The Tay-Sachs
Gene Therapy (TSGT) Consortium was formed in 2007 with the goal of initiating a gene therapy clinical trial for Tay-Sachs disease and Sandhoff disease. Read more about the TSGT Consortium results and progress toward a cure. Clinical trials could begin as early as September 2012!
The TSGT Consortium includes scientists and clinicians from the following institutions:
The TSGT Consortium is led by Dr. Miguel Sena Esteves, recipient of the 2011 Outstanding New Investigator Award from the American Society of Gene & Cell Therapy. Often referred to as the “unit of heredity,” a gene is composed of a sequence of DNA required to produce a functional
protein.
Visit the TSGT website at www.tsgtconsortium.com to read about all the scientists and clinicians in the TSGT Consortium.
The TSGT Consortium seeks rapid development of the most effective gene therapy approach for the clinical trial. To accomplish this goal, they are pooling their resources and extensive experience in experimental gene therapy to devise the most effective adeno-associated virus (AAV)-based gene therapy approach for treating Tay-Sachs and Sandhoff disease.
