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Research Initiatives
Gene Therapy: A consortium of researchers from Auburn University, Boston College, Cambridge University (U.K.), Harvard Medical School and Mass. General Hospital have come together to develop a gene therapy project that has shown very promising results in animal models. Since the formation of this project there has been a grant of $3.5 million from the National Institute of Health given last year to continue work an additional 4 yr. period. Hopefully we will be in clinical human trials at that time.

Gm2 Ganglioside Inhibitors: Research is underway at Boston College to block the production of GM2 Ganglioside. The theory is that reducing the waste created in a cell will slow the progression of cell death caused by the Hex A deficiency in people with Tay-Sachs disease. Currently, there are at least 2 sites where clinical trials are taking place with a drug called Pyrimethamine. The Late Onset Tay-Sachs Research and Education Foundation has given a $15,000 grant toward this project in 2009.
KCBS News Healthwatch Report on Late Onset Tay-Sachs KCBS News Healthwatch Report on Late Onset Tay-Sachs
The Late Onset Tay-Sachs Research and Education Foundation.
Educate and help people affected with this debilitating disease that strikes older children, teens and adults alike.
The world needs to know that Tay-Sachs affects others bedsides infants and very young children.
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