The quest for a treatment for muscular dystrophy has led some scientists to focus on the half-dozen muscles surrounding the eyes. These extraocular muscles, which control eye movement, remain untouched by the disease. Some researchers think they understand why and hope to turn that understanding into a treatment.
Normal distribution of dystrophin at the muscle cell surface and utrophin at sites of neuromuscular junctions (nmj). In animal models of Duchenne dystrophy, utrophin appears to expand beyond nerve-muscle junctions to replace dystrophin in extraocular muscle. |
The disease is marked by genetic mutation resulting in the absence of the membrane protein dystrophin, which is needed for muscle function. Without that protein the...
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