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Children's National Hospital experts developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy (LGMD) 2B, that addresses the primary cellular deficit associated with this disease.
A preclinical study provides proof-of-concept evidence for a new gene therapy approach that offers a potential long-term treatment for limb-girdle muscular dystrophy 2B. Researchers at Children’s National have developed the first non-muscle targeted gene therapy that enhances diseased muscle fiber repair and improves muscle function through a single low dose of an AAV vector that produces human acid sphingomyelinase, released by muscle cells immediately following muscle injury.
Research offers hope for people with limb-girdle muscular dystrophy