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Abstract #3955

Gene Therapy Evaluated using In Vivo Diffusion Tensor Imaging

Joong Hee Kim1, Adarsh S. Reddy2, Mark S. Sands2, Sheng-Kwei Song1

1Radiology, Washington University, St. Louis, MO, United States; 2Internal Medicine, Washington University, St. Louis, MO, United States

Globoid-cell Leukodystrophy (GLD) is an inherited demyelinating disease caused by a deficiency of the lysosomal enzyme Galactosylceramidase (GALC). A previous study demonstrated dramatic synergy between CNS-directed AAV2/5 gene therapy and myeloreductive bone marrow transplantation (BMT) in the murine model of GLD (twitcher). In the present study, in vivo DTI was employed to evaluate the effect of gene therapy of twitcher mice demonstrating the treatment induced recovery of white matter integrity.