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The recessive form of catecholaminergic polymorphic ventricular tachycardia 2 (CPVT2) is caused by mutations in cardiac calsequestrin (CASQ2), leading to protein deficiency.The aims of this study were to develop a viral-delivered gene therapy for CPVT2 and to determine the relationship between CASQ2 expression and antiarrhythmic efficacy in a murine model.We used a murine model of CPVT2 caused by...
Cardiomyopathies arising due to a single gene defect represent various pathways that evoke adverse remodeling and cardiac dysfunction. While the gene therapy approach is slowly evolving and has not yet reached clinical “prime time” and gene correction approaches are applicable at the bench but not at the bedside, major advances are being made with molecular and drug therapies. This review summarizes...
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