JP2022530457A - 遺伝子操作ａａｖ - Google Patents

遺伝子操作ａａｖ Download PDF

Info

Publication number
JP2022530457A
JP2022530457A JP2021563281A JP2021563281A JP2022530457A JP 2022530457 A JP2022530457 A JP 2022530457A JP 2021563281 A JP2021563281 A JP 2021563281A JP 2021563281 A JP2021563281 A JP 2021563281A JP 2022530457 A JP2022530457 A JP 2022530457A
Authority
JP
Japan
Prior art keywords
seq
amino acid
peptide
nucleic acid
capsid protein
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.)
Pending
Application number
JP2021563281A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022530457A5 (https=
JPWO2020219988A5 (https=
Inventor
カイル マクガヴァン，
デイビッド エス． オジャラ，
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sangamo Therapeutics Inc
Original Assignee
Sangamo Therapeutics Inc
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 Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of JP2022530457A publication Critical patent/JP2022530457A/ja
Publication of JP2022530457A5 publication Critical patent/JP2022530457A5/ja
Publication of JPWO2020219988A5 publication Critical patent/JPWO2020219988A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/502—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics for testing non-proliferative effects
    • 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
    • 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
    • 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
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/08—Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1058—Directional evolution of libraries, e.g. evolution of libraries is achieved by mutagenesis and screening or selection of mixed population of organisms
    • 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
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6897—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids involving reporter genes operably linked to promoters
    • C—CHEMISTRY; METALLURGY
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B20/00—Methods specially adapted for identifying library members
    • C40B20/04—Identifying library members by means of a tag, label, or other readable or detectable entity associated with the library members, e.g. decoding processes
    • C—CHEMISTRY; METALLURGY
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00—Libraries per se, e.g. arrays, mixtures
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2563/00—Nucleic acid detection characterized by the use of physical, structural and functional properties
    • C12Q2563/179—Nucleic acid detection characterized by the use of physical, structural and functional properties the label being a nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2563/00—Nucleic acid detection characterized by the use of physical, structural and functional properties
    • C12Q2563/185—Nucleic acid dedicated to use as a hidden marker/bar code, e.g. inclusion of nucleic acids to mark art objects or animals
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/005—Assays involving biological materials from specific organisms or of a specific nature from viruses
    • G01N2333/01—DNA viruses
    • G01N2333/075—Adenoviridae

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Analytical Chemistry (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Ecology (AREA)
  • Cell Biology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Food Science & Technology (AREA)
  • Toxicology (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
JP2021563281A 2019-04-26 2020-04-24 遺伝子操作ａａｖ Pending JP2022530457A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962839421P 2019-04-26 2019-04-26
US62/839,421 2019-04-26
US201962915386P 2019-10-15 2019-10-15
US62/915,386 2019-10-15
US201962939094P 2019-11-22 2019-11-22
US62/939,094 2019-11-22
PCT/US2020/029964 WO2020219988A2 (en) 2019-04-26 2020-04-24 Engineering aav

Publications (3)

Publication Number Publication Date
JP2022530457A true JP2022530457A (ja) 2022-06-29
JP2022530457A5 JP2022530457A5 (https=) 2023-05-01
JPWO2020219988A5 JPWO2020219988A5 (https=) 2023-05-01

Family

ID=70740764

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021563281A Pending JP2022530457A (ja) 2019-04-26 2020-04-24 遺伝子操作ａａｖ

Country Status (11)

Country Link
US (2) US11981967B2 (https=)
EP (1) EP3959226A2 (https=)
JP (1) JP2022530457A (https=)
KR (1) KR20220012249A (https=)
CN (1) CN114269926A (https=)
AU (1) AU2020261435A1 (https=)
CA (1) CA3137961A1 (https=)
IL (1) IL287406A (https=)
SG (1) SG11202111616RA (https=)
TW (1) TW202106700A (https=)
WO (1) WO2020219988A2 (https=)

Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020068990A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
EP3861010A1 (en) 2018-10-02 2021-08-11 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
IL297391A (en) 2020-04-20 2022-12-01 Tenaya Therapeutics Inc An adeno-associated virus with an engineered capsid
CN116096734A (zh) 2020-05-13 2023-05-09 沃雅戈治疗公司 Aav衣壳的向性的重定向
IL301549A (en) * 2020-10-01 2023-05-01 Sangamo Therapeutics Inc Methods of detecting anti-aav antibodies
IL301675A (en) 2020-10-09 2023-05-01 Tenaya Therapeutics Inc Methods and preparations for placophilin-2 gene therapy
EP4259791A1 (en) * 2020-12-09 2023-10-18 Scribe Therapeutics Inc. Aav vectors for gene editing
MX2024004217A (es) 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
MX2024004936A (es) 2021-11-02 2024-05-08 Voyager Therapeutics Inc Variantes de capsides de aav y sus usos.
JP2025500413A (ja) * 2021-12-23 2025-01-09 プロヴェンション・バイオ・インコーポレイテッド バース症候群を治療するための方法及び組成物
CN119325517A (zh) * 2022-04-05 2025-01-17 生物辐射实验室股份有限公司 通过数字试验分析病毒颗粒
WO2024124019A2 (en) * 2022-12-07 2024-06-13 Ginkgo Bioworks, Inc. Aav vectors targeting hematopoietic stem cells
WO2024120528A1 (en) * 2022-12-08 2024-06-13 Huidagene Therapeutics (Singapore) Pte. Ltd. Improved system for producing rna-packaged aav particles
CN116789738B (zh) * 2022-12-08 2023-12-19 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2024228938A1 (en) * 2023-05-01 2024-11-07 St. Jude Children's Research Hospital, Inc. Modified aav p5 promoter for improved vector titer and purity
AU2024274127A1 (en) * 2023-05-15 2025-11-13 Sangamo Therapeutics, Inc. Fitness maturation of engineered aav capsid stac-102
AU2024271436A1 (en) * 2023-05-15 2025-11-13 Sangamo Therapeutics, Inc. Engineered blood brain barrier penetrant aav capsids
WO2025090858A1 (en) * 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
KR102933125B1 (ko) * 2024-02-01 2026-03-04 주식회사 글루진테라퓨틱스 평활근 세포 특이적 유전자 전달이 가능한 아데노연관바이러스 벡터
WO2025194009A1 (en) * 2024-03-13 2025-09-18 Sangamo Therapeutics, Inc. Zinc finger proteins for treating prion disease
KR102828335B1 (ko) * 2024-03-20 2025-07-04 코오롱인더스트리 주식회사 Gne 근육병 치료용 aav 기반의 유전자 치료제
WO2025235682A1 (en) * 2024-05-07 2025-11-13 Sangamo Therapeutics, Inc. Peptides targeting tfrc for blood-brain barrier crossing
WO2025238182A1 (en) * 2024-05-15 2025-11-20 Stichting Amsterdam UMC Optimized aav-mediated transduction of cardiomyocytes
WO2026017727A1 (en) * 2024-07-16 2026-01-22 Genethon Alternative barcoding method to assess transduction efficiency of aav vectors

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016526045A (ja) * 2013-05-31 2016-09-01 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アデノ随伴ウイルス変異体及びその使用方法
JP2016533709A (ja) * 2013-09-26 2016-11-04 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー
JP2017534601A (ja) * 2014-10-03 2017-11-24 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるａａｖベクター
WO2018189244A1 (en) * 2017-04-11 2018-10-18 Ruprecht-Karls-Universität Heidelberg Adeno-associated virus library

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5422251A (en) 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
EP1082413B1 (en) 1998-05-28 2008-07-23 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav5 vector and uses thereof
CA2349838C (en) 1998-11-05 2011-06-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
CA2395872A1 (en) * 2000-01-31 2001-08-02 Human Genome Sciences, Inc. Nucleic acids, proteins, and antibodies
US6916635B2 (en) 2000-10-02 2005-07-12 The Research Foundation Of State University Of New York Hybrid adenovirus/adeno-associated virus vectors and methods of use thereof
US20050106558A1 (en) 2001-12-21 2005-05-19 Luca Perabo Library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
US20080148432A1 (en) * 2005-12-21 2008-06-19 Mark Scott Abad Transgenic plants with enhanced agronomic traits
AU2006204997B2 (en) * 2005-01-12 2011-09-01 Monsanto Technology, Llc Genes and uses for plant improvement
JP6208580B2 (ja) 2010-05-17 2017-10-04 サンガモ セラピューティクス， インコーポレイテッド 新規のｄｎａ結合タンパク質及びその使用
US20140378362A1 (en) * 2012-01-18 2014-12-25 Conopco, Inc., D/B/A Unilever Hair treatment composition
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
WO2017196432A1 (en) * 2016-05-12 2017-11-16 La Jolla Institute For Allergy And Immunology Compositions and methods for diagnosing and treating neurodegenerative disease
EP3526333A4 (en) 2016-10-13 2020-07-29 University of Massachusetts AAV CAPSIDE DESIGNS
WO2018145111A1 (en) 2017-02-06 2018-08-09 Children's Medical Center Corporation Materials and methods for delivering nucleic acids to cochlear and vestibular cells

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016526045A (ja) * 2013-05-31 2016-09-01 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アデノ随伴ウイルス変異体及びその使用方法
JP2016533709A (ja) * 2013-09-26 2016-11-04 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー
JP2017534601A (ja) * 2014-10-03 2017-11-24 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるａａｖベクター
WO2018189244A1 (en) * 2017-04-11 2018-10-18 Ruprecht-Karls-Universität Heidelberg Adeno-associated virus library

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
BUNING, H., ET AL.: ""CAPSID MODIFICATIONS FOR TARGETING AND IMPROVING THE EFFICACY OF AAV VECTORS."", MOLECULAR THERAPY - METHODS & CLINICAL DEVELOPMENT, vol. 12, JPN5022007680, March 2019 (2019-03-01), pages 248 - 265, ISSN: 0005695640 *
DAVIDSSON, M., ET AL.: ""A NOVEL PROCESS OF VIRAL VECTOR BARCODING AND LIBRARY PREPARATION ENABLES HIGH-DIVERSITY LIBRARY GE", SCIENTIFIC REPORTS, vol. 6, no. 1, JPN5022007686, 22 November 2016 (2016-11-22), pages 1 - 18, ISSN: 0005320041 *
KORBELIN, J., ET AL.: ""PULMONARY TARGETING OF ADENO-ASSOCIATED VIRAL VECTORS BY NEXT-GENERATION SEQUENCING-GUIDED SCREENIN", MOLECULAR THERAPY, vol. 24, no. 6, JPN5022007678, June 2016 (2016-06-01), pages 1050 - 1061, ISSN: 0005536659 *
MARSIC, D., ET AL.: ""HIGH-ACCURACY BIODISTRIBUTION ANALYSIS OF ADENO-ASSOCIATED VIRUS VARIANTS BY DOUBLE BARCODE SEQUENC", MOLECULAR THERAPY - METHODS & CLINICAL DEVELOPMENT, vol. 2, JPN5022007683, 2015, pages 1 - 7, ISSN: 0005536661 *
MICHELFELDER, S., ET AL.: ""PEPTIDE LIGANDS INCORPORATED INTO THE THREEFOLD SPIKE CAPSID DOMAIN TO RE-DIRECT GENE TRANSDUCTION", PLOS ONE, vol. 6, no. 8, JPN5022007682, 5 August 2011 (2011-08-05), pages 23101 - 1, ISSN: 0005695639 *
NANCE, M.E., ET AL.: ""PERSPECTIVE ON ADENO-ASSOCIATED VIRUS (AAV) CAPSID MODIFICATION FOR DUCHENNE MUSCULAR DYSTROPHY GEN", HUMAN GENE THERAPY, vol. 26, no. 12, JPN5022007679, December 2015 (2015-12-01), pages 786 - 800, ISSN: 0005536660 *

Also Published As

Publication number Publication date
EP3959226A2 (en) 2022-03-02
US12565685B2 (en) 2026-03-03
US20200370137A1 (en) 2020-11-26
CA3137961A1 (en) 2020-10-29
WO2020219988A2 (en) 2020-10-29
US11981967B2 (en) 2024-05-14
CN114269926A (zh) 2022-04-01
IL287406A (en) 2021-12-01
AU2020261435A1 (en) 2021-11-25
WO2020219988A3 (en) 2020-12-03
SG11202111616RA (en) 2021-11-29
TW202106700A (zh) 2021-02-16
US20240392392A1 (en) 2024-11-28
KR20220012249A (ko) 2022-02-03

Similar Documents

Publication Publication Date Title
US12565685B2 (en) Engineering AAV
US11613766B2 (en) Adeno-associated virus variant capsids and methods of use thereof
CN114174520B (zh) 用于选择性基因调节的组合物和方法
US20200268906A1 (en) Nucleic acid constructs and methods of use
US11408899B2 (en) AAV vector and assay for anti-AAV (adeno-associated virus) neutralizing antibodies
WO2020187268A1 (zh) 一种增强基因编辑的融合蛋白及其应用
CN111718420A (zh) 一种用于基因治疗的融合蛋白及其应用
US20250163469A1 (en) Adeno-associated virus variant capsids and methods of use thereof
KR102957630B1 (ko) 핵산 구조체 및 사용 방법
HK40119094A (zh) 腺相关病毒变异衣壳和其使用方法
WO2020187272A1 (zh) 一种用于基因治疗的融合蛋白及其应用
HK40036161A (zh) 腺相关病毒变异衣壳和其使用方法

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230421

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230421

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240509

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240805

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241111

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250226

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250526

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250728

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20250929