Gene therapy towards a clinical trial for gamma-sarcoglycanopathy, limb-girdle muscular dystrophy
Gamma-sarcoglycanopathy (LGMD2C) is one of the most common limb-girdle myopathies. It affects less than 10 people per 1,000,000 and is characterized by progressive muscle weakness in the pelvis (pelvic girdle) and shoulders (scapular girdle), linked to mutations in the SGCG gene encoding gamma-sarcoglycan. There is currently no cure for this neuromuscular disease. In this study,…
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