Gene therapy for Limb-Girdle Muscular Dystrophy 2g

Gene therapy for Limb-Girdle Muscular Dystrophy 2g refers to investigational gene therapies targeting the genetic cause of LGMD type 2G, a rare autosomal recessive muscular dystrophy caused by mutations in the telethonin (TCAP) gene. These therapies aim to deliver a functional copy of the TCAP gene to muscle cells using viral vectors, most commonly adeno-associated virus (AAV) vectors. The goal is to restore or replace deficient protein function, thereby improving muscle strength and slowing disease progression. As of now, there are no approved gene therapies specifically for LGMD2G; however, similar approaches are in clinical development for other LGMD subtypes such as 2D/R3 (SRP-9004), 2I/R9 (ATA-100 and AB-1003), and 2C/R5 (ATA-200)[3][4][6][7][8]. No specific developer or product has been publicly disclosed for LGMD2G.