KR20230066360A - 신경퇴행성 장애를 위한 유전자 요법 - Google Patents
신경퇴행성 장애를 위한 유전자 요법 Download PDFInfo
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| BR112020006661A2 (pt) | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| CA3136004A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CN116672341A (zh) * | 2022-02-23 | 2023-09-01 | 中国科学院上海药物研究所 | 雷帕霉素在治疗渐冻症和额颞叶痴呆中的应用 |
| EP4598940A1 (en) | 2022-10-04 | 2025-08-13 | Eli Lilly and Company | Gene therapy for trem2-associated diseases and disorders |
| AU2023375569A1 (en) | 2022-11-11 | 2025-05-29 | Eli Lilly And Company | Synthetic nucleic acids including astrocyte-directed promoter constructs and methods of using the same |
| AU2023378899A1 (en) * | 2022-11-13 | 2025-06-26 | Alexion Pharma International Operations Limited | Gene therapy for frontotemporal dementia |
| WO2024163823A1 (en) * | 2023-02-02 | 2024-08-08 | Shape Therapeutics Inc. | Tissue-specific enhancers for regulating transcription |
| KR20250158063A (ko) * | 2023-03-16 | 2025-11-05 | 젠자임 코포레이션 | 건식 연령-관련 황반 변성의 치료 |
| WO2025251041A1 (en) * | 2024-05-31 | 2025-12-04 | Bluerock Therapeutics Lp | METHODS AND COMPOSITIONS FOR TREATING PROGRANULIN DEFICIENCIES USING iPSC-DERIVED CELLS |
| WO2026043765A1 (en) | 2024-08-21 | 2026-02-26 | Eli Lilly And Company | Engineered regulatory splice elements for gene therapies |
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| WO2008024998A2 (en) | 2006-08-24 | 2008-02-28 | Virovek, Inc. | Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor |
| EP3204503A1 (en) * | 2014-10-06 | 2017-08-16 | Arthrogen BV | Aav-based gene therapy |
| HK1256341A1 (zh) * | 2015-05-15 | 2019-09-20 | 明尼苏达大学董事会 | 用於治疗性递送到中枢神经系统的腺相关物 |
| US20190328906A1 (en) * | 2016-03-02 | 2019-10-31 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| JP7496667B2 (ja) | 2016-04-21 | 2024-06-07 | ビロベク，インコーポレイテッド | 昆虫細胞中でのａａｖ生成、方法およびその組成物 |
| IL316926A (en) * | 2017-05-11 | 2025-01-01 | Univ Pennsylvania | Gene therapy for neuronal ceroid lipofuscinosis |
| CA3068328A1 (en) * | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
| CN112501208A (zh) | 2017-10-03 | 2021-03-16 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| AU2018346104B2 (en) | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| BR112020006661A2 (pt) * | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| CA3136004A1 (en) * | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
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