Gene therapy for inherited arrhythmias
Standard
Gene therapy for inherited arrhythmias. / Bezzerides, Vassilios J; Prondzynski, Maksymilian; Carrier, Lucie; Pu, William T.
In: CARDIOVASC RES, Vol. 116, No. 9, 15.07.2020, p. 1635-1650.Research output: SCORING: Contribution to journal › SCORING: Review article › Research
Harvard
APA
Vancouver
Bibtex
}
RIS
TY - JOUR
T1 - Gene therapy for inherited arrhythmias
AU - Bezzerides, Vassilios J
AU - Prondzynski, Maksymilian
AU - Carrier, Lucie
AU - Pu, William T
N1 - Published on behalf of the European Society of Cardiology. All rights reserved. © The Author(s) 2020. For permissions, please email: journals.permissions@oup.com.
PY - 2020/7/15
Y1 - 2020/7/15
N2 - 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.
AB - 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.
U2 - 10.1093/cvr/cvaa107
DO - 10.1093/cvr/cvaa107
M3 - SCORING: Review article
C2 - 32321160
VL - 116
SP - 1635
EP - 1650
JO - CARDIOVASC RES
JF - CARDIOVASC RES
SN - 0008-6363
IS - 9
ER -