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Abstract #4453

Monitoring Treatment Response to Gene Therapy in a Feline Model of Alpha-mannosidosis using 1H MRS

Sanjeev Chawla1, Manoj Kumar1,2, Sea-Young Yoon3, Harish Poptani4, and John H Wolfe3

1Radiology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, United States, 2Center for Perinatal Research, The Research Institute, and Nationwide Children’s Hospital, Columbus, OH, United States, 3Abramson Research Center, Children’s Hospital of Philadelphia, Philadelphia, PA, United States, 4Department of Cellular and Molecular Physiology, University of Liverpool, Liverpool, United Kingdom

To evaluate the potential of in vivo proton MR spectroscopy (1H MRS) in monitoring treatment response to adeno-associated viral (AAV) gene therapy to the cisterna-magna in a cat model of alpha-mannosidosis. Normal (n=3), untreated (n=3) and AAV-treated (n=3) cats underwent multivoxel 1H MRS. Significant elevations in resonances due to mannose-rich oligosaccharides (OG) and N-acetylated sugars (Glc-NAc) were observed in brain of untreated cats compared to normal controls. AAV treated cats demonstrated a decrease (normalization) in the OG and Glc-NAc resonances, which were not significantly different from untreated cats indicating some degree of positive response to this therapy.

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