Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • News & Views
  • Published:

GENE THERAPY

A solid start for gene therapy in Tay–Sachs disease

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

Subjects

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

This is a preview of subscription content, access via your institution

Access options

Buy this article

  • Purchase on SpringerLink
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

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

References

  1. Flotte, T. R. et al. Nat. Med. https://doi.org/10.1038/s41591-021-01664-4 (2022).

    Article  PubMed  Google Scholar 

  2. Platt, F. M., d’Azzo, A., Davidson, B. L., Neufeld, E. F. & Tifft, C. J. Nat. Rev. Dis. Primers 4, 274 (2018).

    Google Scholar 

  3. Bembi, B. et al. Neurology 66, 278–280 (2006).

    Article  CAS  Google Scholar 

  4. Maegawa, G. H. B. et al. Mol. Genet. Metab. 98, 215–224 (2009).

    Article  CAS  Google Scholar 

  5. Hwu, W. L. et al. Sci. Transl. Med. 4, 134ra61 (2012).

    Article  Google Scholar 

  6. Mendell, J. R. et al. N. Engl. J. Med. 377, 1713–1722 (2017).

    Article  CAS  Google Scholar 

  7. Bailey, R. M., Armao, D., Nagabhushan Kalburgi, S. & Gray, S. J. Mol. Ther. Methods Clin. Dev. 9, 160–171 (2018).

    Article  CAS  Google Scholar 

  8. Bainbridge, J. W. B. et al. N. Engl. J. Med. 372, 1887–1897 (2015).

    Article  Google Scholar 

  9. Eichler, F. et al. N. Engl. J. Med. 377, 1630–1638 (2017).

    Article  CAS  Google Scholar 

Download references

Author information

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
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Olaf Bodamer
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Timothy W. Yu.

Ethics declarations

Competing interests

The authors declare no competing interests.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

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

Download citation

  • Published:

  • Issue Date:

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

This article is cited by

Search

Advanced search

Quick links

Nature Briefing: Translational Research

Sign up for the Nature Briefing: Translational Research newsletter — top stories in biotechnology, drug discovery and pharma.

Get what matters in translational research, free to your inbox weekly. Sign up for Nature Briefing: Translational Research