CN113748122B - 具有脑靶向特性的aav突变体 - Google Patents

具有脑靶向特性的aav突变体 Download PDF

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Publication number
CN113748122B
CN113748122B CN202080030827.0A CN202080030827A CN113748122B CN 113748122 B CN113748122 B CN 113748122B CN 202080030827 A CN202080030827 A CN 202080030827A CN 113748122 B CN113748122 B CN 113748122B
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aav
seq
capsid protein
amino acid
peptide
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CN113748122A (zh
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西江敏和
高岛扶有子
榎龙嗣
峰野纯一
田中佳典
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Takara Bio Inc
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Takara Bio Inc
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B50/00—Methods of creating libraries, e.g. combinatorial synthesis
    • C40B50/06—Biochemical methods, e.g. using enzymes or whole viable microorganisms
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

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  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202080030827.0A 2019-04-24 2020-04-23 具有脑靶向特性的aav突变体 Active CN113748122B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2019-082417 2019-04-24
JP2019082417 2019-04-24
PCT/JP2020/017495 WO2020218419A1 (ja) 2019-04-24 2020-04-23 脳に指向性を有するａａｖ変異体

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CN113748122A CN113748122A (zh) 2021-12-03
CN113748122B true CN113748122B (zh) 2024-11-22

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US (1) US12421526B2 (https=)
EP (1) EP3960755A4 (https=)
JP (1) JP7524166B2 (https=)
KR (1) KR102917057B1 (https=)
CN (1) CN113748122B (https=)
AU (1) AU2020263757B2 (https=)
BR (1) BR112021017603A2 (https=)
CA (1) CA3132447A1 (https=)
IL (1) IL286212A (https=)
WO (1) WO2020218419A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3575398A4 (en) * 2017-01-30 2020-11-11 Nippon Medical School Foundation ADENO-ASSOCIATED VIRUS (AAV) CAPSIDE PROTEIN MUTANT
CN115960177B (zh) * 2022-10-09 2023-07-07 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN116789739B (zh) * 2022-12-08 2024-01-26 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN116284262B (zh) * 2023-02-13 2026-03-17 和元生物技术（上海）股份有限公司 高效感染ht-22细胞的腺相关病毒突变体
CN116693633B (zh) * 2023-02-21 2023-12-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN121569040A (zh) 2023-07-27 2026-02-24 宝生物工程株式会社 脑趋向性aav突变体
CN121816425A (zh) * 2023-09-11 2026-04-07 学校法人顺天堂 能够感染内耳的aav变体

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104937100A (zh) * 2012-12-25 2015-09-23 宝生物工程株式会社 Aav变体
JP2017513486A (ja) * 2014-04-17 2017-06-01 ウニヴェルズィテーツクリニクム ハンブルク−エッペンドルフＵｎｉｖｅｒｓｉｔａｅｔｓｋｌｉｎｉｋｕｍ Ｈａｍｂｕｒｇ−Ｅｐｐｅｎｄｏｒｆ 脳および脊髄に標的化遺伝子移入するためのウイルスベクター

Family Cites Families (10)

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DE19827457C1 (de) * 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
EP2176283B1 (en) 2007-07-14 2016-11-02 University of Iowa Research Foundation Methods and compositions for treating brain diseases
WO2010114143A1 (ja) * 2009-03-30 2010-10-07 国立大学法人岡山大学 特異的プロモーターの活性を上昇させるシステム及び該システムを保持したベクター
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
WO2013014764A1 (ja) 2011-07-27 2013-01-31 三菱電機株式会社 通信システム、通信装置および通信方法
HK1222673A1 (zh) 2013-07-12 2017-07-07 The Children's Hospital Of Philadelphia Aav载体和用於抗aav(腺相关病毒)中和抗体的检测
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
CA2942289C (en) * 2014-03-10 2024-05-21 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
US11364308B2 (en) * 2016-05-13 2022-06-21 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
EP3890017B1 (en) 2018-12-04 2025-08-13 Ningbo Sunny Opotech Co., Ltd. Photosensitive assembly, photographic module, manufacturing method for photographic assembly, and electronic device

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104937100A (zh) * 2012-12-25 2015-09-23 宝生物工程株式会社 Aav变体
JP2017513486A (ja) * 2014-04-17 2017-06-01 ウニヴェルズィテーツクリニクム ハンブルク−エッペンドルフＵｎｉｖｅｒｓｉｔａｅｔｓｋｌｉｎｉｋｕｍ Ｈａｍｂｕｒｇ−Ｅｐｐｅｎｄｏｒｆ 脳および脊髄に標的化遺伝子移入するためのウイルスベクター

Also Published As

Publication number Publication date
JP7524166B2 (ja) 2024-07-29
JPWO2020218419A1 (https=) 2020-10-29
BR112021017603A2 (pt) 2021-11-16
IL286212A (en) 2021-10-31
CA3132447A1 (en) 2020-10-29
AU2020263757A1 (en) 2021-10-14
KR20220004072A (ko) 2022-01-11
US12421526B2 (en) 2025-09-23
KR102917057B1 (ko) 2026-01-23
EP3960755A1 (en) 2022-03-02
EP3960755A4 (en) 2023-11-22
US20220162637A1 (en) 2022-05-26
WO2020218419A1 (ja) 2020-10-29
AU2020263757B2 (en) 2025-08-28
CN113748122A (zh) 2021-12-03

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