Large1 gene transfer in older myd mice with severe muscular dystrophy restores muscle function and greatly improves survival

Abstract excerpt

Muscular dystrophy is a progressive and ultimately lethal neuromuscular disease. Although gene editing and gene transfer hold great promise as therapies when administered before the onset of severe clinical symptoms, it is unclear whether these strategies can restore muscle function and improve survival in the late stages of muscular dystrophy. Large myd /Large myd ( myd ) mice lack expression of...

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