Gene therapy for inherited arrhythmias.

Abstract:

: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.

journal_name

Cardiovasc Res

journal_title

Cardiovascular research

authors

Bezzerides VJ,Prondzynski M,Carrier L,Pu WT

doi

10.1093/cvr/cvaa107

subject

Has Abstract

pub_date

2020-07-15 00:00:00

pages

1635-1650

issue

9

eissn

0008-6363

issn

1755-3245

pii

5823887

journal_volume

116

pub_type

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