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

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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
GBGB1610448.1A GB201610448D0 (en) 2016-06-15 2016-06-15 Adeno-associated viral vector system
GB1610448.1 2016-06-15
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 (https=) 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 (https=)
EP (1) EP3472328A1 (https=)
JP (1) JP2019523648A (https=)
KR (1) KR20190020745A (https=)
CN (1) CN109642242A (https=)
AU (1) AU2017286623A1 (https=)
BR (1) BR112018075855A2 (https=)
CA (1) CA3025445A1 (https=)
IL (1) IL263523A (https=)
MX (1) MX2018015629A (https=)
RU (1) RU2765826C2 (https=)
SG (1) SG11201811244SA (https=)
WO (1) WO2017216560A1 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2014255665B2 (en) 2013-04-18 2018-08-02 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
WO2019195726A1 (en) * 2018-04-05 2019-10-10 Oxford University Innovation Limited Compositions and methods for the treatment of stargardt disease
IL282362B2 (en) * 2018-10-15 2025-10-01 Fond Telethon Ets Intein proteins and their uses
GB201817469D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy for retinal disease
WO2020093018A1 (en) 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS
US20220267802A1 (en) * 2019-07-15 2022-08-25 President And Fellows Of Harvard College Methods and compositions for gene delivery
KR20220130093A (ko) 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
CN115666658A (zh) 2020-04-01 2023-01-31 佛罗里达州大学研究基金会 具有提高的治疗ush1b的安全性的双aav-myo7a载体
WO2022036318A1 (en) * 2020-08-14 2022-02-17 Case Western Reserve University Plasmid vectors and nanoparticles for treating ocular disorders
US20240425877A1 (en) * 2021-05-07 2024-12-26 Ucl Business Ltd Abca4 genome editing
WO2023160454A1 (zh) * 2022-02-25 2023-08-31 北京中因科技有限公司 表达盒组合及其应用
EP4728080A1 (en) * 2023-06-16 2026-04-22 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
EP2311966A3 (en) * 2005-10-20 2012-09-05 Amsterdam Molecular Therapeutics (AMT) B.V. Improved AAV vectors produced in insect cells
US20090214478A1 (en) * 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
RU2555346C2 (ru) * 2009-08-07 2015-07-10 Трансген Са Композиция для лечения инфекции вирусом гепатита в
WO2013075008A1 (en) * 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
AU2014255665B2 (en) * 2013-04-18 2018-08-02 Fondazione Telethon Effective delivery of large genes by dual AAV vectors

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