Yuriana Aguilar-Sanchez

1 month ago | digitalcommons.library.tmc.edu | Oliver Moore |Yuriana Aguilar-Sanchez |Satadru K. Lahiri |Mohit Hulsurkar

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.