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scAAVrh10.CK8e.S100A1-ARC
- Development stage
- Preclinical
- Lead developer
- University of Florida
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
**scAAVrh10.CK8e.S100A1-ARC** is an investigational, self-complementary adeno-associated virus rh10 gene therapy designed for striated-muscle expression of the calcium-binding protein S100A1 and apoptosis repressor with caspase recruitment domain ARC from a single bicistronic cassette under the CK8e promoter. It is being studied preclinically as a treatment for Duchenne muscular dystrophy cardiomyopathy. In D2.mdx mouse studies, combined S100A1 and ARC expression improved long-term cardiac outcomes and skeletal-muscle function; the broader S100A1-ARC program also showed safety evidence following intracoronary delivery in dystrophic dogs. This CK8e-promoter construct is distinct from related cTnT-promoter S100A1-ARC vectors evaluated for cardiac-selective expression.
- Other names
- scAAVrh10.CK8e.S100A1-ARCscAAVrh-10.CK8e.S100A1-ARCscAAVrh 10.CK8e.S100A1-ARC
02
Targets
pro-CASP3 (Procaspase-3)S100A1 (S100 calcium-binding protein A1)
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