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GENE THERAPY

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.

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).

    CAS  Article  Google Scholar 

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

    CAS  Article  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).

    CAS  Article  Google Scholar 

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

    CAS  Article  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).

    CAS  Article  Google Scholar 

Download references

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

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