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AAV mediated gene transfer to the CNS for MPS II

Mother and son attend MPS awareness event

July 21, 2017

Currently there is no established treatment for neurologic symptoms of Hunter syndrome, caused absence of the enzyme iduronate sulfatase (IDS). We have established conditions for effective gene transfer to all areas of the brain using a genetically engineered virus (AAV). In this research project, we will test these conditions for introduction of the gene for IDS into the brain in mice that are deficient in IDS, then testing for prevention of neurologic disease. We anticipate that results from these studies will facilitate the development of genetic therapy for neurologic symptoms of Hunter syndrome.

– Scott McIvor, PhD

University of Minnesota Minneapolis, MN