KR20230065367A - Pah 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 - Google Patents
Pah 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 Download PDFInfo
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- KR20230065367A KR20230065367A KR1020237014637A KR20237014637A KR20230065367A KR 20230065367 A KR20230065367 A KR 20230065367A KR 1020237014637 A KR1020237014637 A KR 1020237014637A KR 20237014637 A KR20237014637 A KR 20237014637A KR 20230065367 A KR20230065367 A KR 20230065367A
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- amino acid
- seq
- capsid protein
- protein corresponding
- gly
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| IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| TW202016314A (zh) | 2018-05-09 | 2020-05-01 | 美商拜奧馬林製藥公司 | 治療苯丙酮尿症之方法 |
| 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 |
| WO2020251954A1 (en) * | 2019-06-10 | 2020-12-17 | Homology Medicines, Inc. | Adeno-associated virus compositions for arsa gene transfer and methods of use thereof |
| 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 |
| JP2023510392A (ja) * | 2020-01-16 | 2023-03-13 | 武田薬品工業株式会社 | フェニルケトン尿症に対するアデノ随伴ウイルスベースの遺伝子治療 |
| 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 |
| CN116656740A (zh) * | 2023-05-30 | 2023-08-29 | 浙江大学 | 一种腺相关病毒基因治疗载体及其在制备治疗苯丙酮尿症的药物中的应用 |
| CN119876192B (zh) * | 2024-07-02 | 2025-11-07 | 浙江大学滨江研究院 | 用于治疗苯丙酮尿症的多核苷酸 |
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| DE69918090T2 (de) * | 1998-04-24 | 2005-06-16 | University Of Florida, Gainesville | Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie |
| US20030198620A1 (en) | 2002-04-16 | 2003-10-23 | Keiya Ozawa | Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions |
| US20040142416A1 (en) * | 2002-04-30 | 2004-07-22 | Laipis Philip J. | Treatment for phenylketonuria |
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| US9839696B2 (en) * | 2010-04-30 | 2017-12-12 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| TR201908020T4 (tr) * | 2011-02-17 | 2019-06-21 | Univ Pennsylvania | Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler. |
| JP6506749B2 (ja) | 2013-10-22 | 2019-04-24 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | フェニルケトン尿症のためのｍＲＮＡ療法 |
| AU2015320694B2 (en) * | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| KR102738752B1 (ko) * | 2016-12-30 | 2024-12-06 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 페닐케톤뇨증을 치료하기 위한 유전자 치료 |
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- 2019-02-01 BR BR112020015511-0A patent/BR112020015511A2/pt not_active IP Right Cessation
- 2019-02-01 EA EA202091844A patent/EA202091844A1/ru unknown
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| KR20200127187A (ko) | 2020-11-10 |
| IL276215B (en) | 2022-10-01 |
| PE20210341A1 (es) | 2021-02-23 |
| CN111886021A (zh) | 2020-11-03 |
| BR112020015511A2 (pt) | 2021-01-26 |
| IL276215B2 (en) | 2023-02-01 |
| JP2021512601A (ja) | 2021-05-20 |
| JP7066861B2 (ja) | 2022-05-13 |
| KR102528641B1 (ko) | 2023-05-03 |
| EA202091844A1 (ru) | 2021-02-03 |
| CO2020010783A2 (es) | 2020-11-20 |
| JP2022105105A (ja) | 2022-07-12 |
| CA3089824A1 (en) | 2019-08-08 |
| MX2020008085A (es) | 2020-12-10 |
| AU2019215150A1 (en) | 2020-09-24 |
| JP7433360B2 (ja) | 2024-02-19 |
| IL295830A (en) | 2022-10-01 |
| IL276215A (en) | 2020-09-30 |
| EP3746113A4 (en) | 2022-03-09 |
| WO2019152841A1 (en) | 2019-08-08 |
| EP3746113A1 (en) | 2020-12-09 |
| CL2020001992A1 (es) | 2021-02-12 |
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