AAV9-DYSF-DV3'

AAV9-DYSF-DV3' is an investigational gene therapy being developed by Artgen for the treatment of dysferlinopathy, which includes Limb-Girdle Muscular Dystrophy Type 2B (LGMD2B) and Miyoshi Myopathy. Due to the large size of the dysferlin (DYSF) gene, which exceeds the standard packaging capacity of a single adeno-associated virus (AAV) vector, this therapy utilizes a dual-vector system based on AAV serotype 9 (AAV9). The system delivers a codon-optimized version of the DYSF gene to muscle cells, where the two vector components undergo recombination to produce a full-length, functional dysferlin protein. This protein is essential for muscle membrane repair and maintenance; its restoration is intended to halt the progressive muscle weakness and degeneration characteristic of the disease. Preclinical studies have demonstrated the efficacy and safety of this approach in animal models.

Other names

Artgen dysferlin gene therapyGT DF