Inc. | LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF |
| AU2016353353B2 (en) | 2015-11-13 | 2020-07-09 | Takeda Pharmaceutical Company Limited | Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A |
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