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Abstract #2723

Monitoring micro-dystrophin treatment effects in mdx4cv mice using magnetic resonance imaging and spectroscopy

Ravneet Vohra1, Guy Odom2, Jeffrey S Chamberlain2,3,4, and Donghoon Lee1
1Radiology, University of Washington, Seattle, WA, United States, 2Neurology, University of Washington, Seattle, WA, United States, 3Medicine, University of Washington, Seattle, WA, United States, 4Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington, Seattle, WA, United States

Mutations in the X-linked dystrophin gene disrupts protein expression resulting in Duchenne muscular dystrophy (DMD), a neuromuscular disorder characterized by body-wide muscle cell degeneration. The mdx mouse model is one of the most commonly used animal models for DMD. Recombinant adeno-associated viral (rAAV) vector-mediated gene transfer represents a promising approach for DMD. Magnetic resonance has emerged as a noninvasive method in monitoring disease progression and treatment response for muscular dystrophy. The aim of this study was to elucidate the functional impact of micro-dystrophin on skeletal muscles using magnetic resonance imaging and spectroscopy.

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