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

In a piece written for The Conversation, Miguel Sena-Esteves, PhD, talks about research leading to the first ever gene therapy for Tay-Sachs disease and the launch of the Translational Institute for Molecular Therapeutics at UMass Chan. ....

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THE CANDLE: Genetic disorders to fall at the hands of science

A recent article titled “A solid start for gene therapy in Tay-Sachs disease” was published in “Nature” on Feb. 10. The article offers a glimpse into one of the mechanisms ....

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