CN109642242A - 用于表达abc4a的双重重叠腺伴随病毒载体系统 - Google Patents

用于表达abc4a的双重重叠腺伴随病毒载体系统 Download PDF

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Publication number
CN109642242A
CN109642242A CN201780037639.9A CN201780037639A CN109642242A CN 109642242 A CN109642242 A CN 109642242A CN 201780037639 A CN201780037639 A CN 201780037639A CN 109642242 A CN109642242 A CN 109642242A
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sequence
nucleic acid
nucleotide
aav
seq
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Pending
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CN201780037639.9A
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Chinese (zh)
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R.麦克拉伦
M.麦克莱门茨
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Oxford University Innovation Ltd
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Oxford University Innovation Ltd
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Priority claimed from GBGB1610448.1A external-priority patent/GB201610448D0/en
Priority claimed from GBGB1707261.2A external-priority patent/GB201707261D0/en
Application filed by Oxford University Innovation Ltd filed Critical Oxford University Innovation Ltd
Publication of CN109642242A publication Critical patent/CN109642242A/zh
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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  • 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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
CN201780037639.9A 2016-06-15 2017-06-14 用于表达abc4a的双重重叠腺伴随病毒载体系统 Pending CN109642242A (zh)

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
CN109642242A true CN109642242A (zh) 2019-04-16

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CN201780037639.9A Pending CN109642242A (zh) 2016-06-15 2017-06-14 用于表达abc4a的双重重叠腺伴随病毒载体系统

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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)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Families Citing this family (12)

* 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
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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101287837A (zh) * 2005-10-20 2008-10-15 阿姆斯特丹分子治疗股份有限公司 昆虫细胞中生产的改进的aav载体
WO2009103562A1 (en) * 2008-02-21 2009-08-27 Fondazione Telethon Method of treating ocular diseases by gene therapy
WO2014170480A1 (en) * 2013-04-18 2014-10-23 Fondazione Telethon Effective delivery of large genes by dual aav vectors

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101287837A (zh) * 2005-10-20 2008-10-15 阿姆斯特丹分子治疗股份有限公司 昆虫细胞中生产的改进的aav载体
WO2009103562A1 (en) * 2008-02-21 2009-08-27 Fondazione Telethon Method of treating ocular diseases by gene therapy
WO2014170480A1 (en) * 2013-04-18 2014-10-23 Fondazione Telethon Effective delivery of large genes by dual aav vectors
CN105408352A (zh) * 2013-04-18 2016-03-16 泰莱托恩基金会 通过双重aav载体有效递送大基因

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

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AU2017286623A1 (en) 2018-12-20
RU2019100525A3 (enExample) 2020-07-15
KR20190020745A (ko) 2019-03-04
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
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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