Gene Therapy in Cardiovascular Disease: Recent Advances and Future Directions in Science: A Science Advisory From the American Heart Association

Gene therapy in CVD: core concepts (gene supplementation, genome editing), delivery vectors (adeno-associated viral, lipid and engineered nanoparticles), key obstacles (durability, tissue targeting, vector-associated adverse events, off-target effects), patient risks and ongoing research, for monogenic disorders such as hypertrophic, dilated and arrhythmogenic cardiomyopathies and hypercholesterolemia