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S100A1-ARC gene therapy
- Development stage
- Preclinical
- Lead developer
- University of Florida
- Modality
- Gene Therapies
- Administration
- Intra-arterial
01
Overview
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)
- 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
02
Targets
pro-CASP3 (Procaspase-3)S100A1 (S100 calcium-binding protein A1)ARC (Apoptosis repressor with caspase recruitment domain)
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