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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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  • Toxicology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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
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

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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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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 (14)

* 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
US12359221B2 (en) 2017-05-05 2025-07-15 The Regents Of The University Of California Compositions and methods for expressing otoferlin
US20210147870A1 (en) * 2018-04-05 2021-05-20 Nightstarx Limited Compositions and methods for the treatment of stargardt disease
CA3116606A1 (en) * 2018-10-15 2020-04-23 Fondazione Telethon Intein proteins and uses thereof
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
WO2021015997A1 (en) * 2019-07-15 2021-01-28 President And Fellows Of Harvard College Methods and compositions for gene delivery
TWI896570B (zh) 2019-10-30 2025-09-11 美商迪賽博治療公司 使用耳畸蛋白雙載體系統治療感覺神經性聽力損失之組合物及方法
IL295741A (en) * 2020-04-01 2022-10-01 Univ Florida Dual aav-myo7a vectors with improved safety for the treatment of ush1b
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
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

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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
CA2770075C (en) * 2009-08-07 2021-08-24 Perrine Martin 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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Publication number Publication date
US20190309326A1 (en) 2019-10-10
IL263523A (en) 2019-01-31
WO2017216560A1 (en) 2017-12-21
BR112018075855A2 (pt) 2019-04-02
RU2019100525A3 (enExample) 2020-07-15
CA3025445A1 (en) 2017-12-21
CN109642242A (zh) 2019-04-16
RU2765826C2 (ru) 2022-02-03
JP2019523648A (ja) 2019-08-29
RU2019100525A (ru) 2020-07-15
AU2017286623A1 (en) 2018-12-20
EP3472328A1 (en) 2019-04-24
MX2018015629A (es) 2019-09-26
SG11201811244SA (en) 2019-01-30

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