CA2233167A1 - Method for transferring genes to the heart using aav vectors - Google Patents

Method for transferring genes to the heart using aav vectors Download PDF

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Publication number
CA2233167A1
CA2233167A1 CA002233167A CA2233167A CA2233167A1 CA 2233167 A1 CA2233167 A1 CA 2233167A1 CA 002233167 A CA002233167 A CA 002233167A CA 2233167 A CA2233167 A CA 2233167A CA 2233167 A1 CA2233167 A1 CA 2233167A1
Authority
CA
Canada
Prior art keywords
aav
vector
heart
gene
dna
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.)
Abandoned
Application number
CA002233167A
Other languages
English (en)
French (fr)
Inventor
Martin J. Kaplitt
Edward B. Diethrich
Michael G. Kaplitt
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.)
Rockefeller University
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA2233167A1 publication Critical patent/CA2233167A1/en
Abandoned legal-status Critical Current

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Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Electrotherapy Devices (AREA)
  • Immobilizing And Processing Of Enzymes And Microorganisms (AREA)
CA002233167A 1995-09-27 1996-09-26 Method for transferring genes to the heart using aav vectors Abandoned CA2233167A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US08/534,351 US6162796A (en) 1995-09-27 1995-09-27 Method for transferring genes to the heart using AAV vectors
US08/534,351 1995-09-27

Publications (1)

Publication Number Publication Date
CA2233167A1 true CA2233167A1 (en) 1997-04-03

Family

ID=24129676

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002233167A Abandoned CA2233167A1 (en) 1995-09-27 1996-09-26 Method for transferring genes to the heart using aav vectors

Country Status (7)

Country Link
US (1) US6162796A (cg-RX-API-DMAC7.html)
EP (1) EP0859853A1 (cg-RX-API-DMAC7.html)
JP (1) JPH11512608A (cg-RX-API-DMAC7.html)
AU (1) AU727835B2 (cg-RX-API-DMAC7.html)
CA (1) CA2233167A1 (cg-RX-API-DMAC7.html)
WO (1) WO1997012050A1 (cg-RX-API-DMAC7.html)
ZA (1) ZA968170B (cg-RX-API-DMAC7.html)

Families Citing this family (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6306830B1 (en) 1996-09-05 2001-10-23 The Regents Of The University Of California Gene therapy for congestive heart failure
US6752987B1 (en) 1995-02-28 2004-06-22 The Regents Of The University Of California Adenovirus encoding human adenylylcyclase (AC) VI
US7745416B2 (en) * 1995-04-11 2010-06-29 The Regents Of The University Of California Method for in vivo regulation of cardiac muscle contractility
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
US20030206891A1 (en) * 1995-06-07 2003-11-06 Ariad Gene Therapeutics, Inc. Rapamycin-based biological regulation
ES2337853T3 (es) * 1995-06-07 2010-04-29 University Of North Carolina At Chapel Hill Transduccion de mioblastos por vaa.
US5846528A (en) * 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5962313A (en) * 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US5858351A (en) * 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
CA2247099A1 (en) * 1996-03-04 1997-09-12 Targeted Genetics Corporation Methods for transducing cells in blood vessels using recombinant aav vectors
ATE465267T1 (de) * 1996-09-06 2010-05-15 Univ Pennsylvania Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen
US20020037867A1 (en) * 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
US6486133B1 (en) 1997-03-07 2002-11-26 The Wistar Institute Of Anatomy And Biology Methods for inducing localized vascular development and enhancing the repair of a wound in the mammamian dermis
US6995006B2 (en) * 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6129757A (en) * 1998-05-18 2000-10-10 Scimed Life Systems Implantable members for receiving therapeutically useful compositions
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
JP2002533359A (ja) * 1998-12-28 2002-10-08 アーチ ディベロップメント コーポレイション 組換えアデノ随伴ウイルスベクターを用いる、心筋細胞への効率的かつ安定なインビボでの遺伝子移入
JP4693244B2 (ja) 1999-03-18 2011-06-01 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 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
AU2460001A (en) 1999-12-27 2001-07-09 Regents Of The University Of California, The Gene therapy for congestive heart failure
US20030144237A1 (en) * 2000-04-13 2003-07-31 Peter Carmeliet Use of inhibition of a gas6 function or of a gas6 receptor for preventing and treating a cardiovascular disease
EP1870473A1 (en) * 2000-08-17 2007-12-26 Keiya Ozawa Adeno-associated virus-mediated delivery of angiogenic factors
EP1311699B1 (en) * 2000-08-17 2007-07-04 Keiya Ozawa Adeno-associated virus-mediated delivery of angiogenic factors
US20020192688A1 (en) * 2001-04-05 2002-12-19 Xiaoming Yang Imaging nucleic acid delivery
AU2002305148A1 (en) * 2001-04-05 2002-10-21 Johns Hopkins University Imaging systems for in vivo protocols
WO2003018752A2 (en) 2001-08-23 2003-03-06 The Wistar Institute Of Anatomy And Biology An organotypic intestinal culture and methods of use thereof
AU2003228410A1 (en) * 2002-04-01 2003-10-20 The Johns Hopkins University School Of Medicine Device, systems and methods for heating of a vessel in combination with mr/nmr imaging
US20040009151A1 (en) * 2002-04-04 2004-01-15 Kay Mark A. Methods for delivering recombinant adeno-associated virus virions to the liver of a mammal
EP1486567A1 (en) * 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
WO2005027995A2 (en) * 2003-09-19 2005-03-31 The Trustees Of The University Of Pennsylvania Global myocardial perfusion catheter
US8859273B2 (en) * 2003-12-24 2014-10-14 Medtronic, Inc. Methods of using HCN genes to treat cardiac arrhythmias
US20050244377A1 (en) * 2003-12-24 2005-11-03 Daniel Sigg Methods of treating cardiac disorders by suppressing the expression of the potassium inwardly-rectifying channel
US20060128647A1 (en) * 2003-12-24 2006-06-15 Daniel Sigg Novel oligonucleotides and treating cardiac disorders by using the same
CA2571159A1 (en) * 2004-06-18 2006-03-30 The University Of Montana Aav mediated gene delivery to cochlear cells
CN1286981C (zh) * 2004-11-30 2006-11-29 华中科技大学同济医学院附属同济医院 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法
CN101495627A (zh) * 2006-07-25 2009-07-29 塞拉东公司 用于基因疗法的延长的腺相关病毒载体的顺行性心外膜冠状动脉输注
CN101977632A (zh) 2008-02-19 2011-02-16 塞拉东公司 用于增强病毒载体于心肌中的摄取的组合物
WO2013163346A1 (en) * 2012-04-25 2013-10-31 The Regents Of The University Of California Synthetic promoter for modulating gene expression
US10648000B2 (en) 2015-02-16 2020-05-12 University Of Florida Research Foundation, Incorporated rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes
ES2926977T3 (es) * 2016-01-19 2022-10-31 Univ California Métodos para el tratamiento de la enfermedad de Danon y otros trastornos de autofagia
EP3780952A4 (en) 2018-04-16 2022-03-09 The Trustees of the University of Pennsylvania COMPOSITIONS AND METHODS OF TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) * 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5252479A (en) * 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5478745A (en) * 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
AU1049895A (en) * 1993-11-05 1995-05-23 New York University Methods relating to ir-95
CA2176215C (en) * 1993-11-09 2007-06-26 James P. Trempe Stable cell lines capable of expressing the adeno-associated virus replication gene
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
US5580766A (en) * 1994-01-14 1996-12-03 Alexion Pharmaceuticals, Inc. Retroviral vector particles for transducing non-proliferating cells
FR2716682B1 (fr) * 1994-01-28 1996-04-26 Centre Nat Rech Scient Procédé de préparation de virus adéno-associés (AAV) recombinants et utilisations.
DE9416814U1 (de) * 1994-10-19 1996-02-15 Pöting GmbH & Co. KG, 40699 Erkrath Vorrichtung zur Herstellung von Glasformlingen nach dem Preßverfahren
EP0760682A4 (en) * 1995-02-28 1998-09-09 Univ California ANGIOGENIC THERAPY BY GENE TRANSFER
US5797870A (en) * 1995-06-07 1998-08-25 Indiana University Foundation Pericardial delivery of therapeutic and diagnostic agents

Also Published As

Publication number Publication date
US6162796A (en) 2000-12-19
JPH11512608A (ja) 1999-11-02
AU727835B2 (en) 2001-01-04
ZA968170B (en) 1997-07-21
AU7246796A (en) 1997-04-17
EP0859853A1 (en) 1998-08-26
WO1997012050A1 (en) 1997-04-03

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Legal Events

Date Code Title Description
EEER Examination request
FZDE Discontinued