ATE270324T1 - Methoden zur erhöhung der effizienz rekombinanter aav produkte - Google Patents

Methoden zur erhöhung der effizienz rekombinanter aav produkte

Info

Publication number
ATE270324T1
ATE270324T1 AT98915603T AT98915603T ATE270324T1 AT E270324 T1 ATE270324 T1 AT E270324T1 AT 98915603 T AT98915603 T AT 98915603T AT 98915603 T AT98915603 T AT 98915603T AT E270324 T1 ATE270324 T1 AT E270324T1
Authority
AT
Austria
Prior art keywords
methods
aav
production
expression
compositions
Prior art date
Application number
AT98915603T
Other languages
English (en)
Inventor
Richard J Samulski
Xiao Xiao
Richard Snyder
Original Assignee
Cell Genesys Inc
Univ North Carolina
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 Cell Genesys Inc, Univ North Carolina filed Critical Cell Genesys Inc
Application granted granted Critical
Publication of ATE270324T1 publication Critical patent/ATE270324T1/de

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    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10322—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
    • 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
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
AT98915603T 1997-04-14 1998-04-14 Methoden zur erhöhung der effizienz rekombinanter aav produkte ATE270324T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US4354797P 1997-04-14 1997-04-14
PCT/US1998/007654 WO1998046728A1 (en) 1997-04-14 1998-04-14 Methods for increasing the efficiency of recombinant aav product

Publications (1)

Publication Number Publication Date
ATE270324T1 true ATE270324T1 (de) 2004-07-15

Family

ID=21927717

Family Applications (1)

Application Number Title Priority Date Filing Date
AT98915603T ATE270324T1 (de) 1997-04-14 1998-04-14 Methoden zur erhöhung der effizienz rekombinanter aav produkte

Country Status (10)

Country Link
US (2) US6548286B1 (de)
EP (1) EP1007637B1 (de)
JP (2) JP4135120B2 (de)
AT (1) ATE270324T1 (de)
AU (1) AU728220B2 (de)
CA (1) CA2287478C (de)
DE (1) DE69824859T2 (de)
ES (1) ES2224375T3 (de)
NZ (1) NZ500546A (de)
WO (1) WO1998046728A1 (de)

Families Citing this family (153)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1007637B1 (de) * 1997-04-14 2004-06-30 Cell Genesys, Inc. Methoden zur erhöhung der effizienz rekombinanter aav produkte
US6037177A (en) * 1997-08-08 2000-03-14 Cell Genesys, Inc. Method for increasing the efficiency of recombinant AAV production
US6995006B2 (en) * 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US7078387B1 (en) 1998-12-28 2006-07-18 Arch Development Corp. Efficient and stable in vivo gene transfer to cardiomyocytes using recombinant adeno-associated virus vectors
US7115391B1 (en) 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
AU7841400A (en) * 1999-10-01 2001-05-10 Genovo, Incorporated Production of recombinant aav using adenovirus comprising aav rep/cap genes
JP2002153278A (ja) * 2000-11-22 2002-05-28 Hisamitsu Pharmaceut Co Inc ウイルスベクターの製造に用いられる細胞、その製法およびその細胞を用いたウイルスベクターの製造方法
US20040197312A1 (en) 2003-04-02 2004-10-07 Marina Moskalenko Cytokine-expressing cellular vaccine combinations
US20050136035A1 (en) * 2003-06-03 2005-06-23 Derek Ko Cell specific replication-competent viral vectors comprising a self processing peptide cleavage site
WO2005017149A1 (en) * 2003-06-03 2005-02-24 Cell Genesys, Inc. Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
US7485291B2 (en) * 2003-06-03 2009-02-03 Cell Genesys, Inc. Compositions and methods for generating multiple polypeptides from a single vector using a virus derived peptide cleavage site, and uses thereof
US7186699B2 (en) * 2003-06-03 2007-03-06 Cell Genesys, Inc. Method for treating cancer by vector-mediated delivery of one or more anti-angiogenic or pro-apoptotic genes
CA2559211A1 (en) 2004-03-19 2005-09-29 Yale University Detection, isolation and uses of renalase (monoamine oxidase c)
US20050281786A1 (en) * 2004-06-18 2005-12-22 David Poulsen AAV mediated gene delivery to cochlear cells
EP1765846A4 (de) * 2004-07-13 2010-02-17 Cell Genesys Inc Aav-vektorzusammensetzungen und ihr einsatz in verfahren zür erhöhung der immunoglobulin-expression
WO2006012625A2 (en) * 2004-07-22 2006-02-02 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Stat3 decoy oligonucleotides and uses therefor
US20100132058A1 (en) 2004-07-23 2010-05-27 Diatchenko Luda B Methods and materials for determining pain sensitivity and predicting and treating related disorders
US20060057127A1 (en) * 2004-09-10 2006-03-16 Pocheng Liu Cytokine-expressing cellular vaccines for treatment of prostate cancer
US20060234347A1 (en) * 2005-04-13 2006-10-19 Harding Thomas C Targeting multiple angiogenic pathways for cancer therapy using soluble tyrosine kinase receptors
US7632509B2 (en) * 2005-07-19 2009-12-15 Biosante Pharmaceuticals, Inc. Methods to express recombinant proteins from lentiviral vectors
CN101595228A (zh) 2005-07-21 2009-12-02 艾博特公司 包括sorf构建体的多基因表达和使用多蛋白、前体蛋白和蛋白酶解的方法
WO2007084773A2 (en) * 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
WO2007123737A2 (en) 2006-03-30 2007-11-01 University Of California Methods and compositions for localized secretion of anti-ctla-4 antibodies
US7919079B2 (en) * 2006-03-31 2011-04-05 Biosante Pharmaceuticals, Inc. Cancer immunotherapy compositions and methods of use
US20070292922A1 (en) * 2006-03-31 2007-12-20 Cell Genesys, Inc. Regulated expression of recombinant proteins from adeno-associated viral vectors
CN101511373B (zh) * 2006-06-19 2015-12-09 阿斯克肋匹奥生物制药公司 用于基因治疗的修饰的因子ⅷ和因子ⅸ基因和载体
US20090028857A1 (en) 2007-07-23 2009-01-29 Cell Genesys, Inc. Pd-1 antibodies in combination with a cytokine-secreting cell and methods of use thereof
WO2009032256A2 (en) 2007-08-30 2009-03-12 Cell Genesys, Inc. Apc activators in combination with a cytokine-secreting cell and methods of use thereof
EP2244740A1 (de) 2008-02-19 2010-11-03 Celladon Corporation Zusammensetzungen zur verbesserten aufnahme von virusvektoren in das myokard
US8840881B2 (en) 2008-08-28 2014-09-23 Aduro Gvax Inc. Methods and compositions for treating prostate cancer or inducing a humoral immune response against prostate cancer
US20120172419A1 (en) 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
JP2013509188A (ja) * 2009-10-30 2013-03-14 アボット・ラボラトリーズ Sorf構築物および複数の遺伝子発現
WO2011084694A1 (en) 2009-12-17 2011-07-14 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Stabilized stat3 decoy oligonucleotides and uses therefor
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
CN104159601B (zh) 2012-02-01 2017-05-24 代表亚利桑那大学的亚利桑那董事会 弯曲杆菌属免疫原性组合物及其用途
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
EP2692868A1 (de) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adenoassoziierte virale (AAV) Vektoren für die Transduktion von Fettgewebe
DE102013004595A1 (de) 2013-03-15 2014-09-18 Emergent Product Development Germany Gmbh RSV-Impfstoffe
WO2014201252A2 (en) * 2013-06-13 2014-12-18 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
JP6454352B2 (ja) 2013-11-05 2019-01-16 タカラ バイオ ユーエスエー, インコーポレイテッド 乾燥トランスフェクション組成物、乾燥トランスフェクション組成物を生成する方法、及び乾燥トランスフェクション組成物を使用する方法
GB201322626D0 (en) 2013-12-19 2014-02-05 Immutep S A Combined preparations for the treatment of cancer
BR112016020688B1 (pt) 2014-03-09 2024-01-30 The Trustees Of The University Of Pennsylvania Vetores virais recombinantes, vírus adenoassociado recombinante, composição farmacêutica, bem como uso dos mesmos
ES2822551T3 (es) 2014-03-17 2021-05-04 Adverum Biotechnologies Inc Composiciones y métodos para la expresión génica mejorada en células de los conos
US11555059B2 (en) 2014-04-25 2023-01-17 The Trustees Of The University Of Pennsylvania LDLR variants and their use in compositions for reducing cholesterol levels
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
CR20160519A (es) 2014-05-13 2017-01-27 The Trustees Of The Univ Of Pennysylvania Composiciones que comprenden avv que expresa constructos de anticuerpos duales y sus usos
US10973931B2 (en) 2014-09-16 2021-04-13 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
EP3242945B1 (de) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Einzelvektor-genkonstrukt mit insulin- und glucokinasegenen
GB201500374D0 (en) 2015-01-09 2015-02-25 Immutep S A Combined preparations for the treatment of cancer
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
FI3265178T3 (fi) 2015-03-02 2026-08-03 Adverum Biotechnologies Inc Koostumuksia ja menetelmiä polynukleotidien viemiseksi intravitreaalisesti retinaalisiin tappisoluihin
JP6851319B2 (ja) 2015-04-27 2021-03-31 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ヒト疾患のCRISPR/Cas9媒介性の修正のためのデュアルAAVベクター系
WO2016200543A2 (en) 2015-05-13 2016-12-15 The Trustees Of The University Of Pennsylvania Aav-mediated expression of anti-inluenza antibodies and methods of use thereof
ES3009026T3 (en) 2015-07-22 2025-03-25 Univ Duke High-throughput screening of regulatory element function with epigenome editing technologies
WO2017024198A1 (en) 2015-08-06 2017-02-09 The Trustees Of The University Of Pennsylvania Glp-1 and use thereof in compositions for treating metabolic diseases
KR20240132120A (ko) 2015-08-25 2024-09-02 듀크 유니버시티 Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법
HK1257024A1 (zh) 2015-08-31 2019-10-11 宾夕法尼亚大学 用於治疗伴侣动物的aav-epo
EP3359663A4 (de) 2015-09-24 2019-08-14 The Trustees of The University of Pennsylvania Zusammensetzung und verfahren zur behandlung einer komplementvermittelten erkrankung
US11219694B2 (en) 2015-09-24 2022-01-11 The University Of North Carolina At Chapel Hill Methods and compositions for reducing metastases
WO2017062750A1 (en) 2015-10-09 2017-04-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating stargardt's disease and other ocular disorders
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US11135313B2 (en) 2015-10-28 2021-10-05 The Trustees Of The University Of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
EP4085934A1 (de) 2015-12-11 2022-11-09 The Trustees of The University of Pennsylvania Gentherapeutische behandlung der familiären hypercholesterinämie
CN109069668B (zh) 2015-12-14 2023-04-18 宾夕法尼亚州大学信托人 用于眼病的基因疗法
AU2016370590B2 (en) 2015-12-14 2023-11-02 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
EP3443081A4 (de) 2016-04-13 2019-10-30 Duke University Crispr/cas9-basierte repressoren zur in-vivo-ausschaltung von gen-targets und verfahren zur verwendung
ES2908467T3 (es) 2016-04-29 2022-04-29 Adverum Biotechnologies Inc Evasión de anticuerpos neutralizantes por un virus adenoasociado recombinante
US11197936B2 (en) 2016-07-08 2021-12-14 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving RDH12
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Cpf1に基づくゲノム編集の治療適用
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
ES2982489T3 (es) 2016-08-19 2024-10-16 Univ Florida Composiciones para el tratamiento de afecciones que utilizan virus adeno-asociados recombinantes autocomplementarios
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
ES2931960T3 (es) 2016-12-01 2023-01-05 Inst Nat Sante Rech Med Composiciones farmacéuticas para el tratamiento de enfermedades degenerativas retinianas
US11958886B2 (en) 2016-12-07 2024-04-16 University Of Florida Research Foundation, Incorporated IL-1RA cDNAs
JP2020510648A (ja) 2017-02-20 2020-04-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 家族性高コレステロール血症を処置するための遺伝子治療
TW201837170A (zh) 2017-02-28 2018-10-16 賓州大學委員會 新穎aav媒介的流感疫苗
RS65241B1 (sr) 2017-02-28 2024-03-29 Univ Pennsylvania Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
WO2018160849A1 (en) 2017-03-01 2018-09-07 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
KR102616820B1 (ko) 2017-03-17 2023-12-21 애드베룸 바이오테크놀로지스, 인코포레이티드 향상된 유전자 발현을 위한 조성물 및 방법
BR112019021595A2 (pt) 2017-04-18 2020-05-12 Glaxosmithkline Intellectual Property Development Limited Célula produtora de vírus adeno-associado, vetor de ácido nucleico, métodos para produzir uma linhagem de células de empacotamento estável de vírus adeno-associado e uma partícula de vetor de vírus adeno-associado com defeito de replicação, célula de empacotamento de vírus adeno-associado, e, partícula de vetor de vírus adeno-associado com defeito de replicação.
GB201706090D0 (en) * 2017-04-18 2017-05-31 Glaxosmithkline Ip Dev Ltd Methods for adeno-associated viral vector production
US11879133B2 (en) 2017-04-24 2024-01-23 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
BR112019023303A2 (pt) 2017-05-11 2020-06-16 The Trustees Of The University Of Pennsylvania Terapia de gene para lipofuscinoses ceroides neuronais
BR112019024608A2 (pt) 2017-05-24 2020-06-23 Universitat Autonoma De Barcelona Construto de expressão viral que compreende uma sequência de codificação do fator de crescimento de fibroblastos 21 (fgf21)
KR102765034B1 (ko) 2017-05-24 2025-02-11 유니버시타트 아우토노마 데 바르셀로나 섬유아세포 성장 인자 21 (fgf21) 코딩 서열을 포함하는 바이러스 발현 컨스트럭트
US11793887B2 (en) 2017-05-31 2023-10-24 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
US11827898B2 (en) 2017-06-14 2023-11-28 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
CA3077594C (en) 2017-10-10 2022-11-29 Nantbio, Inc. Modified ec7 cells having low toxicity to viral production payloads
CA3086045A1 (en) 2017-12-19 2019-06-27 Akouos, Inc. Aav-mediated delivery of therapeutic antibodies to the inner ear
GB201800903D0 (en) 2018-01-19 2018-03-07 Oxford Genetics Ltd Vectors
EP3768695A4 (de) 2018-02-27 2022-04-06 The Trustees of the University of Pennsylvania Neuartige adeno-assoziierte virus (aav)-vektoren, aav-vektoren mit reduzierter kapsiddeamidierung und verwendungen davon
EP3860659A1 (de) 2018-10-01 2021-08-11 The Trustees of the University of Pennsylvania Zusammensetzungen zur behandlung von gm1-gangliosidose
IL283452B2 (en) 2018-11-26 2026-04-01 Univ Barcelona Autonoma FIBROBLAST GROWTH FACTOR 21 (FGF21)-BASED GENE THERAPY
GB201901571D0 (en) 2019-02-05 2019-03-27 Oxford Genetics Ltd Inducible AAV sysyem
MX2021010134A (es) 2019-02-22 2021-09-23 Univ Pennsylvania Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn.
EP3931337A1 (de) 2019-02-26 2022-01-05 The Trustees of The University of Pennsylvania Zur behandlung von morbus krabbe nützliche zusammensetzungen
AU2020232692A1 (en) 2019-03-04 2021-09-30 The Trustees Of The University Of Pennsylvania Neuroprotective gene therapy targeting the AKT pathway
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏(duchenne)肌肉失養症之大型突變熱點之aav載體介導之刪除
JP7715640B2 (ja) 2019-05-03 2025-07-30 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異染性白質ジストロフィーの治療に有用な組成物
EP3976087A1 (de) 2019-05-31 2022-04-06 Universitat Autònoma De Barcelona Insulingentherapie
ES2948620T3 (es) 2019-07-10 2023-09-14 Masonic Medical Res Laboratory Vgll4 con elemento regulador CIS de UCP-1 y método de uso del mismo
US10557149B1 (en) * 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
EP4007814A1 (de) 2019-07-26 2022-06-08 Akouos, Inc. Verfahren zur behandlung von hörverlust unter verwendung eines sekretierten zielproteins
KR20220112283A (ko) 2019-12-10 2022-08-10 다케다 야쿠힌 고교 가부시키가이샤 Hunter 질환 치료용 아데노-연합된 바이러스 벡터
BR112022011916A2 (pt) 2019-12-19 2022-09-06 Univ Pennsylvania Composições para tratar a ataxia de friedreich
KR20230041965A (ko) 2020-05-13 2023-03-27 아카우오스, 인크. Slc26a4-연관 청력손실을 치료하기 위한 조성물 및 방법
CN115916985A (zh) 2020-05-26 2023-04-04 巴塞罗那自治大学 用于中枢神经系统障碍的成纤维细胞生长因子21(fgf21)基因疗法
US20230372539A1 (en) 2020-08-24 2023-11-23 The Trustees Of The University Of Pennsylvania Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
AU2021332331A1 (en) 2020-08-26 2023-03-09 The Trustees Of The University Of Pennsylvania Recombinant adeno-associated virus for treatment of GRN-associated adult-onset neurodegeneration
JP2023544803A (ja) 2020-10-07 2023-10-25 レジェンクスバイオ インコーポレーテッド Cln2疾患の眼症状に対する遺伝子療法
GB202016254D0 (en) * 2020-10-13 2020-11-25 Freeline Therapeutics Ltd Plasmid system
WO2022082109A1 (en) 2020-10-18 2022-04-21 The Trustees Of The University Of Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
MX2023005113A (es) 2020-10-29 2023-08-04 Univ Pennsylvania Cápsides de aav y composiciones que las contienen.
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
AR124216A1 (es) 2020-12-01 2023-03-01 Univ Pennsylvania Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen
CN117157108A (zh) 2020-12-29 2023-12-01 阿库斯股份有限公司 用于治疗clrn1相关听力损失和/或视力损失的组合物和方法
CA3206590A1 (en) 2021-01-30 2022-08-04 Maria Fatima Bosch Tubert Gene therapy for monogenic diabetes
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
AR124981A1 (es) 2021-02-26 2023-05-24 Takeda Pharmaceuticals Co Composición y métodos para el tratamiento de la enfermedad de fabry
IL307633A (en) 2021-04-12 2023-12-01 Univ Pennsylvania Useful preparations in the treatment of spinal muscular atrophy (SBMA)
WO2022226296A2 (en) 2021-04-23 2022-10-27 University Of Rochester Genome editing by directed non-homologous dna insertion using a retroviral integrase-cas fusion protein and methods of treatment
EP4089171A1 (de) 2021-05-12 2022-11-16 Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncológicas Carlos III (F.S.P. CNIO) Rekombinante tert-codierende virale genome und vektoren
KR20240107201A (ko) 2021-08-31 2024-07-08 스카우트 바이오, 인코포레이티드 항원-결합 분자 및 이의 용도
US20250288692A1 (en) 2021-09-30 2025-09-18 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
US20240384298A1 (en) 2021-10-02 2024-11-21 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
TW202340467A (zh) 2022-01-10 2023-10-16 賓州大學委員會 有用於治療c9orf72介導之病症之組成物及方法
CN119317718A (zh) 2022-01-25 2025-01-14 宾夕法尼亚州大学信托人 用于改善心脏转导和肝脏去靶向的aav衣壳
CN115851837B (zh) * 2022-01-25 2025-12-16 广州派真生物技术有限公司 一种提高杆状病毒系统生产腺相关病毒的方法及应用
AR128422A1 (es) 2022-02-02 2024-05-08 Akouos Inc Construcciones de anticuerpos anti-vegf y métodos relacionados para el tratamiento de los síntomas asociados al schwannoma vestibular
EP4504950A1 (de) 2022-04-01 2025-02-12 Takeda Pharmaceutical Company Limited Gentherapie für erkrankungen mit zns-manifestationen
CA3247109A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and treatment methods for metastatic breast cancer that is human epidermal growth factor receptor 2 positive and other cancers
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
WO2024015966A2 (en) 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
KR20250052387A (ko) 2022-08-25 2025-04-18 다케다 야쿠힌 고교 가부시키가이샤 파브리병의 치료에서의 사용을 위한 조성물
EP4584383A1 (de) 2022-09-07 2025-07-16 Universitat Autònoma De Barcelona Beta-hexosaminidase-vektoren
WO2024105638A1 (en) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of hunter syndrome
EP4634394A2 (de) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Rekombinante aav-mutantenvektoren mit herz- und skelettmuskelspezifischen zielmotiven und zusammensetzungen damit
KR20260010739A (ko) 2023-05-11 2026-01-21 유니버시티 하스피탈즈 클리블랜드 메디컬 센터 항불안 치료
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
KR20260114566A (ko) 2023-11-14 2026-07-22 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 심근 및 골격근-특이적 표적화 모티프를 갖는 조성물 및 이의 용도
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
TW202548022A (zh) 2024-03-03 2025-12-16 美商帕西奇生物公司 用於治療神經退化性病症之重組腺相關病毒
WO2026122921A1 (en) 2024-12-05 2026-06-11 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2026151888A1 (en) 2025-01-08 2026-07-16 The Trustees Of The University Of Pennsylvania Compositions for treating friedreich's ataxia

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5116742A (en) 1986-12-03 1992-05-26 University Patents, Inc. RNA ribozyme restriction endoribonucleases and methods
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
DE69433922T2 (de) * 1993-11-09 2005-07-28 Medical College Of Ohio, Toledo Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
US5693531A (en) 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US6027931A (en) 1995-08-03 2000-02-22 Avigen, Inc. High-efficiency AA V helper functions
JPH11514853A (ja) 1995-09-08 1999-12-21 ジエンザイム コーポレイション 遺伝子治療のための改良されたaavベクター
US6086913A (en) * 1995-11-01 2000-07-11 University Of British Columbia Liposomal delivery of AAV vectors
EP1007637B1 (de) * 1997-04-14 2004-06-30 Cell Genesys, Inc. Methoden zur erhöhung der effizienz rekombinanter aav produkte
US6037177A (en) * 1997-08-08 2000-03-14 Cell Genesys, Inc. Method for increasing the efficiency of recombinant AAV production

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US20030211614A1 (en) 2003-11-13
AU6974198A (en) 1998-11-11
CA2287478A1 (en) 1998-10-22
EP1007637A4 (de) 2002-04-03
US7229823B2 (en) 2007-06-12
US6548286B1 (en) 2003-04-15
AU728220B2 (en) 2001-01-04
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