AAV1-gamma-sarcoglycan

AAV1-gamma-sarcoglycan is an investigational adeno-associated virus (AAV) serotype 1-based gene therapy developed for the treatment of Limb-Girdle Muscular Dystrophy Type 2C (LGMD2C), also known as gamma-sarcoglycanopathy. LGMD2C is an autosomal recessive muscle-wasting disease caused by mutations in the SGCG gene, which leads to a deficiency in the gamma-sarcoglycan protein, a vital component of the dystrophin-associated glycoprotein complex. The therapy utilizes a self-complementary AAV1 vector to deliver a functional human SGCG transgene under the control of a muscle-specific promoter (tMCK). By restoring gamma-sarcoglycan expression in skeletal muscle cells, the treatment aims to stabilize the sarcolemma, prevent muscle fiber degeneration, and improve motor function. Clinical development has focused on local intramuscular administration to evaluate safety and transgene expression.

Other names

Gamma-sarcoglycan Gene TherapyAAV1-SGCGAAV-1-SGCGAAV 1-SGCGAAV1.gamma-sarcoglycanAAV-1.gamma-sarcoglycanAAV 1.gamma-sarcoglycan