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A solid start for gene therapy in Tay–Sachs disease

A ‘gene addition’ strategy shows promise in a first-in-human trial involving two infant patients.

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Fig. 1: Intrathecal and intrathalamic delivery to the CNS in TSD.


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Correspondence to Timothy W. Yu.

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Yu, T.W., Bodamer, O. A solid start for gene therapy in Tay–Sachs disease. Nat Med 28, 236–237 (2022).

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