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Abstract #4293

Long Term MRI and MR spectroscopic evaluation of gene therapy in a feline model of neurologic disease.

Heather L Gray-Edwards 1 , Nouha Salbi 2,3 , Ashley N Randle 1 , Judith Hudson 4 , Ronald Beyers 5 , Miguel Sena Esteves 6 , Thomas Denney 5,7 , and Douglas Martin 1,8

1 Scott-Ritchey Research Center, Auburn Univeristy, Auburn, Al, United States, 2 Seimens Healthcare, Malvern, PA, United States, 3 Auburn Univeristy MRI Research Center, Auburn Univeristy, AL, United States, 4 Clinical Sciences, Auburn Univeristy, AL, United States, 5 Auburn University MRI Research Center, Auburn Univeristy, AL, United States, 6 Neurology, University of Massachusetts, MA, United States, 7 Department of Electrical Engineering, Auburn Univeristy, AL, United States, 8 Anatomy, Phsiology and Pharmacology, Auburn Univeristy, AL, United States

GM1 gangliosidosis is a fatal neurodegenerative disease of children for which there is no cure. A well characterized feline GM1 model was used to test intracranial gene therapy. Gene therapy resulted in a 5 fold increase in lifespan and marked attenuation of neurologic signs. MRI and Single voxel MR spectroscopy was performed in the thalamus, centrum ovale, parietal cortex, temporal lobe, occipital cortex and cerebellum. AAV gene therapy partially normalized MRI and metabolite alterations in the GM1 cat brain, and data suggest that MRS is a sensitive measure of therapeutic efficacy in discrete brain areas.

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