S100A1-ARC AAV gene therapy

S100A1-ARC AAV gene therapy is a preclinical, research-stage gene therapy designed to treat Duchenne muscular dystrophy (DMD) cardiomyopathy. Developed at the University of Florida, the therapy utilizes an adeno-associated virus (AAV) vector to deliver a bicistronic construct encoding two therapeutic proteins: S100A1 and ARC (Apoptosis Repressor with Caspase Recruitment Domain). S100A1 is a calcium-binding protein that enhances diastolic function in failing cardiomyocytes, while ARC is an anti-apoptotic protein that promotes cell survival. Preclinical studies in mouse (D2.mdx) and canine (GRMD) models have demonstrated that this combination improves long-term cardiac outcomes, prevents heart failure associated with micro-dystrophin expression, and provides functional benefits in skeletal muscle.

Other names

S100A1-ARC gene therapyS-100A1-ARC gene therapyS 100A1-ARC gene therapybicistronic S100A1-ARC AAV