Why the study?
Currently available therapies for inherited arrhythmias offer incomplete protection from arrhythmia and fail to alter disease progression, whereas gene therapies may provide potent, molecularly targeted options.
Gene therapy represents a promising, molecularly targeted approach for treating inherited arrhythmias such as CPVT and hypertrophic cardiomyopathy.
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Gene therapy for inherited arrhythmias remains investigational; leaves open the need for randomized trials to confirm safety and efficacy.
Inherited arrhythmias are disorders caused by one or more genetic mutations that increase the risk of arrhythmia, which result in life-long risk of sudden death. These mutations either primarily perturb electrophysiological homeostasis (e.g. long QT syndrome and catecholaminergic polymorphic ventricular tachycardia), cause structural disease that is closely associated with severe arrhythmias (e.g. hypertrophic cardiomyopathy), or cause a high propensity for arrhythmia in combination with altered myocardial structure and function (e.g. arrhythmogenic cardiomyopathy). Currently available therapies offer incomplete protection from arrhythmia and fail to alter disease progression. Recent studies suggest that gene therapies may provide potent, molecularly targeted options for at least a subset of inherited arrhythmias. Here, we provide an overview of gene therapy strategies, and review recent studies on gene therapies for catecholaminergic polymorphic ventricular tachycardia and hypertrophic cardiomyopathy caused by MYBPC3 mutations.
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Bezzerides et al. (2020) studied this question.
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