Heart Health
Gene therapy for cardiovascular disease
This trailblazing technique is being tested in people with inherited forms of heart disease, but challenges remain.
- Reviewed by Christopher P. Cannon, MD, Editor in Chief, Harvard Heart Letter; Editorial Advisory Board Member, Harvard Health Publishing
Over the past decade, the Nobel prize-winning technology known as CRISPR has transformed biomedical research, enabling scientists to edit DNA far more easily and precisely than ever before. In late 2023, the first medical use of the gene editing tool was approved to treat sickle cell disease, an inherited blood disorder that causes excruciating pain, organ damage, and strokes.
Investigators are also using gene editing to develop novel therapies for several types of heart disease. Clinical trials are currently under way among people with three genetic conditions: abnormally high cholesterol; a type of heart muscle disease; and a form of heart failure that results from amyloid deposits. Preliminary results in small numbers of people appear promising.
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About the Author
Julie Corliss, Executive Editor, Harvard Heart Letter
About the Reviewer
Christopher P. Cannon, MD, Editor in Chief, Harvard Heart Letter; Editorial Advisory Board Member, Harvard Health Publishing
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