JP2023535956A - 改善されたｘ連鎖性網膜分離症のａａｖ媒介療法 - Google Patents

改善されたｘ連鎖性網膜分離症のａａｖ媒介療法 Download PDF

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JP2023535956A
JP2023535956A JP2023505687A JP2023505687A JP2023535956A JP 2023535956 A JP2023535956 A JP 2023535956A JP 2023505687 A JP2023505687 A JP 2023505687A JP 2023505687 A JP2023505687 A JP 2023505687A JP 2023535956 A JP2023535956 A JP 2023535956A
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nucleic acid
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raav
sequence
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イー ボイ，シャノン
エル ボイ，サンフォード
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ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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JP2023505687A 2020-07-29 2021-07-28 改善されたｘ連鎖性網膜分離症のａａｖ媒介療法 Pending JP2023535956A (ja)

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US202063058437P 2020-07-29 2020-07-29
US63/058,437 2020-07-29
PCT/US2021/043582 WO2022026632A2 (en) 2020-07-29 2021-07-28 Improved aav-mediated x-linked retinoschisis therapies

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JP2023535956A true JP2023535956A (ja) 2023-08-22
JP2023535956A5 JP2023535956A5 (https=) 2024-07-05

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US (1) US20230265455A1 (https=)
EP (1) EP4188450A2 (https=)
JP (1) JP2023535956A (https=)
CN (1) CN116568338A (https=)
AU (1) AU2021319065A1 (https=)
CA (1) CA3190214A1 (https=)
IL (1) IL299927A (https=)
WO (1) WO2022026632A2 (https=)

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US20240124893A1 (en) * 2021-11-03 2024-04-18 Teamedon International, Inc. Methods of Treating Human X-Linked Retinoschisis Using Gene Therapy
EP4499847A4 (en) * 2022-03-30 2026-03-25 Univ Pittsburgh Commonwealth Sys Higher Education Adeno-associated viral vectors for the delivery of nucleic acids across retinal regions
CN114848850B (zh) * 2022-04-28 2023-12-15 武汉中眸生物科技有限责任公司 Rs1基因在制备xlrs治疗剂中的应用及治疗剂
CN118359687A (zh) * 2023-01-18 2024-07-19 上海朗昇生物科技有限公司 一种新衣壳蛋白变体以及其用途
EP4704920A1 (en) * 2023-05-04 2026-03-11 Abeona Therapeutics Inc. Recombinant adeno-associated viral vectors for multipartite gene delivery and stargardt disease treatment
WO2025022144A1 (en) * 2023-07-26 2025-01-30 Sorbonne Universite Chimeric promoter for targeted expression in aii amacrine cells
WO2025091025A1 (en) * 2023-10-26 2025-05-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders relating to sirt1
WO2025113432A1 (zh) * 2023-11-27 2025-06-05 北京因诺惟康医药科技有限公司 一种用于治疗x染色体连锁视网膜劈裂症的基因药物
WO2025231339A1 (en) * 2024-05-02 2025-11-06 Avista Therapeutics, Inc. Adeno-associated virus vectors for therapeutic nucleic acid delivery to retinal cells

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EP4188450A2 (en) 2023-06-07
WO2022026632A3 (en) 2022-03-10
AU2021319065A1 (en) 2023-02-16
CA3190214A1 (en) 2022-02-03
US20230265455A1 (en) 2023-08-24
WO2022026632A2 (en) 2022-02-03
IL299927A (en) 2023-03-01
CN116568338A (zh) 2023-08-08

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