Scientists have shown that replacing a dysfunctional gene can slow the development of arrhythmogenic right ventricular cardiomyopathy, a life-threatening inherited disease in which the muscle walls of the heart gradually weaken. The new gene therapy is reported in an article published in Circulation: Genomic and Precision Medicine.
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Arrhythmogenic right ventricular cardiomyopathy results from dysfunction of the plakophilin-2 (PKP2) gene, which encodes a structural protein that binds heart tissue together. As a result, fibrotic and fatty tissue builds up in the walls of the heart, causing an irregular heartbeat and even increasing the risk of cardiac arrest. Current therapies help restore normal rhythm, but are unable to treat the cause of the disease.
