mardi 30 septembre 2014

Liver gene therapy corrects heart symptoms in model of rare enzyme disorder

Researchers examined systemic delivery of a vector to replace the enzyme IDUA, which is deficient in patients with a rare enzyme deficiency disorder. The works describes how an injection of a vector expressing the IDUA enzyme to the liver can prevent most of the systemic manifestations of the disease, including those found in the heart.

Liver gene therapy corrects heart symptoms in model of rare enzyme disorder

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