Key result
Cardiac gene therapy for heart failure requires optimizing viral vectors and delivery strategies to overcome barriers to transduction and improve expression in cardiomyocytes.
This review outlines the barriers to cardiac gene therapy and discusses methodologies to improve viral transduction and gene expression in the heart.
Gene therapy for HF remains investigational; review leaves open clinical translation pending optimized vectors in trials.
Gene therapy strategies are becoming a valuable approach for the treatment of heart failure. Some trials are ongoing and others are being organized. Vascular access in clinical experimentation is still the chosen modality of delivery, but many other approaches are in research and development. A successful gene therapy strategy involves not only the choice of the right vector and gene, but also the correct delivery strategy that allows for transduction of the highest percentage of cardiomyocytes, limited spilling of virus into other organs and the possibility to correlate the amount of injected virus to the rate of the expression within the cardiac tissue. The authors will first concentrate on clarifying what the barriers are that the virus has to overcome in order to reach the nuclei of the target organs and methodologies that have been tested to improve the range of expression.
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Scimia et al. (2014) conducted a review in Heart failure. Cardiac gene therapy was evaluated. Cardiac gene therapy for heart failure requires optimizing viral vectors and delivery strategies to overcome barriers to transduction and improve expression in cardiomyocytes.
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