CA3041548A1 - Gene transfer compositions, methods and uses for treating neurodegenerative diseases - Google Patents
Gene transfer compositions, methods and uses for treating neurodegenerative diseases Download PDFInfo
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- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- 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
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- C12Y—ENZYMES
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- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
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- 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
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Landscapes
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| US201662418033P | 2016-11-04 | 2016-11-04 | |
| US62/418,033 | 2016-11-04 | ||
| PCT/US2017/059986 WO2018085688A1 (en) | 2016-11-04 | 2017-11-03 | Gene transfer compositions, methods and uses for treating neurodegenerative diseases |
Publications (1)
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| CA3041548A1 true CA3041548A1 (en) | 2018-05-11 |
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| US9818239B2 (en) | 2015-08-20 | 2017-11-14 | Zendrive, Inc. | Method for smartphone-based accident detection |
| IL271193B2 (en) | 2017-06-07 | 2025-01-01 | Regeneron Pharma | Preparations and methods for internalizing enzymes |
| WO2019079807A1 (en) | 2017-10-20 | 2019-04-25 | Zendrive, Inc. | METHOD AND SYSTEM FOR VEHICULAR COMMUNICATIONS |
| WO2019157224A1 (en) | 2018-02-07 | 2019-08-15 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| AU2019269685B2 (en) | 2018-05-17 | 2025-12-04 | Regeneron Pharmaceuticals, Inc. | Anti-CD63 antibodies, conjugates, and uses thereof |
| JP7624724B2 (ja) | 2018-09-26 | 2025-01-31 | カリフォルニア インスティテュート オブ テクノロジー | 標的遺伝子療法のためのアデノ随伴ウイルス組成物 |
| AU2019379141A1 (en) * | 2018-11-14 | 2021-06-03 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| MX2021009250A (es) * | 2019-02-01 | 2021-11-04 | Spark Therapeutics Inc | Metodos de tratamiento por vectores aav para la lipofuscinosis ceroidea neuronal infantil tardia tipo 2. |
| WO2021113475A1 (en) | 2019-12-03 | 2021-06-10 | Zendrive, Inc. | Method and system for risk determination of a route |
| EP4118219A4 (en) * | 2020-03-11 | 2024-04-17 | Shanghai Belief-Delivery Biomed Co., Ltd. | Novel use of aspirin compound in increasing nucleic acid expression |
| WO2025231406A1 (en) * | 2024-05-02 | 2025-11-06 | The Children's Hospital Of Philadelphia | Methods to increase transduction of ependyma cells in brain |
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| WO2003089011A1 (en) * | 2002-04-19 | 2003-10-30 | University Of Florida | rAAV VECTOR-BASED COMPOSITIONS AND METHODS FOR THE PREVENTION AND TREATMENT OF MAMMALIAN DISEASES |
| US20040258666A1 (en) * | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
| JPWO2005071085A1 (ja) * | 2004-01-22 | 2007-09-06 | 株式会社ディナベック研究所 | ウイルスベクターの製造方法 |
| HUE041928T2 (hu) * | 2006-02-08 | 2019-06-28 | Genzyme Corp | Génterápia A-típusú Niemann-Pick-betegségre |
| EP3252161B1 (en) * | 2007-06-06 | 2021-11-17 | Genzyme Corporation | Gene therapy for lysosomal storage diseases |
| US9849195B2 (en) * | 2011-03-31 | 2017-12-26 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| MX2016001044A (es) * | 2013-07-22 | 2016-09-09 | The Children's Hospital Of Philadelphia | Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos. |
| MX373332B (es) * | 2013-07-26 | 2020-05-21 | Univ Iowa Res Found | UNA PARTÍCULA rAAV Y UN AGENTE DE INMUNOSUPRESIÓN PARA USARSE EN EL TRATAMIENTO DE ENFERMEDADES. |
| CN105745326A (zh) * | 2013-10-24 | 2016-07-06 | 优尼科Ip有限公司 | 用于基因治疗神经疾病的aav-5假型载体 |
| KR20160079891A (ko) * | 2013-11-20 | 2016-07-06 | 유니버시티 오브 아이오와 리써치 파운데이션 | 아밀로이드 침착의 치료를 위한 방법 및 조성물 |
| WO2017070678A1 (en) * | 2015-10-23 | 2017-04-27 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| WO2018209205A1 (en) * | 2017-05-11 | 2018-11-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for neuronal ceroid lipofuscinoses |
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Also Published As
| Publication number | Publication date |
|---|---|
| EP3534892A4 (en) | 2020-05-27 |
| US20190269797A1 (en) | 2019-09-05 |
| RU2019117062A (ru) | 2020-12-04 |
| EP3534892A1 (en) | 2019-09-11 |
| WO2018085688A1 (en) | 2018-05-11 |
| MX2019005266A (es) | 2019-09-27 |
| CN110198712A (zh) | 2019-09-03 |
| BR112019009074A2 (pt) | 2019-07-16 |
| AU2017355502B2 (en) | 2023-08-31 |
| AU2017355502A1 (en) | 2019-05-16 |
| JP2019537576A (ja) | 2019-12-26 |
| JP2023002721A (ja) | 2023-01-10 |
| RU2019117062A3 (enExample) | 2021-03-11 |
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