JP7470057B2 - 組み換えａａｖベクター及びその使用方法 - Google Patents

組み換えａａｖベクター及びその使用方法 Download PDF

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JP7470057B2
JP7470057B2 JP2020569147A JP2020569147A JP7470057B2 JP 7470057 B2 JP7470057 B2 JP 7470057B2 JP 2020569147 A JP2020569147 A JP 2020569147A JP 2020569147 A JP2020569147 A JP 2020569147A JP 7470057 B2 JP7470057 B2 JP 7470057B2
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seq
sequence
vector
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recombinant
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JP2021526834A (ja
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セルジュ フィトゥシ
バレット カッツ
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0012—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7)
    • C12N9/0026—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on CH-NH groups of donors (1.5)
    • C12N9/0028—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on CH-NH groups of donors (1.5) with NAD or NADP as acceptor (1.5.1)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0012—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7)
    • C12N9/0036—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on NADH or NADPH (1.6)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0053—Oxidoreductases (1.) acting on a heme group of donors (1.9)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y106/00—Oxidoreductases acting on NADH or NADPH (1.6)
    • C12Y106/05—Oxidoreductases acting on NADH or NADPH (1.6) with a quinone or similar compound as acceptor (1.6.5)
    • C12Y106/05003—NADH dehydrogenase (ubiquinone) (1.6.5.3)
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    • C12Y109/00—Oxidoreductases acting on a heme group of donors (1.9)
    • C12Y109/03—Oxidoreductases acting on a heme group of donors (1.9) with oxygen as acceptor (1.9.3)
    • C12Y109/03001—Cytochrome-c oxidase (1.9.3.1)
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    • 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
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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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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
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  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP2020569147A 2018-06-11 2019-06-11 組み換えａａｖベクター及びその使用方法 Active JP7470057B2 (ja)

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Application Number Priority Date Filing Date Title
US201862683501P 2018-06-11 2018-06-11
US62/683,501 2018-06-11
PCT/US2019/036487 WO2019241206A1 (en) 2018-06-11 2019-06-11 Recombinant aav vectors and methods of using the same

Publications (4)

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JP2021526834A JP2021526834A (ja) 2021-10-11
JP2021526834A5 JP2021526834A5 (https=) 2022-06-15
JPWO2019241206A5 JPWO2019241206A5 (https=) 2022-06-15
JP7470057B2 true JP7470057B2 (ja) 2024-04-17

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US (1) US20210189424A1 (https=)
EP (1) EP3802840A1 (https=)
JP (1) JP7470057B2 (https=)
KR (1) KR102946006B1 (https=)
CN (1) CN112384625A (https=)
AU (1) AU2019286386B2 (https=)
CA (1) CA3103231A1 (https=)
WO (1) WO2019241206A1 (https=)

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KR20240014102A (ko) 2018-06-29 2024-01-31 우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법
CA3109432A1 (en) * 2018-08-20 2020-02-27 Wuhan Neurophth Biotechnology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
CN113025633B (zh) 2019-12-09 2024-08-27 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用
CN115710585B (zh) * 2021-08-23 2025-03-28 纽福斯(苏州)生物科技有限公司 编码nd4的核酸及其用途

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006117250A2 (en) 2005-05-03 2006-11-09 Institut National De La Sante Et De La Recherche Medicale (Inserm) Importation of mitochondrial protein by an enhanced allotopic approach

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US6218117B1 (en) * 1998-06-15 2001-04-17 Mitokor Compositions and methods for identifying agents that quantitatively alter detectable extramitochondrial DNA:mitochondrial DNA ratios
EP2121914B1 (en) * 2007-02-16 2014-08-20 John Guy Mitochondrial nucleic acid delivery systems
DE102010013829A1 (de) * 2010-03-26 2011-09-29 Carl Zeiss Microlmaging Gmbh Mikroskop und Verfahren zur Erfassung von Probenlicht
CN102634527B (zh) * 2012-04-11 2013-11-06 华中科技大学同济医学院附属同济医院 重组人nadh脱氢酶亚单位4基因及其表达载体构建方法
TWI775096B (zh) * 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
KR102244434B1 (ko) * 2014-08-11 2021-04-23 삼성전자주식회사 재조합 벡터 및 이를 이용한 목적 폴리펩타이드의 생산 방법
CN104450747B (zh) * 2014-09-23 2018-02-09 武汉纽福斯生物科技有限公司 用于治疗Leber遗传性视神经病变的重组腺相关病毒‑NADH脱氢酶亚单位4基因全长以及药剂

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006117250A2 (en) 2005-05-03 2006-11-09 Institut National De La Sante Et De La Recherche Medicale (Inserm) Importation of mitochondrial protein by an enhanced allotopic approach

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
EBioMedicine，2016年，Vol.10, pp.258-268
Molecular Therapy - Methods & Clinical Development，2015年，Vol.2, 15003 (15 pages)，<doi:10.1038/mtm.2015.3>
Scientific Reports，2016年，Vol.6, 21587 (10 pages)，<DOI: 10.1038/srep21587>

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JP2021526834A (ja) 2021-10-11
KR102946006B1 (ko) 2026-03-30
AU2019286386B2 (en) 2025-05-29
US20210189424A1 (en) 2021-06-24
CA3103231A1 (en) 2019-12-19
AU2019286386A1 (en) 2021-01-28
WO2019241206A1 (en) 2019-12-19
AU2019286386A8 (en) 2021-02-11
CN112384625A (zh) 2021-02-19
EP3802840A1 (en) 2021-04-14
KR20210123285A (ko) 2021-10-13

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