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

This study demonstrates the therapeutic potential of AAV for feline SD and suggests a similar potential for human SD patients.

Source: ASN Neuro - April 13, 2015 Category: Neuroscience Authors: Rockwell, H. E., McCurdy, V. J., Eaton, S. C., Wilson, D. U., Johnson, A. K., Randle, A. N., Bradbury, A. M., Gray-Edwards, H. L., Baker, H. J., Hudson, J. A., Cox, N. R., Sena-Esteves, M., Seyfried, T. N., Martin, D. R. Tags: Original Article Source Type: research