Long-term efficacy and safety of cardiac genome editing for catecholaminergic polymorphic ventricular tachycardia
Oliver M. Moore,
Yuriana Aguilar-Sanchez,
Satadru K. Lahiri
et al.
Abstract:Introduction: Heterozygous autosomal-dominant single nucleotide variants in RYR2 account for 60% of cases of catecholaminergic polymorphic ventricular tachycardia (CPVT), an inherited arrhythmia disorder associated with high mortality rates. CRISPR/Cas9-mediated genome editing is a promising therapeutic approach that can permanently cure the disease by removing the mutant RYR2 allele. However, the safety and long-term efficacy of this strategy have not been established in a relevant disease model.
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