Sandhoff disease research
Sandhoff dieease is a variation of the rare and usually fatal genetic disorder Tay-Sachs disease, which causes progressive damage to the nervous system.
Timothy Cox's research group is working to develop gene therapy for children who have inherited Sandhoff disease. In the most common form of Tay-Sachs condition, symptoms usually begin at around six months of age, when a previously normal child's development begins to slow and they gradually lose their ability to move. Mainly it is in Sandhoff disease models that gene therapy and other enhancing treatments have been first tried; they respond well to gene therapy, which is also being extended to Tay-Sachs disease as well.