Abstract #4453

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

Sanjeev Chawla¹, Manoj Kumar^1,2, 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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