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. Author manuscript; available in PMC: 2012 Oct 1.
Published in final edited form as: J Gene Med. 2011 Oct;13(10):557–565. doi: 10.1002/jgm.1600

Table 1.

Gene transfer vectors used in heart gene therapy

Vector for gene delivery Delivery methods Length of gene expression Advantages Disadvantages References
Nonviral
    Plasmids Intramyocardial 28–60 days Low cost, simplicity in design Restricted transduction, short-term expression 112
    DNA–lipid complexes Intravenous Ability to deliver systemically Limited myocardial transduction, off-target expression 1317
    Oligonucleotides Intravenous 2 weeks to 3 months Restore expression at mRNA level Repeated administration, short half-life, patient-specific design 1819
Viral
    Lentivirus Intramyocardial, coronary arterial 10 weeks to 6 months Ability to package up to 8 kb, persistent expression upon integration to the host genome Myocardial inflammation, insertional mutagenesis 2124
    Adenovirus Intramyocardial, intrapericardial, coronary arterial, intravenous 1–8 weeks Large packaging capacity (up to 35 kb for gutted adenovirus), efficient cardiomyocyte transduction Myocardial inflammation, cellular immune responses 2541
    Adeno-associated virus Intramyocardial, intrapericardial, coronary arterial, intravenous 5 weeks to 12 months Efficient cardiomyocyte transduction, persistent expression Limited packaging capacity 4274