PRJNA483082
In vivo Ryr2 editing corrects catecholaminergic polymorphic ventricular tachycardia
Catecholaminergic polymorphic ventricular tachycardia (CPVT) is a lethal inherited arrhythmia disorder most commonly caused by missense mutations in the RyR2 gene. The goal of this study was to determine whether somatic in vivo gene editing using the CRISPR/Cas9 system delivered by adeno-associated viral (AAV) vectors could correct CPVT arrhythmias in mice heterozygous for RyR2 mutation R176Q (R176Q/+).