- Characterization of inducible models of Tay-Sachs and related diseaseTimothy J Sargeant
Department of Medicine, Addenbrooke s Hospital, University of Cambridge, Cambridge, United Kingdom
PLoS Genet 8:e1002943. 2012..As in germline Hexb-/- mice, these neurodegenerative manifestations advanced rapidly, indicating that the pathogenesis and progression of GM2 gangliosidosis is not influenced by developmental events in the maturing nervous system...
- Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brainTimothy J Sargeant
Department of Medicine, University of Cambridge, Level 5 Addenbrooke s Hospital, Box 157, Hills Road, Cambridge CB2 0QQ, UK
Hum Mol Genet 20:4371-80. 2011..Loss of neurons was localized to only a few regions in the Sandhoff brain and was prevented by rAAV-mediated transfer of β-hexosaminidase gene function at considerable distances from the site of vector administration...