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AAVMYO-CBH-hHSPB8
- Development stage
- Preclinical
- Lead developer
- University of California, Irvine
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAVMYO-CBH-hHSPB8 is a preclinical gene therapy construct designed for the treatment of HSPB8-associated autosomal dominant rimmed vacuolar myopathy. It utilizes a myotropic adeno-associated virus (AAVmyo) vector to deliver a wild-type human heat shock protein family B (small) member 8 (hHSPB8) gene under the control of a chicken beta-actin hybrid (CBH) promoter. The therapy aims to restore normal HSPB8 protein levels and ameliorate muscle weakness, pathology, and protein aggregation (such as TDP-43 and amyloid fibrils) associated with frameshift mutations in the HSPB8 gene.
- Other names
- AAVmyo-CBH-hHSPB8AAVmyo-CBH-hHSPB-8AAVmyo-CBH-hHSPB 8
02
Targets
HSP70 (Heat shock protein 70)
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