Purpose of review
The use of
adeno-associated virus (AAV) as an efficient, cardiotropic, and safe vector, coupled with the identification of key molecular targets, has placed gene-based therapies within reach of cardiovascular diseases. The purpose of this review is to provide a focused update on the current advances related to AAV-mediated
gene therapy in cardiovascular diseases, and particularly in
heart failure (HF), wherein
gene therapy has recently made important progress.
Recent findings
Multiple successful preclinical studies suggest a potential utility of AAV
gene therapy for
arrhythmias and biological heart pacing, as well as RNA overexpression. Moreover, AAV-mediated overexpression of several molecular targets involved in HF has demonstrated promising results in clinically relevant large animal models. In humans, a safe and successful completion of a phase 2 clinical trial targeting the
sarcoplasmic reticulum calcium ATPase pump with AAV has been reported. Serial studies are ongoing to further prove the efficacy of AAV-mediated
sarcoplasmic reticulum calcium ATPase pump gene transfer in human HF.
Summary
Significant progress in clinical translation of AAV-mediated cardiac
gene therapy has been achieved in recent years. This will prompt further clinical trials, and positive results could open a new era for cardiac
gene therapy.
