JP2021520232A5 - - Google Patents

Info

Publication number
JP2021520232A5
JP2021520232A5 JP2021504131A JP2021504131A JP2021520232A5 JP 2021520232 A5 JP2021520232 A5 JP 2021520232A5 JP 2021504131 A JP2021504131 A JP 2021504131A JP 2021504131 A JP2021504131 A JP 2021504131A JP 2021520232 A5 JP2021520232 A5 JP 2021520232A5
Authority
JP
Japan
Prior art keywords
sequence encoding
sequence
cells
item
isolated
Prior art date
Application number
JP2021504131A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021520232A (ja
JPWO2019195727A5 (https=
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/026062 external-priority patent/WO2019195727A1/en
Publication of JP2021520232A publication Critical patent/JP2021520232A/ja
Publication of JPWO2019195727A5 publication Critical patent/JPWO2019195727A5/ja
Publication of JP2021520232A5 publication Critical patent/JP2021520232A5/ja
Withdrawn legal-status Critical Current

Links

JP2021504131A 2018-04-05 2019-04-05 黄斑ジストロフィーを処置するための組成物及び方法 Withdrawn JP2021520232A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862653131P 2018-04-05 2018-04-05
US62/653,131 2018-04-05
PCT/US2019/026062 WO2019195727A1 (en) 2018-04-05 2019-04-05 Compositions and methods for treating macular dystrophy

Publications (3)

Publication Number Publication Date
JP2021520232A JP2021520232A (ja) 2021-08-19
JPWO2019195727A5 JPWO2019195727A5 (https=) 2022-04-12
JP2021520232A5 true JP2021520232A5 (https=) 2022-04-12

Family

ID=66223872

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021504131A Withdrawn JP2021520232A (ja) 2018-04-05 2019-04-05 黄斑ジストロフィーを処置するための組成物及び方法

Country Status (21)

Country Link
US (2) US20190307900A1 (https=)
EP (1) EP3775233A1 (https=)
JP (1) JP2021520232A (https=)
KR (1) KR20210005040A (https=)
CN (1) CN113056561A (https=)
AU (1) AU2019247864A1 (https=)
BR (1) BR112020020204A2 (https=)
CA (1) CA3096088A1 (https=)
CL (1) CL2020002561A1 (https=)
CO (1) CO2020013690A2 (https=)
EA (1) EA202092069A1 (https=)
IL (1) IL277779A (https=)
JO (1) JOP20200253A1 (https=)
MA (1) MA52199A (https=)
MX (1) MX2020010477A (https=)
PE (1) PE20210918A1 (https=)
PH (1) PH12020551641A1 (https=)
RU (1) RU2020132890A (https=)
SG (1) SG11202009759SA (https=)
TW (1) TW202003052A (https=)
WO (1) WO2019195727A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3870148A4 (en) * 2018-10-25 2022-11-09 Takeda Pharmaceutical Company Limited AAV TRIPLE PLASMID SYSTEM
US20220089670A1 (en) * 2018-12-28 2022-03-24 University Of Rochester Gene Therapy for BEST1 Dominant Mutations
US20230112568A1 (en) * 2020-02-28 2023-04-13 The Trustees Of The University Of Pennsylvania Treating autosomal recessive bestrophinopathies and methods for evaluating same
CN111849998A (zh) * 2020-07-29 2020-10-30 武汉纽福斯生物科技有限公司 编码人卵黄状黄斑病蛋白1的核酸分子及其应用
EP4590841A2 (en) * 2022-09-20 2025-07-30 Aldevron, LLC Best1 vectors and uses thereof

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2307551T3 (pl) * 2008-06-18 2017-07-31 Oxford Biomedica (Uk) Limited Oczyszczanie wektorów retrowirusowych
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
EP3071592B1 (en) * 2013-11-20 2021-01-06 Fondazione Telethon Artificial dna-binding proteins and uses thereof
AU2014359136B2 (en) * 2013-12-06 2020-06-25 Centre Hospitalier Universitaire De Montpellier Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject
GB201322798D0 (en) * 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
RS63416B1 (sr) * 2015-03-03 2022-08-31 Fond Telethon Sistem višestrukih vektora i njegove primene
WO2017083722A1 (en) * 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy

Similar Documents

Publication Publication Date Title
JP2021520232A5 (https=)
US20210275614A1 (en) Aav triple-plasmid system
US8129510B2 (en) Minigene expression cassette
IL278213B1 (en) A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd)
US20040142416A1 (en) Treatment for phenylketonuria
JP7541027B2 (ja) 筋肉発現のためのハイブリッドプロモーター
WO2016100575A1 (en) Gene therapy for juvenile batten disease
JP2024099770A5 (https=)
JP2021533805A5 (https=)
JPWO2020106916A5 (https=)
JPWO2019152609A5 (https=)
WO2020142714A1 (en) Aav expression cassette and aav vectors comprising the same
RU2020132890A (ru) Композиции и способы лечения макулярной дистрофии
JP2021534766A5 (https=)
JPWO2020243651A5 (https=)
CN113891712A (zh) 用于晚期婴儿神经元蜡样脂褐质沉积症2型的aav载体治疗方法
JPWO2020041773A5 (https=)
JPWO2019195727A5 (https=)
JPWO2020186207A5 (https=)
JPWO2019204514A5 (https=)
CN120457199A (zh) 截短的atp7b及其用途
JP2025534666A (ja) 操作された核酸調節エレメントならびにその使用方法
JPWO2021076911A5 (https=)
KR102874560B1 (ko) 아데노연관바이러스 변이체
US20250025574A1 (en) Engineered nucleic acid regulatory elements and methods and uses thereof