First Gene Therapy for Tay-Sachs Disease Successfully Given to Two Children

The following essay is reprinted with permission from The Conversation, an online publication covering the latest research. 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 fatlike substance that normally … Read more