scAAVrh10.cTnT.S100A1

scAAVrh10.cTnT.S100A1 is an investigational gene therapy candidate designed to treat cardiomyopathy, particularly the cardiac complications associated with Duchenne muscular dystrophy (DMD). The therapy utilizes a self-complementary adeno-associated virus serotype rh10 (scAAVrh10) vector to deliver the human S100A1 transgene. Expression is controlled by the cardiac-specific troponin T (cTnT) promoter to ensure targeted delivery to cardiomyocytes. S100A1 is a calcium-binding protein that acts as a master regulator of cardiac contractility and calcium handling; its downregulation is a hallmark of heart failure and DMD-associated cardiomyopathy. By overexpressing S100A1, the therapy aims to restore calcium homeostasis, improve diastolic function, and prevent the progression of heart failure in DMD patients.

Other names

S100A1 gene therapyS-100A1 gene therapyS 100A1 gene therapyAAVrh10-cTnT-S100A1AAVrh-10-cTnT-S100A1AAVrh 10-cTnT-S100A1