Liver gene therapy corrects heart symptoms in model of rare enzyme disorder
Tuesday, September 30, 2014 - 05:31
in Health & Medicine
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.