CN109154002B - 用于治疗显性视网膜色素变性的aav载体 - Google Patents

用于治疗显性视网膜色素变性的aav载体 Download PDF

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CN109154002B
CN109154002B CN201780014309.8A CN201780014309A CN109154002B CN 109154002 B CN109154002 B CN 109154002B CN 201780014309 A CN201780014309 A CN 201780014309A CN 109154002 B CN109154002 B CN 109154002B
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rna
rho
sequence
vector
gene
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CN109154002A (zh
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阿尔弗雷德·S·莱温
威廉·W·豪斯维特
迈克尔·T·马森吉尔
威廉·贝尔特兰
古斯塔沃·D·阿吉雷
阿图尔·西代西扬
塞缪尔·雅各布森
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University of Pennsylvania Penn
University of Florida Research Foundation Inc
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CN201780014309.8A 2016-03-01 2017-03-01 用于治疗显性视网膜色素变性的aav载体 Active CN109154002B (zh)

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US201662302122P 2016-03-01 2016-03-01
US62/302,122 2016-03-01
US201662398451P 2016-09-22 2016-09-22
US62/398,451 2016-09-22
PCT/US2017/020289 WO2017151823A1 (en) 2016-03-01 2017-03-01 Aav vectors for treatment of dominant retinitis pigmentosa

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IL261014B2 (en) 2016-03-01 2023-10-01 Univ Florida Aav vectors for treatment of dominant retinitis pigmentosa
EP3707257A4 (en) * 2017-11-07 2021-12-08 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR CIRCULAR RNA MOLECULES
EP3793566A4 (en) * 2018-05-15 2022-03-16 University of Washington Compositions and methods for reducing spliceopathy and treating rna dominance disorders
JP7563694B2 (ja) * 2018-06-01 2024-10-08 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 優性網膜色素変性症の処置のための組成物および方法
GB201817469D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy for retinal disease
CN111518813B (zh) * 2019-02-03 2023-04-28 武汉纽福斯生物科技有限公司 视紫红质的编码序列、其表达载体构建及其应用
WO2021030745A1 (en) * 2019-08-15 2021-02-18 The Children's Hospital Of Philadelphia Combined transgene and intron-derived mirna therapy for treatment of sca1
CN113038971B (zh) * 2020-04-21 2022-06-24 北京中因科技有限公司 RHO-adRP基于基因编辑的方法和组合物
CN111926044B (zh) * 2020-10-12 2021-01-22 北京大学第三医院（北京大学第三临床医学院） 结合突变rho基因的核酸分子和试剂盒
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2025231487A1 (en) * 2024-05-03 2025-11-06 The Trustees Of The University Of Pennsylvania Photoreceptor specific promotors for use in gene therapies for treatment of retinal degeneration

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JP2019511216A (ja) 2019-04-25
EP3423582A4 (en) 2019-11-13
EP3423582B1 (en) 2022-05-04
JP2025016451A (ja) 2025-02-04
IL261014B2 (en) 2023-10-01
CN109154002A (zh) 2019-01-04
CA3014671A1 (en) 2017-09-08
EP4119668A1 (en) 2023-01-18
JP7642211B2 (ja) 2025-03-10
JP2022185052A (ja) 2022-12-13
US11118185B2 (en) 2021-09-14
AU2017227776B2 (en) 2023-07-06
US20210324387A1 (en) 2021-10-21
AU2017227776A1 (en) 2018-08-23
AU2017227776C1 (en) 2024-01-25
JP7272795B2 (ja) 2023-05-12
AU2023237106B2 (en) 2025-08-28
AU2023237106A1 (en) 2023-10-19
IL302455A (en) 2023-06-01
ES2923877T3 (es) 2022-10-03
EP3423582A1 (en) 2019-01-09
WO2017151823A1 (en) 2017-09-08
IL261014B1 (en) 2023-06-01
IL261014A (en) 2018-10-31
US20190093111A1 (en) 2019-03-28
CN115896106A (zh) 2023-04-04

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