CN112204145B - 用于治疗显性视网膜色素变性的组合物和方法 - Google Patents

用于治疗显性视网膜色素变性的组合物和方法 Download PDF

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CN112204145B
CN112204145B CN201980036506.9A CN201980036506A CN112204145B CN 112204145 B CN112204145 B CN 112204145B CN 201980036506 A CN201980036506 A CN 201980036506A CN 112204145 B CN112204145 B CN 112204145B
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rho
vector
seq
shrna
nucleotide sequence
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CN112204145A (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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CN201980036506.9A 2018-06-01 2019-06-03 用于治疗显性视网膜色素变性的组合物和方法 Active CN112204145B (zh)

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US201862679585P 2018-06-01 2018-06-01
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US201962809539P 2019-02-22 2019-02-22
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PCT/US2019/035159 WO2019232517A1 (en) 2018-06-01 2019-06-03 Compositions and methods for treatment of dominant retinitis pigmentosa

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JP7334346B2 (ja) * 2020-07-24 2023-08-28 アールズィーノミクス・インコーポレイテッド ロドプシン転写体に特異的なトランススプライシングリボザイム及びその用途
US20220098615A1 (en) * 2020-09-30 2022-03-31 NGGT, Inc. Dual functional expression vectors and methods of use thereof
WO2024211266A1 (en) * 2023-04-07 2024-10-10 University Of Houston System Downregulation of rhodopsin as a therapeutic strategy for peripherin-2-associated inherited retinal disorders

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WO2019232517A1 (en) 2019-12-05
US12203074B2 (en) 2025-01-21
US20210207147A1 (en) 2021-07-08
AU2019276645A1 (en) 2020-11-26
JP7563694B2 (ja) 2024-10-08
EP3802836A4 (en) 2022-10-19
EP3802836A1 (en) 2021-04-14
CN118480544A (zh) 2024-08-13
CN112204145A (zh) 2021-01-08
AU2019276645B2 (en) 2025-06-26
CA3102095A1 (en) 2019-12-05

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