KR20190020745A - Abc4a를 발현시키기 위한 이중 중첩 아데노-관련 바이러스 벡터 시스템 - Google Patents

Abc4a를 발현시키기 위한 이중 중첩 아데노-관련 바이러스 벡터 시스템 Download PDF

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KR20190020745A
KR20190020745A KR1020197001281A KR20197001281A KR20190020745A KR 20190020745 A KR20190020745 A KR 20190020745A KR 1020197001281 A KR1020197001281 A KR 1020197001281A KR 20197001281 A KR20197001281 A KR 20197001281A KR 20190020745 A KR20190020745 A KR 20190020745A
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nucleic acid
acid sequence
sequence
abca4
aav vector
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KR1020197001281A
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Korean (ko)
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로버트 맥클라렌
미셸 맥클레멘츠
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옥스포드 유니버시티 이노베이션 리미티드
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Priority claimed from GBGB1610448.1A external-priority patent/GB201610448D0/en
Priority claimed from GBGB1707261.2A external-priority patent/GB201707261D0/en
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KR1020197001281A 2016-06-15 2017-06-14 Abc4a를 발현시키기 위한 이중 중첩 아데노-관련 바이러스 벡터 시스템 Ceased KR20190020745A (ko)

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
GBGB1707261.2A GB201707261D0 (en) 2017-05-05 2017-05-05 Adeno-associated viral vector system
GB1707261.2 2017-05-05
PCT/GB2017/051741 WO2017216560A1 (en) 2016-06-15 2017-06-14 Dual overlapping adeno-associated viral vector system for expressing abc4a

Publications (1)

Publication Number Publication Date
KR20190020745A true KR20190020745A (ko) 2019-03-04

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KR1020197001281A Ceased KR20190020745A (ko) 2016-06-15 2017-06-14 Abc4a를 발현시키기 위한 이중 중첩 아데노-관련 바이러스 벡터 시스템

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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
US10494645B2 (en) 2013-04-18 2019-12-03 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
CN110892062A (zh) 2017-05-05 2020-03-17 佛罗里达大学研究基金会 表达耳畸蛋白的组合物和方法
US20210147870A1 (en) * 2018-04-05 2021-05-20 Nightstarx Limited Compositions and methods for the treatment of stargardt disease
IL320368A (en) * 2018-10-15 2025-06-01 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
WO2020163743A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
WO2021015997A1 (en) * 2019-07-15 2021-01-28 President And Fellows Of Harvard College Methods and compositions for gene delivery
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
US20230338580A1 (en) * 2020-08-14 2023-10-26 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
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

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007046703A2 (en) * 2005-10-20 2007-04-26 Amsterdam Molecular Therapeutics 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
NZ598000A (en) * 2009-08-07 2013-10-25 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
US10494645B2 (en) * 2013-04-18 2019-12-03 Fondazione Telethon Effective delivery of large genes by dual AAV vectors

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AU2017286623A1 (en) 2018-12-20
RU2019100525A3 (enExample) 2020-07-15
RU2765826C2 (ru) 2022-02-03
CA3025445A1 (en) 2017-12-21
MX2018015629A (es) 2019-09-26
IL263523A (en) 2019-01-31
SG11201811244SA (en) 2019-01-30
EP3472328A1 (en) 2019-04-24
CN109642242A (zh) 2019-04-16
RU2019100525A (ru) 2020-07-15
BR112018075855A2 (pt) 2019-04-02
WO2017216560A1 (en) 2017-12-21
JP2019523648A (ja) 2019-08-29
US20190309326A1 (en) 2019-10-10

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