Journal
JOURNAL OF GENE MEDICINE
Volume 13, Issue 10, Pages 557-565Publisher
WILEY-BLACKWELL
DOI: 10.1002/jgm.1600
Keywords
AAV; adenovirus; adeno-associated virus; heart gene delivery; lentivirus; nonviral vector; viral vector
Categories
Funding
- National Institutes of Health [HL-91883, AR-49419]
- Muscular Dystrophy Association
- Parent Project Muscular Dystrophy
- Jesse's Journey: The Foundation for Gene and Cell Therapy
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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 (C) 2011 John Wiley & Sons, Ltd.
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