JP2018531614A5 - - Google Patents
Download PDFInfo
- Publication number
- 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.)
- Granted
Links
- 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 (16)
| 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) |
| 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 (91)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | 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 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US20150166984A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | 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 |
| CA2991301A1 (en) | 2015-07-13 | 2017-01-19 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| WO2017070632A2 (en) | 2015-10-23 | 2017-04-27 | President And Fellows Of Harvard College | Nucleobase editors and uses thereof |
| US10143760B2 (en) * | 2015-10-28 | 2018-12-04 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor VIII expression cassettes and methods 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 |
| JP6994018B2 (ja) | 2016-07-26 | 2022-01-14 | バイオマリン ファーマシューティカル インコーポレイテッド | 新規アデノ随伴ウイルスキャプシドタンパク質 |
| WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
| AU2017308889B2 (en) | 2016-08-09 | 2023-11-09 | 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 |
| JP7256739B2 (ja) | 2016-09-07 | 2023-04-12 | サンガモ セラピューティクス， インコーポレイテッド | 肝臓遺伝子のモジュレーション |
| KR20240007715A (ko) | 2016-10-14 | 2024-01-16 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| KR102712926B1 (ko) | 2016-10-20 | 2024-10-07 | 상가모 테라퓨틱스, 인코포레이티드 | 파브리병의 치료를 위한 방법 및 조성물 |
| CN110214184B (zh) | 2016-12-01 | 2024-07-02 | 桑格摩生物治疗股份有限公司 | τ蛋白调节剂以及用于其递送的方法和组合物 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| KR20190123328A (ko) | 2017-03-09 | 2019-10-31 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 암 백신 |
| CN110914310A (zh) | 2017-03-10 | 2020-03-24 | 哈佛大学的校长及成员们 | 胞嘧啶至鸟嘌呤碱基编辑器 |
| IL306092A (en) | 2017-03-23 | 2023-11-01 | Harvard College | Nucleic base editors that include nucleic acid programmable DNA binding proteins |
| 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 |
| EP3658573A1 (en) | 2017-07-28 | 2020-06-03 | President and Fellows of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace) |
| US20200237930A1 (en) * | 2017-08-01 | 2020-07-30 | 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 |
| CA3082251A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | Uses of adenosine base editors |
| US20220080055A9 (en) * | 2017-10-17 | 2022-03-17 | Crispr Therapeutics Ag | Compositions and methods for gene editing for hemophilia a |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | 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 |
| AU2019215152A1 (en) | 2018-02-01 | 2020-08-20 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof |
| WO2019152841A1 (en) | 2018-02-01 | 2019-08-08 | Homology Medicines, Inc. | Adeno-associated virus compositions for pah gene transfer and methods of use thereof |
| BR112020015488A2 (pt) | 2018-02-08 | 2020-12-08 | Sangamo Therapeutics, Inc. | Nucleases alvo-específicas modificadas |
| EP3752616A1 (en) * | 2018-02-16 | 2020-12-23 | CRISPR Therapeutics AG | Compositions and methods for gene editing by targeting fibrinogen-alpha |
| US11306329B2 (en) | 2018-02-19 | 2022-04-19 | City Of Hope | Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof |
| KR20210008501A (ko) | 2018-05-09 | 2021-01-22 | 바이오마린 파머수티컬 인크. | 페닐케톤뇨증을 치료하는 방법 |
| CN112424345A (zh) * | 2018-05-14 | 2021-02-26 | 生物马林药物股份有限公司 | Aav载体在青少年对象中的稳定表达 |
| 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 |
| US11690921B2 (en) | 2018-05-18 | 2023-07-04 | Sangamo Therapeutics, Inc. | Delivery of target specific nucleases |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | 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 |
| EP3820495A4 (en) | 2018-07-09 | 2022-07-20 | The Broad Institute Inc. | RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES |
| AU2019314513B2 (en) * | 2018-08-03 | 2026-04-30 | Sangamo Therapeutics, Inc. | Improved clinical parameters by expression of factor viii |
| CN113226352B (zh) * | 2018-08-24 | 2025-08-26 | 星火治疗有限公司 | 最优化启动子序列、无内含子的表达结构及使用方法 |
| EP3852911B1 (en) | 2018-09-21 | 2025-01-22 | Acuitas Therapeutics, Inc. | Systems and methods for manufacturing lipid nanoparticles and liposomes |
| AU2019354296A1 (en) * | 2018-10-01 | 2021-04-29 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| IL326834A (en) | 2018-10-02 | 2026-04-01 | Sangamo Therapeutics Inc | Artificial zinc finger protein transcription factors repressing mapt expression for the prevention and/or treatment of tauopathies |
| IL326835A (en) * | 2018-10-16 | 2026-04-01 | Blueallele Llc | Methods for targeted insertion of dna genes |
| JP7578590B2 (ja) | 2018-10-18 | 2024-11-06 | インテリア セラピューティクス，インコーポレーテッド | 第ｉｘ因子を発現するための組成物及び方法 |
| CA3116576A1 (en) | 2018-10-18 | 2020-04-23 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| JP7529286B2 (ja) * | 2018-10-26 | 2024-08-06 | フリーイェ・ユニヴェルシテイト・ブリュッセル | 肝特異的核酸調節エレメント並びにその方法及び使用 |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| CN113423419A (zh) | 2019-01-04 | 2021-09-21 | 桑格摩生物治疗股份有限公司 | 用于治疗法布里病的方法和组合物 |
| PL3908568T3 (pl) | 2019-01-11 | 2024-12-02 | Acuitas Therapeutics, Inc. | Lipidy do dostarczania środków czynnych w nanocząstkach lipidowych |
| GB201900741D0 (en) | 2019-01-18 | 2019-03-06 | Synpromics Ltd | Liver-specifc inducible prometers and methods of use thereof |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| CA3106693A1 (en) | 2019-02-06 | 2020-08-13 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type i |
| SG11202108357PA (en) | 2019-02-15 | 2021-08-30 | Crispr Therapeutics Ag | Gene editing for hemophilia a with improved factor viii expression |
| KR20210151785A (ko) * | 2019-03-13 | 2021-12-14 | 제너레이션 바이오 컴퍼니 | 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도 |
| WO2020191241A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| JP7506686B2 (ja) * | 2019-04-04 | 2024-06-26 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | ヒト化凝固因子１２遺伝子座を含む非ヒト動物 |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | 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 |
| CN113939595A (zh) | 2019-06-07 | 2022-01-14 | 瑞泽恩制药公司 | 包括人源化白蛋白基因座的非人动物 |
| 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 |
| WO2021144649A2 (en) * | 2020-01-16 | 2021-07-22 | Takeda Pharmaceutical Company Limited | Adeno associated virus based gene therapy for phenylketonuria |
| US12686874B2 (en) | 2020-03-31 | 2026-07-21 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| CA3177481A1 (en) | 2020-05-08 | 2021-11-11 | David R. Liu | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| 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 |
| KR20230051172A (ko) | 2020-07-16 | 2023-04-17 | 아퀴타스 테라퓨틱스 인크. | 지질 나노 입자에 사용하기 위한 양이온성 지질 |
| AU2021375404A1 (en) | 2020-11-03 | 2023-06-08 | Pfizer Inc. | Methods for purification of aav vectors by anion exchange chromatography |
| 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 |
| MX2023015329A (es) | 2021-06-22 | 2024-01-23 | Pfizer | Produccion de vector de virus adeno-asociado en celulas de insecto. |
| 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 |
| WO2023114943A2 (en) | 2021-12-16 | 2023-06-22 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
| 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 |
| WO2025250454A1 (en) | 2024-05-28 | 2025-12-04 | University Of Rochester | Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo |
Family Cites Families (57)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1998024479A1 (en) | 1996-12-02 | 1998-06-11 | Cell Genesys, Inc. | Adeno-associated viral vector-mediated delivery of dna to cells of the liver |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| US6221349B1 (en) * | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US6503717B2 (en) | 1999-12-06 | 2003-01-07 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
| 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 |
| AU2001250774B2 (en) | 2000-02-08 | 2005-01-27 | Sangamo Biosciences, Inc. | Cells expressing zinc finger proteins for drug discovery |
| 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 |
| ATE347593T1 (de) | 2002-01-23 | 2006-12-15 | Univ Utah Res Found | Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen |
| 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 |
| CA2541523A1 (en) * | 2003-10-21 | 2005-05-06 | Applied Research Systems Ars Holding N.V. | 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 |
| KR20070060115A (ko) | 2004-09-16 | 2007-06-12 | 상가모 바이오사이언스 인코포레이티드 | 단백질 생산을 위한 조성물 및 방법 |
| WO2007021353A2 (en) | 2005-06-10 | 2007-02-22 | Genentech, Inc. | Improving antibody expression using vectors containing insulator elements |
| US7951925B2 (en) | 2006-05-25 | 2011-05-31 | Sangamo Biosciences, Inc. | Methods and compositions for gene inactivation |
| EP2027262B1 (en) | 2006-05-25 | 2010-03-31 | Sangamo Biosciences Inc. | Variant foki cleavage half-domains |
| EP1985631A1 (en) * | 2007-04-20 | 2008-10-29 | LFB Biotechnologies | Demannosylated recombinant factor VIII for the treatment of patients with hemophiila A |
| US8110379B2 (en) | 2007-04-26 | 2012-02-07 | Sangamo Biosciences, Inc. | Targeted integration into the PPP1R12C locus |
| CA2720903C (en) | 2008-04-14 | 2019-01-15 | Sangamo Biosciences, Inc. | Linear donor constructs for targeted integration |
| US9617548B2 (en) | 2008-04-22 | 2017-04-11 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| JP5908725B2 (ja) | 2008-08-22 | 2016-04-26 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 標的一本鎖開裂および標的組込みのための方法、並びに組成物 |
| EP3156494B8 (en) | 2008-12-04 | 2018-09-19 | Sangamo Therapeutics, Inc. | Genome editing in rats using zinc-finger nucleases |
| 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 |
| JP6137596B2 (ja) | 2010-02-08 | 2017-05-31 | サンガモ セラピューティクス， インコーポレイテッド | 遺伝子操作された切断ハーフドメイン |
| US9255259B2 (en) | 2010-02-09 | 2016-02-09 | Sangamo Biosciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| US9567573B2 (en) | 2010-04-26 | 2017-02-14 | Sangamo Biosciences, Inc. | Genome editing of a Rosa locus using nucleases |
| AU2011256838B2 (en) | 2010-05-17 | 2014-10-09 | Sangamo Therapeutics, Inc. | Novel DNA-binding proteins and uses thereof |
| CA2805442C (en) | 2010-07-21 | 2020-05-12 | Sangamo Biosciences, Inc. | Methods and compositions for modification of an hla locus |
| EP2627665B1 (en) * | 2010-10-12 | 2015-12-16 | The Children's Hospital Of Philadelphia | 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 |
| EP2758529B1 (en) * | 2011-09-21 | 2019-03-20 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of transgene expression |
| US9222105B2 (en) | 2011-10-27 | 2015-12-29 | Sangamo Biosciences, Inc. | Methods and compositions for modification of the HPRT locus |
| WO2013074999A1 (en) | 2011-11-16 | 2013-05-23 | Sangamo Biosciences, Inc. | Modified dna-binding proteins and uses thereof |
| HRP20191920T1 (hr) | 2012-01-12 | 2020-01-10 | Bioverativ Therapeutics Inc. | Kimerni polipeptidi faktora viii i njihova uporaba |
| HUE051612T2 (hu) | 2012-07-11 | 2021-03-01 | Sangamo Therapeutics Inc | Eljárások és készítmények lizoszomális tárolási betegségek kezelésére |
| EP2890780B8 (en) | 2012-08-29 | 2020-08-19 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| 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 |
| EP3513802B1 (en) * | 2012-10-26 | 2023-11-29 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| DK2911687T3 (da) * | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
| CA2892448A1 (en) | 2012-12-05 | 2014-06-12 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of metabolic disorders |
| MX2015013842A (es) * | 2013-03-30 | 2017-01-23 | Usha Biotech Ltd | Metodos y constructos para expresar proteinas biologicamente activas en celulas de mamifero. |
| EP2997146A4 (en) | 2013-05-15 | 2017-04-26 | Sangamo BioSciences, Inc. | Methods and compositions for treatment of a genetic condition |
| ES2813698T3 (es) * | 2013-09-12 | 2021-03-24 | Biomarin Pharm Inc | Vectores de AAV que comprenden un gen que codifica el factor VIII |
| CA3229275A1 (en) | 2013-12-09 | 2015-06-18 | Sangamo Biosciences, Inc. | Methods and compositions for genome engineering |
| CN115094062A (zh) * | 2015-04-16 | 2022-09-23 | 埃默里大学 | 用于肝脏中蛋白质表达的重组启动子和载体及其用途 |
| CA2991301A1 (en) | 2015-07-13 | 2017-01-19 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| JP2018535929A (ja) | 2015-09-24 | 2018-12-06 | バイオマリン ファーマシューティカル インコーポレイテッド | アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤 |
| US10143760B2 (en) | 2015-10-28 | 2018-12-04 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor VIII expression cassettes and methods of use thereof |
| CN108884145B (zh) | 2015-11-13 | 2023-08-22 | 武田药品工业株式会社 | 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体 |
-
2016
- 2016-07-13 US US15/209,363 patent/US10143760B2/en active Active
- 2016-07-13 AU AU2016343887A patent/AU2016343887B2/en active Active
- 2016-07-13 KR KR1020257003908A patent/KR20250022906A/ko active Pending
- 2016-07-13 JP JP2018521965A patent/JP6909212B2/ja active Active
- 2016-07-13 EP EP16860431.2A patent/EP3368664A4/en active Pending
- 2016-07-13 CN CN202210744050.5A patent/CN115141836B/zh active Active
- 2016-07-13 MY MYPI2018000606A patent/MY189674A/en unknown
- 2016-07-13 PE PE2018000630A patent/PE20181206A1/es unknown
- 2016-07-13 KR KR1020187014472A patent/KR102766526B1/ko active Active
- 2016-07-13 WO PCT/US2016/042099 patent/WO2017074526A1/en not_active Ceased
- 2016-07-13 MX MX2018005272A patent/MX2018005272A/es unknown
- 2016-07-13 CN CN201680067341.8A patent/CN108473976B/zh active Active
- 2016-07-13 PH PH1/2018/500927A patent/PH12018500927B1/en unknown
- 2016-07-13 SG SG11201803547RA patent/SG11201803547RA/en unknown
-
2018
- 2018-04-22 IL IL258826A patent/IL258826B2/en unknown
- 2018-04-24 ZA ZA2018/02723A patent/ZA201802723B/en unknown
- 2018-04-28 SA SA518391456A patent/SA518391456B1/ar unknown
- 2018-05-18 CO CONC2018/0005215A patent/CO2018005215A2/es unknown
- 2018-10-18 US US16/164,346 patent/US10828376B2/en active Active
- 2018-10-26 US US16/172,461 patent/US11452782B2/en active Active
-
2021
- 2021-07-02 JP JP2021110561A patent/JP7324249B2/ja active Active
-
2022
- 2022-09-01 US US17/929,157 patent/US20230119850A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7324249B2 (ja) | 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 | |
| Singh et al. | Efficient in vivo liver-directed gene editing using CRISPR/Cas9 | |
| Liu et al. | CRISPR modeling and correction of cardiovascular disease | |
| Newby et al. | In vivo somatic cell base editing and prime editing | |
| Huai et al. | CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice | |
| CN105940013B (zh) | 用于治疗血友病的方法和组合物 | |
| JP2016512683A5 (enExample) | ||
| WO2009130208A1 (en) | Liver-specific nucleic acid regulatory elements and methods and use thereof | |
| JP2017500061A5 (enExample) | ||
| JP2017504354A5 (enExample) | ||
| US20230190958A1 (en) | AAV Vector for Disrupting Coagulation Factor-Related Gene on Liver Genome | |
| Kantor et al. | Trends and challenges of AAV-delivered gene editing therapeutics for unmet needs in CNS disorders: Implications for neurodegenerative disease | |
| García‐Olloqui et al. | Effect of heart ischemia and administration route on biodistribution and transduction efficiency of AAV9 vectors | |
| Simoni et al. | In vivo liver targeted genome editing as therapeutic approach: progresses and challenges | |
| JP2020517270A5 (enExample) | ||
| JP2022529650A (ja) | 酵素発現を調節および自己不活性化するための組成物ならびに酵素のオフターゲット活性を調整するための方法 | |
| Trevisan et al. | Genome editing technologies to treat rare liver diseases | |
| Vo | Gene editing therapy as a therapeutic approach for cardiovascular diseases in animal models: A scoping review | |
| Thomas et al. | A promoterless AAV6. 2FF-based lung gene editing platform for the correction of surfactant protein B deficiency | |
| Segurado et al. | Challenges and opportunities when transitioning from in vivo gene replacement to in vivo CRISPR/Cas9 therapies–a spotlight on hemophilia | |
| Ameratunga et al. | Challenges for gene editing in common variable immunodeficiency disorders: Current and future prospects | |
| JP2019531754A5 (enExample) | ||
| Iizuka et al. | Neonatal gene therapy for hemophilia B by a novel adenovirus vector showing reduced leaky expression of viral genes | |
| JP2023524247A (ja) | アンジェルマン症候群の治療のための組成物及びその使用 | |
| Musunuru | An Overview of Genome Editing |