Initiate a state of the art clinical trial for Tay-Sachs disease (and Sandhoff disease) in the next 3-4 years.
Tay-Sachs Disease (TSD) is an inherited neurological disease that severely affects the nervous system, and causes premature death. Children with the most common and severe form of Tay-Sachs Disease develop normally for the first months of life, but slowly deteriorate until death, usually by age 5. Juvenile and Late Onset forms are also devastating conditions. Although TSD was first described in the 1880's, this disease remains untreated. In recent years, outstanding therapeutic results have been achieved in mouse models of TSD using state-of-the-art gene therapy systems known as adeno-associated viral (AAV) vectors. Translation of these findings from mice to humans is a significant challenge that requires a considerable amount of research.
The Tay-Sachs Gene Therapy (TSGT) Consortium is an international collaborative group of scientists committed to translating current results from animal experiments into a human clinical trial within the next 3 years. The TSGT Consortium consists of physicians and scientists experienced in gene therapy and basic disease research, from four institutions: Auburn University, Boston College, Cambridge University (U.K.), and the Massachusetts General Hospital/Harvard University.