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

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