Variable rescue of microtubule and physiological phenotypes in mdx muscle expressing different miniaturized dystrophins
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DOI: 10.1093/hmg/ddy113
Abstract: Delivery of miniaturized dystrophin genes via adeno-associated viral vectors is one leading approach in development to treat Duchenne muscular dystrophy. Here we directly compared the functionality of five mini- and micro-dystrophins via skeletal muscle-specific transgenic… read more here.
Keywords: microtubule; mini micro; mdx muscle; micro dystrophins ... See more keywords