Meeting Banner
Abstract #4644

Longitudinal 7T MRI and MRS in a sheep model of Tay-Sachs disease and the effect of AAV gene therapy.

Heather Gray-Edwards1, Elise Diffie1, Ashley Randle1, Amanda Gross1, Nouha Salibi2, Lauren Ellis 1, Ronald Beyers1, Miguel Sena-Esteves3, Thomas Denney1, and Douglas Martin1

1Auburn University, Auburn, AL, United States, 2MR R&D, Seimens Healthcare, Malvern, PA, United States, 3University of Massachusetts, Worcester, MA, United States

Tay-Sachs Disease (TSD) is a fatal neurodegenerative disorder of children and the sheep model of TSD is a powerful tool to study the disease and evaluate novel therapies. One such therapy, adeno associated viral (AAV) gene therapy has resulted in a 2-fold increase in lifespan and biomarkers are needed for clinical trials. 7T MRI shows white and gray matter alterations and MR spectroscopic abnormalities that worsen with TSD disease progression. At humane endpoint the AAV treated sheep has normalization of gray and white matter intensities, but cortical atrophy and MRS alterations persist. 7T MRI and MRS reflect TSD disease severity.

This abstract and the presentation materials are available to members only; a login is required.

Join Here