A research team has succeeded in restoring a missing repair protein in skeletal muscle of patients with muscular dystrophy, a scientific first. The team has offered a proof-of-principle study and restored the missing protein in skeletal muscle of patients with muscular dystrophy. Three patients carrying a dysferlin mutation received a single systemic dose of a proteasome inhibitor. After only a few days the patients’ musculature produced the missing dysferlin protein at levels that could be therapeutically effective.

Source: Missing Protein Restored in Patients with Muscular Dystrophy

David Cottle

UBB Owner & Administrator