Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translation.

ABSTRACT: Gene therapy is a promising modality for the treatment of inherited and acquired cardiovascular diseases. The identification of the molecular pathways involved in the pathophysiology of heart failure and other associated cardiac diseases led to encouraging preclinical gene therapy studies in small and large animal models. However, the initial clinical results yielded only modest or no improvement in clinical endpoints. The presence of neutralizing antibodies and cellular immune responses directed against the viral vector and/or the gene-modified cells, the insufficient gene expression levels, and the limited gene transduction efficiencies accounted for the overall limited clinical improvements. Nevertheless, further improvements of the gene delivery technology and a better understanding of

SUBMITTER: Rincon MY 

PROVIDER: S-EPMC4571836 | biostudies-literature | 2015 Oct

REPOSITORIES: biostudies-literature