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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
First gene therapy for Tay-Sachs disease successfully given to two childrenAbout 1 in 300 people in the general population carry the Tay-Sachs disease gene.Ray Kachatorian/Stone via Getty Images

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...

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

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