Heart failure & cardiomyopathies
Gene Therapy in Cardiovascular Disease: Recent Advances and Future Directions in Science: A Science Advisory From the American Heart Association
Scope
As issued by the authors
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
Original publication
Gene Therapy in Cardiovascular Disease: Recent Advances and Future Directions in Science: A Science Advisory From the American Heart Association. Circulation. Published 11 November 2024. doi:10. 1161/ cir. 0000000000001296
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