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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