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Miguel Sena-Esteves

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

theconversation.com
2025-12-302022-02-20 by Miguel Sena-Esteves
first-gene-therapy-for-tay-sachs-disease-successfully-given-to-two-children

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 … Read more

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