GT DF 102

GT DF 102 is an investigational gene therapy being developed by Artgen (formerly known as the Human Stem Cells Institute) in collaboration with its subsidiary Genotarget for the treatment of dysferlinopathy. Dysferlinopathy is a group of rare genetic muscle-wasting disorders, including Limb-Girdle Muscular Dystrophy type 2B (LGMD2B) and Miyoshi Myopathy, caused by mutations in the *DYSF* gene. These mutations result in a deficiency of the dysferlin protein, which is critical for the calcium-dependent repair of muscle cell membranes. GT DF 102 is designed to deliver a functional copy of the *DYSF* gene to muscle cells to restore dysferlin expression and improve membrane repair mechanisms. The program is currently in the preclinical stage of development.

Other names

Genotarget dysferlin gene therapy