The evolution of heart gene delivery vectors

Gene therapy holds promise for treating numerous heart diseases. A key premise for the success of cardiac gene therapy is the development of powerful gene transfer vehicles that can achieve highly efficient and persistent gene transfer specifically in the heart. Other features of an ideal vector inc...

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Bibliographic Details
Published inThe journal of gene medicine Vol. 13; no. 10; pp. 557 - 565
Main Authors Wasala, Nalinda B., Shin, Jin-Hong, Duan, Dongsheng
Format Journal Article
LanguageEnglish
Published Chichester, UK John Wiley & Sons, Ltd 01.10.2011
Wiley Periodicals Inc
Subjects
AAV
Adeno-associated virus
adenovirus
Animal models
Animals
Clinical Trials as Topic
Gene therapy
Gene Transfer Techniques
Genetic Therapy - methods
Genetic Vectors - genetics
Genetic Vectors - metabolism
Heart - physiology
Heart Diseases - therapy
heart gene delivery
Humans
lentivirus
nonviral vector
viral vector
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Summary:Gene therapy holds promise for treating numerous heart diseases. A key premise for the success of cardiac gene therapy is the development of powerful gene transfer vehicles that can achieve highly efficient and persistent gene transfer specifically in the heart. Other features of an ideal vector include negligible toxicity, minimal immunogenicity and easy manufacturing. Rapid progress in the fields of molecular biology and virology has offered great opportunities to engineer various genetic materials for heart gene delivery. Several nonviral vectors (e.g. naked plasmids, plasmid lipid/polymer complexes and oligonucleotides) have been tested. Commonly used viral vectors include lentivirus, adenovirus and adeno‐associated virus. Among these, adeno‐associated virus has shown many attractive features for pre‐clinical experimentation in animal models of heart diseases. We review the history and evolution of these vectors for heart gene transfer. Copyright © 2011 John Wiley & Sons, Ltd.
Bibliography:ArticleID:JGM1600
istex:5FF37FC8BF96FBA95681ADDB243DC71DA9D1B9BC
ark:/67375/WNG-TT2B1TCZ-5
National Institutes of Health HL-91883 and AR-49419 (DD), Muscular Dystrophy Association (DD), Parent Project Muscular Dystrophy (DD) and Jesse's Journey: The Foundation for Gene and Cell Therapy (DD)
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ISSN:1099-498X
1521-2254
1521-2254
DOI:10.1002/jgm.1600