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New gene therapy addresses primary cellular deficit associated with limb-girdle muscular dystrophy 2B

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. ....

Sreetama Sen Chandra , Daniel Bittel , Jyotik Jaiswal , Emily Henderson , Children National , Genetic Medicine Research At Children National , Children National Hospital , Rare Disease Institute , Genetic Medicine Research , National Hospital , Sen Chandra , Gene Therapy , Muscular Dystrophy ,

Gene Therapy for a Rare Muscular Dystrophy Overcomes Earlier Roadblocks

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. ....

United States , Sreetama Sen Chandra , Jyoti Jaiswal , Daniel Bittel , Jyotik Jaiswal , Eric Hoffmann , Bradley Williams , Children National , Genetic Medicine Research At Children National , Binghamton University , Children National Hospital , Jain Foundation , George Washington University School Of Medicine , Health Sciences , National Hospital , Clinical Investigation , Genetic Medicine Research , National Health System , George Washington University School , Sen Chandra ,