Article
Single Construct Suppression and Replacement Gene Therapy for the Treatment of All CALM1-, CALM2-, and CALM3-Mediated Arrhythmia Disorders.
Circulation. Arrhythmia and electrophysiology - 1 Aug 2024
Hamrick Samantha K, Kim C S John, Tester David J, Gencarelli Manuela, Tobert Kathryn E, Gluscevic Martina, Ackerman Michael J
Abstract excerpt
BACKGROUND: CaM (calmodulin)-mediated long-QT syndrome is a genetic arrhythmia disorder (calmodulinopathies) characterized by a high prevalence of life-threatening ventricular arrhythmias occurring early in life. Three distinct genes (CALM1, CALM2, and CALM3) encode for the identical CaM protein. Conventional pharmacotherapies fail to adequately protect against potentially lethal cardiac events in patients with...
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