Tay-Sachs Disease Ganglioside research hub

Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis

This study is looking at a new way to treat rare conditions called Tay-Sachs and Sandhoff disease. Researchers are testing a gene therapy using a person's own blood cells to see if it can help correct the problem causing these diseases. The aim is to find an effective treatment for people.