JP2022542829A - 改善されたパッケージング効率を有する組換え改変アデノ随伴ウイルス - Google Patents

改善されたパッケージング効率を有する組換え改変アデノ随伴ウイルス Download PDF

Info

Publication number
JP2022542829A
JP2022542829A JP2022502942A JP2022502942A JP2022542829A JP 2022542829 A JP2022542829 A JP 2022542829A JP 2022502942 A JP2022502942 A JP 2022502942A JP 2022502942 A JP2022502942 A JP 2022502942A JP 2022542829 A JP2022542829 A JP 2022542829A
Authority
JP
Japan
Prior art keywords
raav
cis
domain
aav
associated virus
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
JP2022502942A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022542829A5 (https=
JPWO2021011034A5 (https=
Inventor
ワング,チィツァオ
Original Assignee
チャールズ・リバー・ラボラトリーズ・インコーポレーテッド
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 チャールズ・リバー・ラボラトリーズ・インコーポレーテッド filed Critical チャールズ・リバー・ラボラトリーズ・インコーポレーテッド
Publication of JP2022542829A publication Critical patent/JP2022542829A/ja
Publication of JP2022542829A5 publication Critical patent/JP2022542829A5/ja
Publication of JPWO2021011034A5 publication Critical patent/JPWO2021011034A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C07K14/075Adenoviridae
    • 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0601Invertebrate cells or tissues, e.g. insect cells; Culture media therefor
    • 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0684Cells of the urinary tract or kidneys
    • C12N5/0686Kidney cells
    • 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
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343Use 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
    • C12N2750/14122New 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
    • C12N2750/14151Methods of production or purification of viral material
    • C12N2750/14152Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/40Systems of functionally co-operating 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
    • C12N2820/00Vectors comprising a special origin of replication system
    • C12N2820/60Vectors comprising a special origin of replication system from viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Urology & Nephrology (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2022502942A 2019-07-15 2020-03-12 改善されたパッケージング効率を有する組換え改変アデノ随伴ウイルス Pending JP2022542829A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US16/512,051 US10653731B1 (en) 2019-07-15 2019-07-15 Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
US16/512,051 2019-07-15
PCT/US2020/022326 WO2021011034A1 (en) 2019-07-15 2020-03-12 Recombinantly-modified adeno-associated virus (raav) having improved packaging efficiency

Publications (3)

Publication Number Publication Date
JP2022542829A true JP2022542829A (ja) 2022-10-07
JP2022542829A5 JP2022542829A5 (https=) 2023-03-16
JPWO2021011034A5 JPWO2021011034A5 (https=) 2023-03-16

Family

ID=70736392

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022502942A Pending JP2022542829A (ja) 2019-07-15 2020-03-12 改善されたパッケージング効率を有する組換え改変アデノ随伴ウイルス

Country Status (7)

Country Link
US (2) US10653731B1 (https=)
EP (1) EP3999648A4 (https=)
JP (1) JP2022542829A (https=)
KR (1) KR20220034214A (https=)
CN (1) CN114375337A (https=)
AU (1) AU2020315513A1 (https=)
WO (1) WO2021011034A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
EP4658759A2 (en) * 2023-02-02 2025-12-10 Agathos Biologics Baby hamster kidney (bhk) cells transformed with the adenoviral e1 gene for production of recombinant adeno-associated virus

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014503173A (ja) * 2009-11-05 2014-02-13 プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ 制御された発現系
JP2018537969A (ja) * 2015-10-29 2018-12-27 テンプル ユニヴァーシティ − オブ ザ コモンウェルス システム オブ ハイアー エデュケーション Dnaポリメラーゼシータによる核酸の3’末端の修飾
US20190211357A1 (en) * 2012-12-14 2019-07-11 Case Western Reserve University Genomic rna packaging enhancer element

Family Cites Families (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6342390B1 (en) 1994-11-23 2002-01-29 The United States Of America As Represented By The Secretary Of Health And Human Services Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
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
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6642051B1 (en) 1997-10-21 2003-11-04 Targeted Genetics Corporation Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
IT1297074B1 (it) 1997-11-21 1999-08-03 Angeletti P Ist Richerche Bio Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le
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
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
DE19905501B4 (de) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
US7115391B1 (en) 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
US7208315B2 (en) 2000-01-05 2007-04-24 Fred Hutchinson Cancer Research Center Compositions and methods for efficient AAV vector production
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
US6841357B1 (en) 2001-03-30 2005-01-11 Stratagene California Method and kits for titering adeno-associated virus vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
WO2003042361A2 (en) 2001-11-09 2003-05-22 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
NZ532635A (en) * 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
US7122348B2 (en) 2002-12-11 2006-10-17 City Of Hope AAV2 Rep protein fusions
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
US7625570B1 (en) 2005-03-10 2009-12-01 The Regents Of The University Of California Methods for purifying adeno-associated virus
EP2311967B1 (en) 2005-10-20 2017-09-20 UniQure IP B.V. Improved AAV vectors produced in insect cells
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
WO2010022195A2 (en) 2008-08-20 2010-02-25 Virxsys Corporation Non-integrating lenti/adeno-associated virus hybrid vector system
HUE028341T2 (en) 2009-06-16 2016-12-28 Genzyme Corp Improved methods for purification of recombinant aav vectors
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
DK2675902T3 (da) 2011-02-17 2019-06-03 Univ Pennsylvania Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
WO2012145509A2 (en) 2011-04-19 2012-10-26 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors, and viruses
HRP20171334T1 (hr) 2011-04-22 2017-11-17 The Regents Of The University Of California Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe
US9441206B2 (en) 2011-10-28 2016-09-13 The University Of North Carolina At Chapel Hill Cell line for production of adeno-associated virus
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
US9447433B2 (en) 2013-03-15 2016-09-20 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
CN105765066B (zh) 2013-11-29 2018-12-14 宝生物工程株式会社 用于定量腺伴随病毒的方法
GB201407852D0 (en) * 2014-05-02 2014-06-18 Iontas Ltd Preparation of libraries od protein variants expressed in eukaryotic cells and use for selecting binding molecules
US11208630B2 (en) 2015-12-24 2021-12-28 University Of Florida Research Foundation, Incorporated AAV production using suspension adapted cells
CA3012409A1 (en) * 2016-01-29 2017-08-03 Sirion Biotech Gmbh Aav-based conditional expression system
JP7632835B2 (ja) * 2016-05-20 2025-02-19 プレジデント アンド フェローズ オブ ハーバード カレッジ 加齢関連疾患及び症状の遺伝子治療法
US10294452B2 (en) 2016-11-22 2019-05-21 Dao-Yao He Lysis, extraction and purification of adeno-associated virus and adenovirus from host cells
CN108727486A (zh) * 2017-04-24 2018-11-02 舒泰神(北京)生物制药股份有限公司 长效神经生长因子、制备方法及其组合物
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014503173A (ja) * 2009-11-05 2014-02-13 プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ 制御された発現系
US20190211357A1 (en) * 2012-12-14 2019-07-11 Case Western Reserve University Genomic rna packaging enhancer element
JP2018537969A (ja) * 2015-10-29 2018-12-27 テンプル ユニヴァーシティ − オブ ザ コモンウェルス システム オブ ハイアー エデュケーション Dnaポリメラーゼシータによる核酸の3’末端の修飾

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
DARBY L THOMAS ET AL.: "Scalable recombinant adeno-associated virus production using recombinant herpes simplex vir", HUM GENE THER., vol. 20(8), JPN7024001111, 31 August 2009 (2009-08-31), pages 861 - 70, ISSN: 0005286214 *
HAIFENG CHEN ET AL.: "Adeno-associated virus vectors for human gene therapy", WORLD JOURNAL OF MEDICAL GENETICS, vol. 5, no. 3, JPN7024001112, 1 January 2015 (2015-01-01), pages 28 - 45, ISSN: 0005286213 *
STIFANI SATKUNANATHAN ET AL: "The function of DNA binding protein nucleophosmin in AAV replication", VIROLOGY, vol. 510, JPN7024001110, 31 December 2017 (2017-12-31), pages 46 - 54, ISSN: 0005286215 *

Also Published As

Publication number Publication date
KR20220034214A (ko) 2022-03-17
US12156894B2 (en) 2024-12-03
US10653731B1 (en) 2020-05-19
US20220257678A1 (en) 2022-08-18
AU2020315513A1 (en) 2022-02-17
WO2021011034A1 (en) 2021-01-21
EP3999648A1 (en) 2022-05-25
CN114375337A (zh) 2022-04-19
EP3999648A4 (en) 2023-08-16

Similar Documents

Publication Publication Date Title
CN114450393B (zh) 重组修饰腺相关病毒辅助载体及其在提高重组修饰腺相关病毒的包装效率的用途
KR102370675B1 (ko) 표적 핵산의 변형을 위한 개선된 방법
US20220282277A1 (en) Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
KR20220161297A (ko) 신규 세포주
KR20130132405A (ko) 형질전환 빈도를 증가시키기 위해 변형된 아그로박테리움 균주
CN113518825A (zh) Wiskott-aldrich综合征的造血干细胞基因疗法
CN114645066B (zh) 一种用于艾滋病基因治疗的核酸构建体
KR20240029020A (ko) Dna 변형을 위한 crispr-트랜스포손 시스템
CN117480257A (zh) 用于高水平rAAV生产的系统
US12156894B2 (en) Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
HK40067794A (en) Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
HK40067796A (en) Recombinantly-modified adeno-associated virus (raav) having improved packaging efficiency
CN114874332B (zh) 经修饰的rnf112作为治疗als药物的应用
CN111534542A (zh) piggyBac转座子系统介导的真核生物转基因细胞系及构建方法
HK40067797A (en) Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
KR20150123741A (ko) 다수의 단백질-단백질 상호작용 동시 분석을 위한 벡터
KR102752143B1 (ko) 재조합 구제역 바이러스 a형 및 이를 포함하는 구제역 백신 조성물

Legal Events

Date Code Title Description
A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A712

Effective date: 20220526

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20220526

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220916

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230308

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230308

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20240228

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240319

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240612

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240912

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20250306