KR20230039592A - 재조합 아데노 연관 바이러스의 패키징 효율을 증가시키기 위한 이온 농도의 사용 - Google Patents

재조합 아데노 연관 바이러스의 패키징 효율을 증가시키기 위한 이온 농도의 사용 Download PDF

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KR20230039592A
KR20230039592A KR1020227004928A KR20227004928A KR20230039592A KR 20230039592 A KR20230039592 A KR 20230039592A KR 1020227004928 A KR1020227004928 A KR 1020227004928A KR 20227004928 A KR20227004928 A KR 20227004928A KR 20230039592 A KR20230039592 A KR 20230039592A
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raav
cells
aav
culture medium
production
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퀴즈하오 왕
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찰스리버레보레토리스,인코포레이티드
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020227004928A 2019-07-15 2020-08-12 재조합 아데노 연관 바이러스의 패키징 효율을 증가시키기 위한 이온 농도의 사용 Ceased KR20230039592A (ko)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US16/511,612 US10801042B1 (en) 2019-07-15 2019-07-15 Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
PCT/US2020/045906 WO2021011941A1 (en) 2019-07-15 2020-08-12 Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus

Publications (1)

Publication Number Publication Date
KR20230039592A true KR20230039592A (ko) 2023-03-21

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KR1020227004928A Ceased KR20230039592A (ko) 2019-07-15 2020-08-12 재조합 아데노 연관 바이러스의 패키징 효율을 증가시키기 위한 이온 농도의 사용

Country Status (7)

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US (2) US10801042B1 (https=)
EP (1) EP3999633A4 (https=)
JP (1) JP2022541238A (https=)
KR (1) KR20230039592A (https=)
CN (1) CN114402066A (https=)
AU (1) AU2020315477A1 (https=)
WO (1) WO2021011941A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10801042B1 (en) * 2019-07-15 2020-10-13 Vigene Biosciences, Inc. Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
WO2022109241A1 (en) * 2020-11-20 2022-05-27 University Of Florida Research Foundation, Incorporated Improved aav production using potassium chloride
WO2024227006A2 (en) * 2023-04-26 2024-10-31 Genzyme Corporation Methods of improving raav production
CN117683797B (zh) * 2023-12-04 2024-07-05 广州派真生物技术有限公司 一种用于重组腺相关病毒包装的质粒系统及其应用

Family Cites Families (46)

* 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
WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Aav4 vector and uses thereof
US6989264B2 (en) 1997-09-05 2006-01-24 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
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
WO1999061601A2 (en) 1998-05-28 1999-12-02 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
WO2002053703A2 (en) 2001-01-05 2002-07-11 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
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
WO2003093479A1 (en) * 2002-05-01 2003-11-13 University Of Florida Research Foundation, Inc. Improved raav expression systems and methods for enhancing transduction of mammalian neural cells
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
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
US7625570B1 (en) 2005-03-10 2009-12-01 The Regents Of The University Of California Methods for purifying adeno-associated virus
EP2311966A3 (en) 2005-10-20 2012-09-05 Amsterdam Molecular Therapeutics (AMT) B.V. Improved AAV vectors produced in insect cells
WO2008024998A2 (en) 2006-08-24 2008-02-28 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
GB2445441B (en) * 2006-09-26 2010-06-30 Ge Healthcare Bio Sciences Nucleic acid purification method
KR20180000341A (ko) 2009-06-16 2018-01-02 젠자임 코포레이션 재조합 aav 벡터에 대한 개선된 정제 방법
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
TR201908020T4 (tr) 2011-02-17 2019-06-21 Univ Pennsylvania Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler.
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
US20140155469A1 (en) 2011-04-19 2014-06-05 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors and viruses
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
CA2853482C (en) 2011-10-28 2020-05-19 Joshua Grieger Cell line for production of adeno-associated virus
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER 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
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
WO2017112948A1 (en) 2015-12-24 2017-06-29 University Of Florida Research Foundation, Inc. Improved aav production using suspension adapted cells
PL3526321T3 (pl) * 2016-10-14 2025-04-28 Ultragenyx Pharmaceutical Inc. Zastosowanie środków regulujących ciśnienie osmotyczne w celu zwiększenia wydajności rekombinacyjnego wirusa związanego z adenowirusami
US10294452B2 (en) 2016-11-22 2019-05-21 Dao-Yao He Lysis, extraction and purification of adeno-associated virus and adenovirus from host cells
WO2019094253A1 (en) * 2017-11-08 2019-05-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
EP3833745A1 (en) * 2018-08-10 2021-06-16 REGENXBIO Inc. Scalable method for recombinant aav production
CN109666697B (zh) * 2019-01-30 2020-03-10 广州派真生物技术有限公司 一种提高腺相关病毒产量的生产工艺
US10801042B1 (en) * 2019-07-15 2020-10-13 Vigene Biosciences, Inc. Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus

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JP2022541238A (ja) 2022-09-22
EP3999633A4 (en) 2023-08-16
WO2021011941A1 (en) 2021-01-21
US20220282277A1 (en) 2022-09-08
EP3999633A1 (en) 2022-05-25
CN114402066A (zh) 2022-04-26
AU2020315477A1 (en) 2022-02-17
US10801042B1 (en) 2020-10-13

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