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
Scott-Ritchey Research Center, Auburn
Univeristy, Auburn, Al, United States,
Healthcare, Malvern, PA, United States,
Univeristy MRI Research Center, Auburn Univeristy, AL,
Sciences, Auburn Univeristy, AL, United States,
University MRI Research Center, Auburn Univeristy, AL,
University of Massachusetts, MA, United States,
of Electrical Engineering, Auburn Univeristy, AL, United
Phsiology and Pharmacology, Auburn Univeristy, AL,
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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