AAV9-mediated gene transfer of desmin ameliorates cardiomyopathy in desmin-deficient mice

Mutations of the human desmin ( DES ) gene cause autosomal dominant and recessive myopathies affecting skeletal and cardiac muscle tissue. Desmin knockout mice (DES-KO), which develop progressive myopathy and cardiomyopathy, mirror rare human recessive desminopathies in which mutations on both DES a...

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