KR20200015701A - 레트 증후군에서의 MeCP2의 안전한 발현을 위한 자가-조절 AAV 벡터 - Google Patents
레트 증후군에서의 MeCP2의 안전한 발현을 위한 자가-조절 AAV 벡터 Download PDFInfo
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- KR20200015701A KR20200015701A KR1020207000120A KR20207000120A KR20200015701A KR 20200015701 A KR20200015701 A KR 20200015701A KR 1020207000120 A KR1020207000120 A KR 1020207000120A KR 20207000120 A KR20207000120 A KR 20207000120A KR 20200015701 A KR20200015701 A KR 20200015701A
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| US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
| EP3256170B1 (en) | 2015-02-13 | 2020-09-23 | University of Massachusetts | Compositions and methods for transient delivery of nucleases |
| JP7106534B2 (ja) * | 2016-11-17 | 2022-07-26 | ネイションワイド チルドレンズ ホスピタル， インコーポレイテッド | メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達 |
| JP7618386B2 (ja) | 2017-01-30 | 2025-01-21 | ブレインヴェクティス | ポリグルタミン反復脊髄小脳失調症の処置におけるコレステロール２４－ヒドロキシラーゼ（ｈｙｄｒｏｌａｓｅ）のための発現ベクター |
| AU2019247748A1 (en) | 2018-04-03 | 2020-10-08 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| EP3774852A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| CN113166208B (zh) | 2018-10-02 | 2024-11-22 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| CA3133889A1 (en) * | 2019-04-24 | 2020-10-29 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| JP7624410B2 (ja) * | 2019-05-21 | 2025-01-30 | アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル | レット症候群の治療におけるコレステロール ２４－ヒドロラーゼのための発現ベクター |
| US12611436B2 (en) | 2019-10-17 | 2026-04-28 | Sarepta Therapeutics, Inc. | AAV transfer cassette |
| JP2022551739A (ja) | 2019-10-17 | 2022-12-13 | ストライドバイオ，インコーポレイテッド | ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター |
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| US12497612B2 (en) | 2020-03-09 | 2025-12-16 | University Of Massachusetts | Gene replacement therapy for FOXG1 syndrome |
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| TWI900553B (zh) | 2020-05-13 | 2025-10-11 | 美商航海家醫療公司 | Aav蛋白殼之趨性重定向 |
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| AU2021328475A1 (en) * | 2020-08-19 | 2023-03-16 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of Rett syndrome |
| CN113754727B (zh) * | 2020-09-30 | 2022-07-12 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2022094078A1 (en) * | 2020-10-28 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| EP4426716A1 (en) | 2021-11-02 | 2024-09-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| GB202201242D0 (en) * | 2022-01-31 | 2022-03-16 | Univ Edinburgh | Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders |
| CN121219020A (zh) * | 2023-03-22 | 2025-12-26 | 圣拉斐尔医院有限责任公司 | 基因疗法 |
| EP4715058A1 (en) * | 2023-05-15 | 2026-03-25 | Genecombio Ltd. | Gene therapy for rett syndrome |
| CN119101681A (zh) * | 2023-06-09 | 2024-12-10 | 仁景(苏州)生物科技有限公司 | 表达可调控的工程化rna分子 |
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