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GENE THERAPY

A solid start for gene therapy in Tay–Sachs disease

Nature Medicine volume 28, pages 236–237 (2022)Cite this article

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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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Authors and Affiliations

  1. Division of Genetics and Genomics, Boston Children’s Hospital, Boston, MA, USA

    Timothy W. Yu & Olaf Bodamer

  2. Broad Institute of MIT and Harvard, Cambridge, MA, USA

    Timothy W. Yu & Olaf Bodamer

  3. Harvard Medical School, Boston, MA, USA

    Timothy W. Yu & Olaf Bodamer

  4. Department of Neurology, Boston Children’s Hospital, Boston, MA, USA

    Timothy W. Yu

Authors
  1. Timothy W. Yu
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  2. Olaf Bodamer
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Correspondence to Timothy W. Yu.

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The authors declare no competing interests.

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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). https://doi.org/10.1038/s41591-022-01687-5

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