KR102917057B1 - 뇌에 지향성을 가지는 aav 돌연변이체 - Google Patents

뇌에 지향성을 가지는 aav 돌연변이체

Info

Publication number
KR102917057B1
KR102917057B1 KR1020217035927A KR20217035927A KR102917057B1 KR 102917057 B1 KR102917057 B1 KR 102917057B1 KR 1020217035927 A KR1020217035927 A KR 1020217035927A KR 20217035927 A KR20217035927 A KR 20217035927A KR 102917057 B1 KR102917057 B1 KR 102917057B1
Authority
KR
South Korea
Prior art keywords
capsid protein
aav
gly
peptide
aav capsid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
KR1020217035927A
Other languages
English (en)
Korean (ko)
Other versions
KR20220004072A (ko
Inventor
토시카즈 니시에
후유코 다카시마
타츠지 에노키
준이치 미네노
요시노리 다나카
Original Assignee
다카라 바이오 가부시키가이샤
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 다카라 바이오 가부시키가이샤 filed Critical 다카라 바이오 가부시키가이샤
Publication of KR20220004072A publication Critical patent/KR20220004072A/ko
Application granted granted Critical
Publication of KR102917057B1 publication Critical patent/KR102917057B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01DNA viruses
    • C07K14/015Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C40COMBINATORIAL TECHNOLOGY
    • C40BCOMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B50/00Methods of creating libraries, e.g. combinatorial synthesis
    • C40B50/06Biochemical methods, e.g. using enzymes or whole viable microorganisms
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10041Use of virus, viral particle or viral elements as a vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors

Landscapes

  • 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)
KR1020217035927A 2019-04-24 2020-04-23 뇌에 지향성을 가지는 aav 돌연변이체 Active KR102917057B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JPJP-P-2019-082417 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

Publications (2)

Publication Number Publication Date
KR20220004072A KR20220004072A (ko) 2022-01-11
KR102917057B1 true KR102917057B1 (ko) 2026-01-23

Family

ID=72942142

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217035927A Active KR102917057B1 (ko) 2019-04-24 2020-04-23 뇌에 지향성을 가지는 aav 돌연변이체

Country Status (10)

Country Link
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 (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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 (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US9938541B2 (en) 2012-12-25 2018-04-10 Takara Bio Inc. AAV variant
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 (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP3960755A1 (en) 2022-03-02
EP3960755A4 (en) 2023-11-22
US20220162637A1 (en) 2022-05-26
WO2020218419A1 (ja) 2020-10-29
CN113748122B (zh) 2024-11-22
AU2020263757B2 (en) 2025-08-28
CN113748122A (zh) 2021-12-03

Similar Documents

Publication Publication Date Title
JP6363958B2 (ja) Aav変異体
KR102917057B1 (ko) 뇌에 지향성을 가지는 aav 돌연변이체
JP2022107646A (ja) アデノ随伴ウイルス(aav)キャプシドタンパク質の変異体
CN111850045B (zh) 靶向心脏血管内皮的腺相关病毒载体及其应用
EP4159863A1 (en) Codon-optimized nucleic acid encoding smn1 protein
CN111718418A (zh) 一种增强基因编辑的融合蛋白及其应用
RU2809389C2 (ru) Мутант aav, обладающий способностью нацеливаться на головной мозг
AU2019277217B2 (en) AAV polynucleotides, polypeptides and virions
HK40062349A (en) Aav mutant having brain-targeting property
HK40062349B (zh) 具有脑靶向特性的aav突变体
HK40069854A (en) Aav mutant having brain-targeting property
WO2025023319A1 (ja) 脳指向性aav変異体
EP4410987A1 (en) An aav9 capsid variant for targeted gene transfer
HK1215277B (zh) Aav变体
WO2025057943A1 (ja) 内耳に感染性を有するaav変異体
OA21075A (en) Codon-optimized nucleic acid that encodes SMN1 protein, and use thereof
WO2025051747A1 (en) Viral vector for fibroblast transfection
CN117886897A (zh) 一种腺相关病毒变体及其应用
WO2024186275A1 (en) Novel compositions of adeno-associated viruses engineered for enhanced tissue transduction and specificity
CN117886898A (zh) 腺相关病毒变体及其应用
HK40014535A (en) Mutant of adeno-associated virus (aav) capsid protein

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

A201 Request for examination
PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

D22 Grant of ip right intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D22-EXM-PE0701 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0701 Decision of registration

St.27 status event code: A-1-2-D10-D22-exm-PE0701

F11 Ip right granted following substantive examination

Free format text: ST27 STATUS EVENT CODE: A-2-4-F10-F11-EXM-PR0701 (AS PROVIDED BY THE NATIONAL OFFICE)

PR0701 Registration of establishment

St.27 status event code: A-2-4-F10-F11-exm-PR0701

PR1002 Payment of registration fee

St.27 status event code: A-2-2-U10-U12-oth-PR1002

Fee payment year number: 1

U12 Designation fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U12-OTH-PR1002 (AS PROVIDED BY THE NATIONAL OFFICE)

Year of fee payment: 1

PG1601 Publication of registration

St.27 status event code: A-4-4-Q10-Q13-nap-PG1601

Q13 Ip right document published

Free format text: ST27 STATUS EVENT CODE: A-4-4-Q10-Q13-NAP-PG1601 (AS PROVIDED BY THE NATIONAL OFFICE)