JP2019523648A5 - - Google Patents

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Publication number
JP2019523648A5
JP2019523648A5 JP2018565324A JP2018565324A JP2019523648A5 JP 2019523648 A5 JP2019523648 A5 JP 2019523648A5 JP 2018565324 A JP2018565324 A JP 2018565324A JP 2018565324 A JP2018565324 A JP 2018565324A JP 2019523648 A5 JP2019523648 A5 JP 2019523648A5
Authority
JP
Japan
Prior art keywords
nucleic acid
acid sequence
aav vector
seq
nucleotides
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
JP2018565324A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019523648A (ja
Filing date
Publication date
Priority claimed from GBGB1610448.1A external-priority patent/GB201610448D0/en
Priority claimed from GBGB1707261.2A external-priority patent/GB201707261D0/en
Application filed filed Critical
Priority claimed from PCT/GB2017/051741 external-priority patent/WO2017216560A1/en
Publication of JP2019523648A publication Critical patent/JP2019523648A/ja
Publication of JP2019523648A5 publication Critical patent/JP2019523648A5/ja
Pending legal-status Critical Current

Links

JP2018565324A 2016-06-15 2017-06-14 Abc4aを発現させるためのデュアル重複アデノ随伴ウイルスベクターシステム Pending JP2019523648A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GB1610448.1 2016-06-15
GBGB1610448.1A GB201610448D0 (en) 2016-06-15 2016-06-15 Adeno-associated viral vector system
GB1707261.2 2017-05-05
GBGB1707261.2A GB201707261D0 (en) 2017-05-05 2017-05-05 Adeno-associated viral vector system
PCT/GB2017/051741 WO2017216560A1 (en) 2016-06-15 2017-06-14 Dual overlapping adeno-associated viral vector system for expressing abc4a

Publications (2)

Publication Number Publication Date
JP2019523648A JP2019523648A (ja) 2019-08-29
JP2019523648A5 true JP2019523648A5 (enExample) 2020-07-27

Family

ID=59101505

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2018565324A Pending JP2019523648A (ja) 2016-06-15 2017-06-14 Abc4aを発現させるためのデュアル重複アデノ随伴ウイルスベクターシステム

Country Status (13)

Country Link
US (1) US20190309326A1 (enExample)
EP (1) EP3472328A1 (enExample)
JP (1) JP2019523648A (enExample)
KR (1) KR20190020745A (enExample)
CN (1) CN109642242A (enExample)
AU (1) AU2017286623A1 (enExample)
BR (1) BR112018075855A2 (enExample)
CA (1) CA3025445A1 (enExample)
IL (1) IL263523A (enExample)
MX (1) MX2018015629A (enExample)
RU (1) RU2765826C2 (enExample)
SG (1) SG11201811244SA (enExample)
WO (1) WO2017216560A1 (enExample)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2986635T3 (en) 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
EP3635100A4 (en) 2017-05-05 2021-03-24 University of Florida Research Foundation, Incorporated COMPOSITIONS AND METHOD OF EXPRESSION OF OTOFERLIN
US20210147870A1 (en) * 2018-04-05 2021-05-20 Nightstarx Limited Compositions and methods for the treatment of stargardt disease
US20210371878A1 (en) * 2018-10-15 2021-12-02 Fondazione Telethon Intein proteins and uses thereof
GB201817469D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy for retinal disease
US12188041B2 (en) 2018-11-01 2025-01-07 University Of Florida Research Foundation, Incorporated Codon optimized otoferlin AAV dual vector gene therapy
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
US20220267802A1 (en) * 2019-07-15 2022-08-25 President And Fellows Of Harvard College Methods and compositions for gene delivery
CA3174500A1 (en) 2020-04-01 2021-10-07 University Of Florida Research Foundation, Incorporated Dual aav-myo7a vectors with improved safety for the treatment of ush1b
EP4196140A4 (en) * 2020-08-14 2024-11-27 Case Western Reserve University PLASMIDIC VECTORS AND NANOPARTICLES FOR TREATING EYE DISORDERS
EP4334447A1 (en) * 2021-05-07 2024-03-13 UCL Business Ltd Abca4 genome editing
CN118742649A (zh) * 2022-02-25 2024-10-01 北京中因科技有限公司 表达盒组合及其应用
WO2024257061A1 (en) * 2023-06-16 2024-12-19 Arkasubhra Ghosh A hybrid dual aav vector system with splice enhancer elements for expression of large genes

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3272872T (pt) * 2005-10-20 2020-06-26 Uniqure Ip Bv Vetores aav melhorados produzidos em células de insetos
US20090214478A1 (en) * 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
WO2011015656A2 (en) * 2009-08-07 2011-02-10 Transgene Sa Composition for treating hbv infection
WO2013075008A1 (en) * 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
DK2986635T3 (en) * 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS

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