S100A1-ARC gene therapy

**S100A1-ARC gene therapy** is an investigational adeno-associated virus gene-augmentation therapy developed at the University of Florida for Duchenne muscular dystrophy cardiomyopathy. A single bicistronic AAV vector delivers transgenes encoding S100 calcium-binding protein A1 and apoptosis repressor with caspase recruitment domain to striated muscle. In D2.mdx mice, combined expression improved long-term cardiac outcomes and skeletal-muscle function; intracoronary administration was assessed for safety in a canine Duchenne muscular dystrophy model. ([insight.jci.org](https://insight.jci.org/articles/view/204852))

Other names

Dual S100A1 and ARC gene therapyS100A1-ARC gene therapyS-100A1-ARC gene therapyS 100A1-ARC gene therapyS100A1-ARC therapyS-100A1-ARC therapyS 100A1-ARC therapyS100A1/ARC gene therapy