Abstract #1238

7T MR spectroscopy reflects disease severity in a large animal model of neurologic disease and the effects of gene therapy

Heather Gray-Edwards¹, Nouha Salibi², Anne Maguire¹, Taylor Voss¹, Lauren Ellis¹, Ashley Randle¹, Ronald Beyers¹, Miguel Sena-Esteves³, Thomas Denney¹, and Douglas Martin¹

¹Auburn University, Auburn, AL, United States, ²Siemens Healthcare, Malvern, PA, United States, ³University of Massachusetts, Worcester, MA, United States

GM1 gangliosidosis is a fatal neurodegenerative disorder of children and currently only palliative care is available to patients. Preclinical gene therapy experiments in the GM1 cat resulted in >5 fold increased lifespan, prompting human clinical trials, however objective markers are lacking. 7T MR spectroscopy reliably predicts feline GM1 neurodegeneration with several alterations occurring presymptomatically and worsening with disease progression. Gene therapy results in partial correction of several parameters and changes correlate with clinical assessment scores. Post-mortem analyses included assessment of microgliosis and demyelination, and these findings also correlated with MRS.

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