AAV-Mediated Gene Delivery in a Feline Model of Sandhoff Disease Corrects Lysosomal Storage in the Central Nervous System

by Hannah E. Rockwell, Victoria J. McCurdy, Samuel C. Eaton, Diane U. Wilson, Aime K. Johnson, Ashley N. Randle, Allison M. Bradbury, Heather L. Gray-Edwards, Henry J. Baker, Judith A. Hudson, Nancy R. Cox, Miguel Sena-Esteves, Thomas N. Seyfried, Douglas R. Martin
Published 2015-04-01