State of Gene Therapy for Monogenic Cardiovascular Diseases.

Bains, Sahej; Giudicessi, John R; Odening, Katja E; Ackerman, Michael J (2024). State of Gene Therapy for Monogenic Cardiovascular Diseases. Mayo Clinic proceedings, 99(4), pp. 610-629. Elsevier 10.1016/j.mayocp.2023.11.003

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Over the past 2 decades, significant efforts have been made to advance gene therapy into clinical practice. Although successful examples exist in other fields, gene therapy for the treatment of monogenic cardiovascular diseases lags behind. In this review, we (1) highlight a brief history of gene therapy, (2) distinguish between gene silencing, gene replacement, and gene editing technologies, (3) discuss vector modalities used in the field with a special focus on adeno-associated viruses, (4) provide examples of gene therapy approaches in cardiomyopathies, channelopathies, and familial hypercholesterolemia, and (5) present current challenges and limitations in the gene therapy field.

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