Adeno-associated virus-mediated gene therapy in cardiovascular disease. (May 2015)

• Record Type: Journal Article
• Authors: Hammoudi, Nadjib; Ishikawa, Kiyotake; Hajjar, Roger J.
• Is Part Of: Current opinion in cardiology. Volume 30:Number 3(2015:May)
• Publication Date: 2015-05
• Subjects: adeno-associated virus -- arrhythmias -- gene therapy -- heart failure -- sarcoplasmic reticulum calcium ATPase pump; Cardiology -- Periodicals; Cardiology -- Periodicals; Heart Diseases -- Periodicals; Cardiology; Periodicals; 616.12
• DOI: 10.1097/HCO.0000000000000159
• Languages: English
• ISSNs: 0268-4705

Abstract

Abstract : 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.