Gene therapy for Pompe disease effective in mice, poised for human trials

(Duke University Medical Center) After decades investigating a rare, life-threatening condition that cripples the muscles, Duke Health researchers have developed a gene therapy they hope could enhance or even replace the only FDA-approved treatment currently available to patients.The therapy uses a modified virus to deliver a gene to the liver where it produces GAA, an enzyme missing in people with Pompe disease.

from EurekAlert! - Social and Behavioral Science http://ift.tt/2jj5cg8