JP2022523050A5 - - Google Patents

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Publication number
JP2022523050A5
JP2022523050A5 JP2021543230A JP2021543230A JP2022523050A5 JP 2022523050 A5 JP2022523050 A5 JP 2022523050A5 JP 2021543230 A JP2021543230 A JP 2021543230A JP 2021543230 A JP2021543230 A JP 2021543230A JP 2022523050 A5 JP2022523050 A5 JP 2022523050A5
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JP
Japan
Prior art keywords
acid sequence
nucleic acid
raav
mammal
therapeutic agent
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JP2021543230A
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English (en)
Japanese (ja)
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JP7624727B2 (ja
JP2022523050A (ja
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Priority claimed from PCT/US2020/014838 external-priority patent/WO2020154535A1/en
Publication of JP2022523050A publication Critical patent/JP2022523050A/ja
Publication of JP2022523050A5 publication Critical patent/JP2022523050A5/ja
Application granted granted Critical
Publication of JP7624727B2 publication Critical patent/JP7624727B2/ja
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JP2021543230A 2019-01-23 2020-01-23 合理的設計により増強された新規なaavウイルスによる網膜における高効率の形質導入および側方への広がり Active JP7624727B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962795695P 2019-01-23 2019-01-23
US62/795,695 2019-01-23
PCT/US2020/014838 WO2020154535A1 (en) 2019-01-23 2020-01-23 Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design

Publications (3)

Publication Number Publication Date
JP2022523050A JP2022523050A (ja) 2022-04-21
JP2022523050A5 true JP2022523050A5 (https=) 2023-02-06
JP7624727B2 JP7624727B2 (ja) 2025-01-31

Family

ID=71737005

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021543230A Active JP7624727B2 (ja) 2019-01-23 2020-01-23 合理的設計により増強された新規なaavウイルスによる網膜における高効率の形質導入および側方への広がり

Country Status (7)

Country Link
US (1) US20220133909A1 (https=)
EP (1) EP3914229A4 (https=)
JP (1) JP7624727B2 (https=)
CN (1) CN113347962B (https=)
AU (1) AU2020212026B2 (https=)
CA (1) CA3125294A1 (https=)
WO (1) WO2020154535A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024223696A1 (en) * 2023-04-24 2024-10-31 Universiteit Gent Compounds to treat inherited retinal disease
CN121046460A (zh) * 2024-05-31 2025-12-02 上海朗昇生物科技有限公司 用于治疗Prom1相关视网膜疾病的新型治疗药物

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0977880A2 (en) * 1997-04-21 2000-02-09 University Of Florida Materials and methods for treatment of retinal diseases
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
ES2525067T3 (es) * 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
SG10201800541SA (en) 2010-04-23 2018-03-28 Univ Florida Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-1 (lca1)
EP3113787B1 (en) * 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
US10533187B2 (en) 2015-03-18 2020-01-14 University Of Florida Research Foundation, Incorporated Methods and compositions for restoration of cone function in BCM
JP6805174B2 (ja) 2015-05-12 2020-12-23 アメリカ合衆国 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むaav分離株及び融合タンパク質
US20170348387A1 (en) 2016-02-29 2017-12-07 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for nphp5 lca-ciliopathy
IL305149A (en) * 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF

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