Correction of cardiovascular symptoms of MPS I in animal model

REGENXBIO Inc. today announced that gene transfer mediated by REGENXBIO's NAV AAV8 vectors resulted in sustained serum I -L-iduronidase expression, as well as correction of systemic features of MPS I, or Hurler syndrome, a lysosomal storage disease caused by the body's inability to produce the IDUA enzyme. Data from a study performed by researchers at the Perelman School of Medicine at the University of Pennsylvania show animals treated with a single intravenous injection of NAV AAV8 vectors expressing the IDUA gene not only demonstrated meaningful improvements in the biochemical features of MPS I in most tissues, but the majority also exhibited complete resolution of aortic valve lesions.

http://ift.tt/1rHvB8B