First gene therapy for Tay-Sachs disease successfully given to two children
- Written by Miguel Sena-Esteves, Associate Professor of Neurology, UMass Chan Medical School
Two babies have received the first-ever gene therapy for Tay-Sachs disease after over 14 years of development.
Tay-Sachs is a severe neurological disease caused by a deficiency in an enzyme called HexA. This enzyme breaks down a...