Feb 15, 2022

First gene therapy for Tay-Sachs disease successfully given to two children

Posted by in categories: biotech/medical, neuroscience

About 1 in 300 people in the general population carry the Tay-Sachs disease gene. Ray Kachatorian/Stone via Getty ImagesTwo 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 exists in very small, harmless amounts in the brain. Without HexA, however, this fatlike substance can accumulate to toxic levels that damage and kill neurons.

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