JP2025120285A5 - - Google Patents

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Publication number
JP2025120285A5
JP2025120285A5 JP2025093546A JP2025093546A JP2025120285A5 JP 2025120285 A5 JP2025120285 A5 JP 2025120285A5 JP 2025093546 A JP2025093546 A JP 2025093546A JP 2025093546 A JP2025093546 A JP 2025093546A JP 2025120285 A5 JP2025120285 A5 JP 2025120285A5
Authority
JP
Japan
Prior art keywords
promoter
vector
pharmaceutical composition
seq
composition according
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2025093546A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025120285A (ja
Filing date
Publication date
Priority claimed from GBGB1707212.5A external-priority patent/GB201707212D0/en
Application filed filed Critical
Publication of JP2025120285A publication Critical patent/JP2025120285A/ja
Publication of JP2025120285A5 publication Critical patent/JP2025120285A5/ja
Pending legal-status Critical Current

Links

JP2025093546A 2017-05-05 2025-06-04 繊毛病のための遺伝子治療 Pending JP2025120285A (ja)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
GBGB1707212.5A GB201707212D0 (en) 2017-05-05 2017-05-05 Gene therapy for ciliopathies
GB1707212.5 2017-05-05
PCT/GB2018/051219 WO2018203092A1 (en) 2017-05-05 2018-05-04 Gene therapy for ciliopathies
JP2019560667A JP7759174B2 (ja) 2017-05-05 2018-05-04 繊毛病のための遺伝子治療

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2019560667A Division JP7759174B2 (ja) 2017-05-05 2018-05-04 繊毛病のための遺伝子治療

Publications (2)

Publication Number Publication Date
JP2025120285A JP2025120285A (ja) 2025-08-15
JP2025120285A5 true JP2025120285A5 (https=) 2026-01-09

Family

ID=59065740

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019560667A Active JP7759174B2 (ja) 2017-05-05 2018-05-04 繊毛病のための遺伝子治療
JP2025093546A Pending JP2025120285A (ja) 2017-05-05 2025-06-04 繊毛病のための遺伝子治療

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2019560667A Active JP7759174B2 (ja) 2017-05-05 2018-05-04 繊毛病のための遺伝子治療

Country Status (8)

Country Link
US (2) US12171794B2 (https=)
EP (1) EP3628011A1 (https=)
JP (2) JP7759174B2 (https=)
CN (1) CN110753559A (https=)
AU (1) AU2018262427B2 (https=)
CA (1) CA3060187A1 (https=)
GB (1) GB201707212D0 (https=)
WO (1) WO2018203092A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201707212D0 (en) 2017-05-05 2017-06-21 Ucl Business Plc Gene therapy for ciliopathies
CN118949075A (zh) * 2019-02-14 2024-11-15 埃泽瑞斯公司 纤毛疾病的治疗
KR20220083714A (ko) * 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
GB202005641D0 (en) * 2020-04-17 2020-06-03 Ucl Business Plc Gene therapy for bardet-biedl syndrome
CN116042719B (zh) * 2022-12-28 2025-11-11 蓝图生物医药(广州)有限公司 一种重组腺相关病毒载体及其应用
WO2025049923A2 (en) * 2023-08-31 2025-03-06 The Board Of Trustees Of The Leland Stanford Junior University Novel treatments of ciliopathies

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6962788B2 (en) 2002-05-30 2005-11-08 University Of Iowa Research Foundation Identification of a gene causing the most common form of bardet-biedl syndrome and uses thereof
US8163482B2 (en) * 2006-02-23 2012-04-24 Nicholas Katsanis BBS10 related diagnostic methods for Bardet-Biedl syndrome
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
ES2697249T3 (es) * 2013-03-11 2019-01-22 Fond Telethon miR-204 y miR-211 y usos de los mismos
GB201707212D0 (en) 2017-05-05 2017-06-21 Ucl Business Plc Gene therapy for ciliopathies

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