JPWO2020214809A5 - - Google Patents

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Publication number
JPWO2020214809A5
JPWO2020214809A5 JP2021562000A JP2021562000A JPWO2020214809A5 JP WO2020214809 A5 JPWO2020214809 A5 JP WO2020214809A5 JP 2021562000 A JP2021562000 A JP 2021562000A JP 2021562000 A JP2021562000 A JP 2021562000A JP WO2020214809 A5 JPWO2020214809 A5 JP WO2020214809A5
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JP
Japan
Prior art keywords
nucleic acid
isolated nucleic
vector
raav
promoter
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
JP2021562000A
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English (en)
Japanese (ja)
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JP2022529782A5 (https=
JP2022529782A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/028504 external-priority patent/WO2020214809A2/en
Publication of JP2022529782A publication Critical patent/JP2022529782A/ja
Publication of JPWO2020214809A5 publication Critical patent/JPWO2020214809A5/ja
Publication of JP2022529782A5 publication Critical patent/JP2022529782A5/ja
Ceased legal-status Critical Current

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JP2021562000A 2019-04-19 2020-04-16 シュタルガルト病（ａｂｃａ４）の遺伝子治療 Ceased JP2022529782A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962836493P 2019-04-19 2019-04-19
US62/836,493 2019-04-19
PCT/US2020/028504 WO2020214809A2 (en) 2019-04-19 2020-04-16 Gene therapies for stargardt disease (abca4)

Publications (3)

Publication Number Publication Date
JP2022529782A JP2022529782A (ja) 2022-06-24
JPWO2020214809A5 true JPWO2020214809A5 (https=) 2023-04-25
JP2022529782A5 JP2022529782A5 (https=) 2023-04-25

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ID=72838359

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021562000A Ceased JP2022529782A (ja) 2019-04-19 2020-04-16 シュタルガルト病（ａｂｃａ４）の遺伝子治療

Country Status (7)

Country Link
US (2) US12516097B2 (https=)
EP (1) EP3955973A4 (https=)
JP (1) JP2022529782A (https=)
CN (1) CN114126665B (https=)
AU (1) AU2020257392A1 (https=)
CA (1) CA3137135A1 (https=)
WO (1) WO2020214809A2 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3137135A1 (en) 2019-04-19 2020-10-22 University Of Massachusetts Gene therapies for stargardt disease (abca4)
GB2700048B (en) 2019-09-18 2026-03-04 Aldevron Llc synthetic dna vectors and method of use
CN116234917A (zh) 2020-07-27 2023-06-06 安杰瑞姆生物科学公司 Dna分子组合物及其制备方法和使用方法
KR20230161941A (ko) * 2021-01-22 2023-11-28 코넬 유니버시티 리포푸신 세포독성으로부터 보호하는 화합물의 식별을위한 조성물 및 방법
CA3211687A1 (en) * 2021-03-19 2022-09-22 Ozan ALKAN Non-viral dna vectors and uses thereof for expressing pfic therapeutics
EP4330409A4 (en) 2021-04-26 2025-04-09 University of Massachusetts GENE THERAPIES FOR STARGARDT'S DISEASE (ABCA4)
EP4334447A1 (en) * 2021-05-07 2024-03-13 UCL Business Ltd Abca4 genome editing
CN115074369B (zh) * 2022-06-24 2023-10-20 成都金唯科生物科技有限公司 表达全长abca4基因的腺相关病毒载体及应用

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
US20090214478A1 (en) 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
WO2009134418A2 (en) * 2008-04-30 2009-11-05 Fox Chase Cancer Center Assay for identifying agents that modulate epigenetic silencing, and agents identified thereby
KR102537394B1 (ko) * 2014-03-17 2023-05-30 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
EP3377116A4 (en) * 2015-11-19 2019-07-10 The Trustees of The University of Pennsylvania COMPOSITIONS AND METHODS FOR CORRECTING ECG-RELATED EYE DISEASE
WO2018109011A1 (en) 2016-12-13 2018-06-21 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of stargardt disease
AU2018223810B2 (en) 2017-02-27 2022-04-21 Case Western Reserve University Visual cycle modulators
CA3137135A1 (en) 2019-04-19 2020-10-22 University Of Massachusetts Gene therapies for stargardt disease (abca4)
EP4330409A4 (en) 2021-04-26 2025-04-09 University of Massachusetts GENE THERAPIES FOR STARGARDT'S DISEASE (ABCA4)

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