GNT0007

GNT0007 is an investigational gene therapy developed by Genethon for the treatment of gamma-sarcoglycanopathy, a form of Limb-Girdle Muscular Dystrophy (LGMD) also known as LGMD R5 (formerly LGMD2C). The therapy is designed to deliver a functional copy of the SGCG gene, which encodes the gamma-sarcoglycan protein, to muscle cells. Gamma-sarcoglycan is a transmembrane glycoprotein that is part of the sarcoglycan complex, which in turn is a component of the dystrophin-associated glycoprotein complex (DAGC). This complex plays a crucial role in maintaining the structural integrity of the sarcolemma during muscle contraction and relaxation. Mutations in the SGCG gene lead to a deficiency of gamma-sarcoglycan, resulting in progressive muscle fiber damage and weakness. GNT0007 aims to restore protein expression and stabilize muscle tissue. It is currently in the preclinical stage of development.

Other names

SGCG gene therapygamma-sarcoglycan gene therapy