Ontology highlight
ABSTRACT:
SUBMITTER: Lodola F
PROVIDER: S-EPMC5133973 | biostudies-literature | 2016 Oct
REPOSITORIES: biostudies-literature
Lodola Francesco F Morone Diego D Denegri Marco M Bongianino Rossana R Nakahama Hiroko H Rutigliano Lucia L Gosetti Rosanna R Rizzo Giulia G Vollero Alessandra A Buonocore Michelangelo M Napolitano Carlo C Condorelli Gianluigi G Priori Silvia G SG Di Pasquale Elisa E
Cell death & disease 20161006 10
Catecholaminergic Polymorphic Ventricular Tachycardia type 2 (CPVT2) is a highly lethal recessive arrhythmogenic disease caused by mutations in the calsequestrin-2 (CASQ2) gene. We have previously demonstrated that viral transfer of the wild-type (WT) CASQ2 gene prevents the development of CPVT2 in a genetically induced mouse model of the disease homozygous carrier of the R33Q mutation. In the present study, we investigated the efficacy of the virally mediated gene therapy in cardiomyocytes (CMs ...[more]