Abstract #3903

Assessment of gene therapy efficacy by neurochemical profiling

Ivan Tkac¹, Igor Nastrasil², Kanut Laoharawee³, Kelly M Podetz-Pedersen³, Kelley F Kitto⁴, Carolyn A Fairbanks⁵, Walter C Low⁶, Karen Kozarsky⁷, and R Scott McIvor³

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 Neuroscience, University of Minnesota, Minneapolis, MN, United States, ⁵Dept. of Pharmaceutics, University of Minnesota, Minneapolis, MN, United States, ⁶Dept. of Neurosurgery, University of Minnesota, Minneapolis, MN, United States, ⁷REGENXBIO Inc., Rockville, MD, United States

Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is a rare X-linked recessive lysosomal disorder caused by defective iduronate-2-sulfatase (IDS). Enzyme replacement is the only FDA–approved therapy available for MPS II, but it does not improve neurologic outcomes in MPS II patients. The 1H MRS data acquired from the hippocampus and cerebellum of untreated and AAV9-IDS treated MPS II mice and heterozygote controls clearly demonstrate that the direct transfer of the missing IDS gene to the CNS at 12 weeks of age prevented neurochemical alternations typical for MPS II at 9 months of age.

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