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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| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| US10584321B2 (en) | 2015-02-13 | 2020-03-10 | 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 |
| WO2018138371A1 (en) | 2017-01-30 | 2018-08-02 | Brainvectis | Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias |
| SG11202009451VA (en) | 2018-04-03 | 2020-10-29 | Stridebio Inc | Antibody-evading virus vectors |
| JP7406677B2 (ja) | 2018-04-03 | 2023-12-28 | ギンコ バイオワークス インコーポレイテッド | 抗体を回避するウイルスベクター |
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| US12467046B2 (en) | 2018-10-02 | 2025-11-11 | Voyager Therapeutics, Inc. | Redirection of tropism of AAV capsids |
| CA3133453A1 (en) | 2019-03-21 | 2020-09-24 | Daniel Mccoy | Recombinant adeno-associated virus vectors |
| KR20220003553A (ko) * | 2019-04-24 | 2022-01-10 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 레트 증후군의 치료에 유용한 조성물 |
| EP3972631B1 (en) * | 2019-05-21 | 2024-07-10 | Institut National de la Santé et de la Recherche Médicale (INSERM) | Expression vector for cholesterol 24-hydrolase in therapy of rett syndrome |
| TW202128736A (zh) | 2019-10-17 | 2021-08-01 | 美商史崔德生物公司 | 用於治療ｃ型尼曼—匹克病之腺相關病毒載體 |
| JP7702949B2 (ja) * | 2019-12-05 | 2025-07-04 | ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム | ヒトｍｅｃｐ２遺伝子を発現するように設計されたトランスジーンカセット |
| WO2021183433A1 (en) | 2020-03-09 | 2021-09-16 | University Of Massachusetts | Gene replacement therapy for foxg1 syndrome |
| CN116096734A (zh) * | 2020-05-13 | 2023-05-09 | 沃雅戈治疗公司 | Aav衣壳的向性的重定向 |
| IL299414A (en) * | 2020-06-30 | 2023-02-01 | Univ Court Univ Of Edinburgh | Transgene expression system |
| CN116745409A (zh) * | 2020-08-19 | 2023-09-12 | 萨雷普塔治疗公司 | 用于治疗雷特综合征的腺相关病毒载体 |
| CN113717248B (zh) * | 2020-09-30 | 2022-07-08 | 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 |
| US20250001012A1 (en) | 2021-11-02 | 2025-01-02 | 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 |
| AU2024241563A1 (en) * | 2023-03-22 | 2025-10-09 | Consiglio Nazionale Delle Ricerche | Gene therapy |
| CN121175427A (zh) * | 2023-05-15 | 2025-12-19 | 上海金珂博生物技术有限公司 | 一种Rett综合症的基因治疗 |
| TW202513796A (zh) * | 2023-06-09 | 2025-04-01 | 大陸商仁景（蘇州）生物科技有限公司 | 表達可調控的工程化ｒｎａ分子及其用途 |
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| NZ618298A (en) | 2001-11-13 | 2015-04-24 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
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| EP3564379A1 (en) | 2013-09-13 | 2019-11-06 | California Institute of Technology | Selective recovery |
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