Journal
CURRENT OPINION IN CARDIOLOGY
Volume 32, Issue 3, Pages 275-282Publisher
LIPPINCOTT WILLIAMS & WILKINS
DOI: 10.1097/HCO.0000000000000386
Keywords
adeno-associated virus; gene therapy; heart failure; inherited cardiac diseases
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Funding
- Trans-Atlantic Network of Excellence grant from the Leducq Foundation [14CVD03]
- [R01 HL131404]
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Purpose of review Cardiac gene therapy with adeno-associated virus (AAV)-based vectors is emerging as an entirely new platform to treat, or even cure, so far intractable cardiac disorders. This review describes our current knowledge of cardiac AAV gene therapy with a particular focus on the biggest obstacle for the successful translation of cardiac AAV gene therapy into the clinic, namely the efficient delivery of the therapeutic gene to the myocardium. Recent findings We summarize the significant recent progress that has been made in treating heart failure in preclinically relevant animal models with AAV gene therapy and the recent results of clinical trials with cardiac AAV gene therapy for the treatment of heart failure. We also discuss the benefits and shortcomings of the currently available delivery methods of AAV to the heart. Finally, we describe the current state of identifying novel AAV variants that have enhanced tropism for human cardiomyocytes and that show increased resistance to preexisting neutralizing antibodies. Summary Here, we describe the successes and challenges in cardiac AAV gene therapy, a treatment modality that has the potential to transform current treatment approaches for cardiac diseases.
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