scAAVrh10.cTnT.ARC

scAAVrh10.cTnT.ARC is an experimental gene therapy construct designed to treat cardiomyopathy, specifically in patients with Duchenne muscular dystrophy (DMD). It utilizes a self-complementary adeno-associated virus serotype rh10 (scAAVrh10) vector to deliver the cDNA for the Apoptosis Repressor with Caspase recruitment domains (ARC). The expression of ARC is driven by the cardiac-specific Troponin T (cTnT) promoter, ensuring targeted delivery to heart tissue. ARC is a multifunctional protein that inhibits both the extrinsic and intrinsic apoptotic pathways by binding to and neutralizing pro-apoptotic factors such as BAX and caspases. By preventing cardiomyocyte loss and reducing cardiac fibrosis, this therapy aims to preserve heart function and extend the lifespan of individuals with DMD. Preclinical studies in mouse and canine models have demonstrated its potential to improve cardiac outcomes and mitigate heart failure.

Other names

AAVrh10-cTnT-ARCAAVrh-10-cTnT-ARCAAVrh 10-cTnT-ARCscAAVrh10-ARCscAAVrh-10-ARCscAAVrh 10-ARC