Leading the worldwide fight to treat and cure
Tay-Sachs, Canavan, GM1 and Sandhoff diseases

Tay-Sachs Gene Therapy Consortium

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.

The TSGT Consortium includes scientists and clinicians from the following institutions:

  • Auburn University, AL
  • Boston College, MA
  • Cambridge University - UK
  • Massachusetts General Hospital/Harvard Medical School
  • University of Massachusetts Medical School
  • New York University Medical School

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.

Progress Reports & Funding

The Tay-Sachs Gene Therapy (TSGT) Consortium Results and Progress toward a Cure

Funding the TSGT Consortium

Jacob Sheep