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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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • A—HUMAN NECESSITIES
    • 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
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a 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
    • C12N2750/14141—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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  • Ophthalmology & Optometry (AREA)
  • Biochemistry (AREA)
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  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • 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)
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
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 (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 (13)

* 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
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

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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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

Patent Citations (2)

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Publication number Priority date Publication date Assignee Title
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
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
KR20190020745A (ko) 2019-03-04
CN109642242A (zh) 2019-04-16
RU2765826C2 (ru) 2022-02-03
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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