JP2020518269A5 - - Google Patents

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Publication number
JP2020518269A5
JP2020518269A5 JP2019560667A JP2019560667A JP2020518269A5 JP 2020518269 A5 JP2020518269 A5 JP 2020518269A5 JP 2019560667 A JP2019560667 A JP 2019560667A JP 2019560667 A JP2019560667 A JP 2019560667A JP 2020518269 A5 JP2020518269 A5 JP 2020518269A5
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JP
Japan
Prior art keywords
vector
ciliopathy
seq
promoter
vector 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.)
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Application number
JP2019560667A
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English (en)
Japanese (ja)
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JP7759174B2 (ja
JP2020518269A (ja
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Priority claimed from GBGB1707212.5A external-priority patent/GB201707212D0/en
Application filed filed Critical
Publication of JP2020518269A publication Critical patent/JP2020518269A/ja
Publication of JP2020518269A5 publication Critical patent/JP2020518269A5/ja
Priority to JP2025093546A priority Critical patent/JP2025120285A/ja
Application granted granted Critical
Publication of JP7759174B2 publication Critical patent/JP7759174B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2019560667A 2017-05-05 2018-05-04 繊毛病のための遺伝子治療 Active JP7759174B2 (ja)

Priority Applications (1)

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

Applications Claiming Priority (3)

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

Related Child Applications (1)

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

Publications (3)

Publication Number Publication Date
JP2020518269A JP2020518269A (ja) 2020-06-25
JP2020518269A5 true JP2020518269A5 (https=) 2021-07-26
JP7759174B2 JP7759174B2 (ja) 2025-10-23

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 After (1)

Application Number Title Priority Date Filing Date
JP2025093546A Pending JP2025120285A (ja) 2017-05-05 2025-06-04 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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