JP2019523648A - 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 - Google Patents

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 Download PDF

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JP2019523648A
JP2019523648A JP2018565324A JP2018565324A JP2019523648A JP 2019523648 A JP2019523648 A JP 2019523648A JP 2018565324 A JP2018565324 A JP 2018565324A JP 2018565324 A JP2018565324 A JP 2018565324A JP 2019523648 A JP2019523648 A JP 2019523648A
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nucleic acid
acid sequence
abca4
sequence
seq
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JP2019523648A5 (enExample
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マクラーレン，ロバート
マックレメンツ，ミシェル
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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
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Publication of JP2019523648A publication Critical patent/JP2019523648A/ja
Publication of JP2019523648A5 publication Critical patent/JP2019523648A5/ja
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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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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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (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)
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
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 (2)

Publication Number Publication Date
JP2019523648A true JP2019523648A (ja) 2019-08-29
JP2019523648A5 JP2019523648A5 (enExample) 2020-07-27

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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

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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)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023537631A (ja) * 2020-08-14 2023-09-04 ケース ウエスタン リザーブ ユニバーシティ 眼障害を処置するためのプラスミドベクターおよびナノ粒子

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
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

Citations (2)

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WO2013075008A1 (en) * 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
JP2016516424A (ja) * 2013-04-18 2016-06-09 フォンダッツィオーネ・テレソン デュアルａａｖベクターによる大型遺伝子の効果的送達

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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

Patent Citations (2)

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WO2013075008A1 (en) * 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
JP2016516424A (ja) * 2013-04-18 2016-06-09 フォンダッツィオーネ・テレソン デュアルａａｖベクターによる大型遺伝子の効果的送達

Non-Patent Citations (2)

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Title
TRAPANI, I. ET AL.: "Effective delivery of large genes to the retina by dual AAV vectors", EMBO MOL. MED., vol. Vol. 6(2), JPN6021026553, 2014, pages 194 - 211, ISSN: 0004550571 *
TRAPANI, I. ET AL.: "Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in th", HUM. MOL. GENET., vol. Vol. 24(23), JPN6021026555, 2015, pages 6811 - 6825, ISSN: 0004550570 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023537631A (ja) * 2020-08-14 2023-09-04 ケース ウエスタン リザーブ ユニバーシティ 眼障害を処置するためのプラスミドベクターおよびナノ粒子

Also Published As

Publication number Publication date
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
CN109642242A (zh) 2019-04-16
RU2019100525A (ru) 2020-07-15
BR112018075855A2 (pt) 2019-04-02
WO2017216560A1 (en) 2017-12-21
US20190309326A1 (en) 2019-10-10

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