REGENXBIO Inc. today announced that gene transfer mediated by REGENXBIO's NAVA AAV8 vectors resulted in sustained serum a-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.
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