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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English (en)
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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    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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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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  • Gastroenterology & Hepatology (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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
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 (1)

Publication Number Publication Date
CN109642242A true CN109642242A (zh) 2019-04-16

Family

ID=59101505

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Application Number Title Priority Date Filing Date
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

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=)

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 (13)

* Cited by examiner, † Cited by third party
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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
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

Citations (3)

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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

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

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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CA3025445A1 (en) 2017-12-21
US20190309326A1 (en) 2019-10-10
KR20190020745A (ko) 2019-03-04
WO2017216560A1 (en) 2017-12-21
MX2018015629A (es) 2019-09-26
RU2019100525A3 (https=) 2020-07-15
RU2765826C2 (ru) 2022-02-03
JP2019523648A (ja) 2019-08-29
EP3472328A1 (en) 2019-04-24
IL263523A (en) 2019-01-31
BR112018075855A2 (pt) 2019-04-02
SG11201811244SA (en) 2019-01-30
RU2019100525A (ru) 2020-07-15
AU2017286623A1 (en) 2018-12-20

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