biostudies-literatureUnknown28(1)Cacheux MCentre National de la Recherche ScientifiqueInstitut National de la Santé et de la Recherche MédicaleAssociation Française contre les MyopathiesAFM-TéléthonRecessive forms of catecholaminergic polymorphic ventricular tachycardia (CPVT) are induced by mutations in genes encoding triadin or calsequestrin, two proteins that belong to the Ca²⁺ release complex, responsible for intracellular Ca²⁺ release triggering cardiac contractions. To better understand the mechanisms of triadin-induced CPVT and to assay multiple therapeutic interventions, we used a triadin knockout mouse model presenting a CPVT-like phenotype associated with a decrease in calsequestrin protein level. We assessed different approaches to rescue protein expression and to correct intracellular Ca²⁺ release and cardiac function: pharmacological treatment with kifunensine or a viral gene transfer-based approach, using adeno-associated virus serotype Molecular therapy : the journal of the American Society of Gene Therapy171-179https://www.ebi.ac.uk/biostudies/studies/S-EPMC6952166biostudies-literatureInterplay between Triadin and Calsequestrin in the Pathogenesis of CPVT in the Mouse.PMC6952166Cacheux MBrocard JFauconnier JThireau JFaure JOsseni ARoux-Buisson NLacampagne AMarty IfalseInterplay between Triadin and Calsequestrin in the Pathogenesis of CPVT in the Mouse.Recessive forms of catecholaminergic polymorphic ventricular tachycardia (CPVT) are induced by mutations in genes encoding triadin or calsequestrin, two proteins that belong to the Ca²⁺ release complex, responsible for intracellular Ca²⁺ release triggering cardiac contractions. To better understand the mechanisms of triadin-induced CPVT and to assay multiple therapeutic interventions, we used a triadin knockout mouse model presenting a CPVT-like phenotype associated with a decrease in calsequestrin protein level. We assessed different approaches to rescue protein expression and to correct intracellular Ca²⁺ release and cardiac function: pharmacological treatment with kifunensine or a viral gene transfer-based approach, using adeno-associated virus serotype 2020-01-01T00:00:00Z2020 Jan2026-04-18T19:08:22.179Z2025-05-29T20:01:41.217ZS-EPMC69521663160754210.1016/j.ymthe.2019.09.012