TW202334194A - 用於表現因子ｉｘ以便治療ｂ型血友病的組成物及方法 - Google Patents
用於表現因子ｉｘ以便治療ｂ型血友病的組成物及方法 Download PDFInfo
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- TW202334194A TW202334194A TW111140939A TW111140939A TW202334194A TW 202334194 A TW202334194 A TW 202334194A TW 111140939 A TW111140939 A TW 111140939A TW 111140939 A TW111140939 A TW 111140939A TW 202334194 A TW202334194 A TW 202334194A
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
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- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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| WO2025160324A2 (en) | 2024-01-26 | 2025-07-31 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for using plasma cell depleting agents and/or b cell depleting agents to suppress host anti-aav antibody response and enable aav transduction and re-dosing |
| US20250276092A1 (en) | 2024-03-01 | 2025-09-04 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response |
| WO2025265017A1 (en) * | 2024-06-20 | 2025-12-26 | Regeneron Pharmaceuticals, Inc. | Ass1 gene insertion for the treatment of citrullinemia type i |
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| WO2003087341A2 (en) | 2002-01-23 | 2003-10-23 | The University Of Utah Research Foundation | Targeted chromosomal mutagenesis using zinc finger nucleases |
| WO2003080809A2 (en) | 2002-03-21 | 2003-10-02 | Sangamo Biosciences, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
| EP2806025B1 (en) | 2002-09-05 | 2019-04-03 | California Institute of Technology | Use of zinc finger 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 |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
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| EP2027262B1 (en) | 2006-05-25 | 2010-03-31 | Sangamo Biosciences Inc. | Variant foki cleavage half-domains |
| ES2465996T3 (es) | 2006-05-25 | 2014-06-09 | Sangamo Biosciences, Inc. | Métodos y composiciones para la inactivación genética |
| US8022187B2 (en) | 2007-02-01 | 2011-09-20 | Baxter International Inc. | FVIII-independent FIX-mutant proteins for hemophilia A treatment |
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| DE602008003684D1 (de) | 2007-04-26 | 2011-01-05 | Sangamo Biosciences Inc | Gezielte integration in die ppp1r12c-position |
| CA2720903C (en) | 2008-04-14 | 2019-01-15 | Sangamo Biosciences, Inc. | Linear donor constructs for targeted integration |
| EP2149603A1 (en) | 2008-07-28 | 2010-02-03 | DRK-Blutspendedienst Baden-Württemberg-Hessen gGmbH | Factor IX variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders |
| SG191561A1 (en) | 2008-08-22 | 2013-07-31 | Sangamo Biosciences Inc | Methods and compositions for targeted single-stranded cleavage and targeted integration |
| US8586526B2 (en) | 2010-05-17 | 2013-11-19 | Sangamo Biosciences, Inc. | DNA-binding proteins and uses thereof |
| EP2660318A1 (en) | 2010-02-09 | 2013-11-06 | 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 |
| CA2848417C (en) | 2011-09-21 | 2023-05-02 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
| CA3099582A1 (en) | 2011-10-27 | 2013-05-02 | Sangamo Biosciences, Inc. | Methods and compositions for modification of the hprt locus |
| AU2013266968B2 (en) | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| EP2881463A1 (en) | 2013-12-09 | 2015-06-10 | DRK-Blutspendedienst Baden-Württemberg-Hessen gGmbH | Factor IX variants with clotting activity in absence of their cofactor and/or with increased F.IX clotting activity and their use for treating bleeding disorders |
| 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 |
| MX390848B (es) * | 2015-06-23 | 2025-03-21 | Childrens Hospital Philadelphia | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. |
| CN110945127B (zh) * | 2017-05-22 | 2024-07-12 | 武田药品工业株式会社 | 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体 |
| JP7119009B2 (ja) * | 2017-05-31 | 2022-08-16 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | 最適化されたヒト凝固第ｉｘ因子遺伝子発現カセットおよびそれらの使用 |
| JP2021533799A (ja) * | 2018-08-20 | 2021-12-09 | ユーシーエル ビジネス リミテッド | 第ｉｘ因子をコードするヌクレオチド |
| US20200270617A1 (en) | 2018-10-18 | 2020-08-27 | Intellia Therapeutics, Inc. | Compositions and methods for transgene expression from an albumin locus |
| CN113272428A (zh) | 2018-10-18 | 2021-08-17 | 英特利亚治疗股份有限公司 | 核酸构建体和使用方法 |
| AU2019360270B2 (en) | 2018-10-18 | 2025-08-07 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor IX. |
| US11596671B2 (en) * | 2019-02-01 | 2023-03-07 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia B using viral vectors encoding recombinant fix variants with increased expression |
| AU2020242945B2 (en) * | 2019-03-19 | 2025-06-26 | CSL Innovation Pty Ltd | Factor IX variants and uses thereof in therapy |
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| US20230149563A1 (en) | 2023-05-18 |
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