WO2000038518A1 - Transfert efficace et stable in vivo de genes dans des cardiomyocytes a l'aide de vecteurs viraux adeno-associes de recombinaison - Google Patents

Transfert efficace et stable in vivo de genes dans des cardiomyocytes a l'aide de vecteurs viraux adeno-associes de recombinaison Download PDF

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Publication number
WO2000038518A1
WO2000038518A1 PCT/US1999/031093 US9931093W WO0038518A1 WO 2000038518 A1 WO2000038518 A1 WO 2000038518A1 US 9931093 W US9931093 W US 9931093W WO 0038518 A1 WO0038518 A1 WO 0038518A1
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Prior art keywords
aav
body weight
infused
per gram
cardiomyocytes
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PCT/US1999/031093
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English (en)
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WO2000038518A9 (fr
Inventor
Jeffrey M. Leiden
Eric Svensson
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Arch Development Corporation
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Publication date
Application filed by Arch Development Corporation filed Critical Arch Development Corporation
Priority to CA002356551A priority Critical patent/CA2356551A1/fr
Priority to AU23942/00A priority patent/AU763049B2/en
Priority to EP99967703A priority patent/EP1139751A4/fr
Priority to JP2000590483A priority patent/JP2002533359A/ja
Publication of WO2000038518A1 publication Critical patent/WO2000038518A1/fr
Publication of WO2000038518A9 publication Critical patent/WO2000038518A9/fr

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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
    • 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
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • 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
    • A61P9/06—Antiarrhythmics
    • 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
    • A61P9/08—Vasodilators for multiple indications
    • 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
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Cardiology (AREA)
  • General Health & Medical Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Hospice & Palliative Care (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

L'invention concerne l'utilisation de vecteurs viraux adéno-associés de recombinaison (rAAV) pour la transduction de cardiomyocytes in vivo par injection de vecteurs rAAV dans une artère ou un sinus coronaire. L'infection au virus rAAV n'est pas associée à une inflammation du myocarde ou à la nécrose des myocytes détectable. Ainsi, le vecteur rAAV est un vecteur utile pour l'expression stable de gènes thérapeutiques dans le myocarde, et il peut être utilisé pour un apport de gènes destiné à induire l'angiogenèse, à inhiber l'angiogenèse, à stimuler la prolifération cellulaire, à inhiber la prolifération cellulaire et/ou à traiter ou à soulager d'autres troubles cardiovasculaires.
PCT/US1999/031093 1998-12-28 1999-12-28 Transfert efficace et stable in vivo de genes dans des cardiomyocytes a l'aide de vecteurs viraux adeno-associes de recombinaison WO2000038518A1 (fr)

Priority Applications (4)

Application Number Priority Date Filing Date Title
CA002356551A CA2356551A1 (fr) 1998-12-28 1999-12-28 Transfert efficace et stable in vivo de genes dans des cardiomyocytes a l'aide de vecteurs viraux adeno-associes de recombinaison
AU23942/00A AU763049B2 (en) 1998-12-28 1999-12-28 Efficient and stable (in vivo) gene transfer to cardiomyocytes using recombinantadeno-associated virus vectors
EP99967703A EP1139751A4 (fr) 1998-12-28 1999-12-28 TRANSFERT EFFICACE ET STABLE $i(IN VIVO) DE GENES DANS DES CARDIOMYOCYTES A L'AIDE DE VECTEURS VIRAUX ADENO-ASSOCIES DE RECOMBINAISON
JP2000590483A JP2002533359A (ja) 1998-12-28 1999-12-28 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

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US11392398P 1998-12-28 1998-12-28
US60/113,923 1998-12-28

Publications (2)

Publication Number Publication Date
WO2000038518A1 true WO2000038518A1 (fr) 2000-07-06
WO2000038518A9 WO2000038518A9 (fr) 2002-04-11

Family

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Application Number Title Priority Date Filing Date
PCT/US1999/031093 WO2000038518A1 (fr) 1998-12-28 1999-12-28 Transfert efficace et stable in vivo de genes dans des cardiomyocytes a l'aide de vecteurs viraux adeno-associes de recombinaison

Country Status (5)

Country Link
EP (1) EP1139751A4 (fr)
JP (1) JP2002533359A (fr)
AU (1) AU763049B2 (fr)
CA (1) CA2356551A1 (fr)
WO (1) WO2000038518A1 (fr)

Cited By (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001034208A1 (fr) * 1999-11-05 2001-05-17 The Regents Of The University Of California Techniques et compositions permettant de traiter les maladies cardiovasculaires par l'administration de genes in vivo
WO2002014487A2 (fr) * 2000-08-17 2002-02-21 Keiya Ozawa Distribution de facteurs angiogeniques a mediation assuree par virus associe aux adenovirus
WO2002098432A1 (fr) * 2001-06-01 2002-12-12 The Brigham And Women's Hospital, Inc. Procedes de traitement de troubles cardiaques
EP1324780A2 (fr) * 2000-09-06 2003-07-09 Johns Hopkins University Procedes de traitement de l'arrhythmie cardiaque
WO2004062618A3 (fr) * 2003-01-13 2004-12-09 Univ California Procede de regulation in vivo de la contractilite du muscle cardiaque
EP1870473A1 (fr) * 2000-08-17 2007-12-26 Keiya Ozawa Libération de facteurs angiogéniques à médiation virale et adéno associée
US20080076730A1 (en) * 2006-07-25 2008-03-27 Celladon Corporation Extended antegrade epicardial coronary infusion of adeno-associated viral vectors for gene therapy
US8003618B2 (en) 2002-10-02 2011-08-23 The John Hopkins University Focal calcium channel modulation
US8221738B2 (en) 2008-02-19 2012-07-17 Celladon Corporation Method for enhanced uptake of viral vectors in the myocardium
WO2012162705A3 (fr) * 2011-05-26 2013-02-28 University Of Washington Procédés basés sur des cellules et des gènes permettant d'améliorer la fonction cardiaque
WO2019097001A1 (fr) * 2017-11-16 2019-05-23 Universite D'aix-Marseille Fgf10 pour le traitement de cardiopathies

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070134204A1 (en) * 2005-12-09 2007-06-14 Henrich Cheng Method for treating nerve injury and vector construct for the same

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5792483A (en) * 1993-04-05 1998-08-11 Vickers, Inc. Injection molding machine with an electric drive
US6162796A (en) * 1995-09-27 2000-12-19 The Rockefeller University Method for transferring genes to the heart using AAV vectors

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
ALEXANDER ET AL.: "Gene Transfer and Models of Gene Therapy for the Myocardium", CLINICAL AND EXPERIMENTAL PHARMACOLOGY AND PHYSIOLOGY, vol. 26, no. 9, September 1999 (1999-09-01), pages 661 - 668, XP002925647 *
GNATENKO ET AL.: "Characterization of Recombinant Adeno-Associated Virus-1 as a Vehicle for Gene Delivery and expression into Vascular Cells", JOURNAL OF INVESTIGATIVE MEDICINE, vol. 45, no. 2, February 1997 (1997-02-01), pages 87 - 98, XP002925645 *
KAPLITT ET AL.: "Long-Term Gene Transfer in Porcine Myocardium After Coronary Infusion of an Adeno-Associated Virus Vector", ANNALS OF THORACIC SURGERY, vol. 62, no. 6, December 1996 (1996-12-01), pages 1669 - 1676, XP002925644 *
See also references of EP1139751A4 *
SVENSSON ET AL.: "Efficient and Stable Transduction of Cardiomyocytes After Intramyocardial Injection of Intracoronary Perfusion with Recombinant Adeno-Associated Virus Vectors", CIRCULATION, vol. 99, no. 2, 19 January 1999 (1999-01-19), pages 201 - 205, XP002925646 *

Cited By (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7745416B2 (en) * 1995-04-11 2010-06-29 The Regents Of The University Of California Method for in vivo regulation of cardiac muscle contractility
EA008538B1 (ru) * 1999-11-05 2007-06-29 Те Риджентс Оф Те Юниверсити Оф Калифорния Способы и композиции для лечения сердечно-сосудистого заболевания доставкой генов in vivo
WO2001034208A1 (fr) * 1999-11-05 2001-05-17 The Regents Of The University Of California Techniques et compositions permettant de traiter les maladies cardiovasculaires par l'administration de genes in vivo
WO2002014487A2 (fr) * 2000-08-17 2002-02-21 Keiya Ozawa Distribution de facteurs angiogeniques a mediation assuree par virus associe aux adenovirus
WO2002014487A3 (fr) * 2000-08-17 2003-03-13 Keiya Ozawa Distribution de facteurs angiogeniques a mediation assuree par virus associe aux adenovirus
EP1870473A1 (fr) * 2000-08-17 2007-12-26 Keiya Ozawa Libération de facteurs angiogéniques à médiation virale et adéno associée
JP2004506658A (ja) * 2000-08-17 2004-03-04 小澤　敬也 アデノ随伴ウイルスを介した血管形成因子のデリバリー
JP2004533405A (ja) * 2000-09-06 2004-11-04 ジョンズ・ホプキンス・ユニバーシティ 心不整脈治療法
US7034008B2 (en) 2000-09-06 2006-04-25 The Johns Hopkins University Cardiac arrhythmia treatment methods
EP2363152A1 (fr) * 2000-09-06 2011-09-07 Johns Hopkins University Procédés de traitements de l'arythmie cardiaque
EP1324780A4 (fr) * 2000-09-06 2005-06-22 Univ Johns Hopkins Procedes de traitement de l'arrhythmie cardiaque
EP1324780A2 (fr) * 2000-09-06 2003-07-09 Johns Hopkins University Procedes de traitement de l'arrhythmie cardiaque
WO2002098432A1 (fr) * 2001-06-01 2002-12-12 The Brigham And Women's Hospital, Inc. Procedes de traitement de troubles cardiaques
US8003618B2 (en) 2002-10-02 2011-08-23 The John Hopkins University Focal calcium channel modulation
AU2004204815B2 (en) * 2003-01-13 2010-03-11 The Regents Of The University Of California Method for in vivo regulation of cardiac muscle contractility
WO2004062618A3 (fr) * 2003-01-13 2004-12-09 Univ California Procede de regulation in vivo de la contractilite du muscle cardiaque
JP2006518996A (ja) * 2003-01-13 2006-08-24 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 心筋収縮性をｉｎｖｉｖｏで調節するための方法
US20080076730A1 (en) * 2006-07-25 2008-03-27 Celladon Corporation Extended antegrade epicardial coronary infusion of adeno-associated viral vectors for gene therapy
EP2460879A1 (fr) 2006-07-25 2012-06-06 Celladon Corporation Transfusion coronaire épicardique antérograde prolongée de vecteurs viraux associés à l'adénovirus pour thérapie génique
US8221738B2 (en) 2008-02-19 2012-07-17 Celladon Corporation Method for enhanced uptake of viral vectors in the myocardium
US8636998B2 (en) 2008-02-19 2014-01-28 Celladon Corporation Method for enhanced uptake of viral vectors in the myocardium
WO2012162705A3 (fr) * 2011-05-26 2013-02-28 University Of Washington Procédés basés sur des cellules et des gènes permettant d'améliorer la fonction cardiaque
US9868937B2 (en) 2011-05-26 2018-01-16 University Of Washington Cell and gene based methods to improve cardiac function
WO2019097001A1 (fr) * 2017-11-16 2019-05-23 Universite D'aix-Marseille Fgf10 pour le traitement de cardiopathies

Also Published As

Publication number Publication date
EP1139751A4 (fr) 2003-03-19
WO2000038518A9 (fr) 2002-04-11
JP2002533359A (ja) 2002-10-08
EP1139751A1 (fr) 2001-10-10
AU2394200A (en) 2000-07-31
AU763049B2 (en) 2003-07-10
CA2356551A1 (fr) 2000-07-06

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