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Abstract #2406

AAV serotype 9 vector transducing the human alpha-L-iduronidase gene normalizes hippocampal and cerebellar neurochemical profiles in a mouse model of mucopolysaccharidosis type I

Ivan Tkac1, Igor Nestrasil2, R Scott McIvor3, Kelley Kitto4, Carolyn A Fairbanks4, Karen Kozarsky5, Walter C Low6, Chester B Whitley2, and Lalitha Belur3

1Center for Magnetic Resonance Research, University of Minnesota, Minneapolis, MN, United States, 2Dept. of Pediatrics, University of Minnesota, Minneapolis, MN, United States, 3Dept. of Genetics and Cell Biology, University of Minnesota, Minneapolis, MN, United States, 4Dept. of Pharmaceutics, University of Minnesota, Minneapolis, MN, United States, 5REGENXBIO Inc., Rockville, MD, United States, 6Dept. of Surgery, University of Minnesota, Minneapolis, MN, United States

Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disease caused by the deficiency in α-L-iduronidase (IDUA) enzyme which results in lysosomal accumulation of glycosaminoglycans. The purpose of this study was to assess the ability of the adeno-associated virus (AAV) - mediated IDUA gene therapy to prevent the pathological neurochemical changes associated with the MPS I disease. The efficacy of the gene therapy was assessed by in vivo 1H MRS at 9.4T using knockout mice deficient for IDUA, a well-established murine model of MPS I.

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