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Gene therapy for cardiovascular disease.
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- Abstract:
The article discusses several gene therapies for heart diseases. Topics discussed include gene editing therapy for familial hypercholesterolemia which turns off the PCSK9 gene in the liver, a gene therapy trial in patients with hypertrophic cardiomyopathy which uses a virus to deliver a working copy of the MYBPC3 gene to the heart muscle, and gene editing to stop the production of abnormal transthyretin protein in transthyretin amyloid cardiomyopathy.
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