NEWS FEATURE
06 April 2021

Genetic therapies offer new hope against incurable brain diseases

A class of drugs that silence the effects of faulty genes could help tackle brain diseases — but a halted clinical trial has brought the field up short.

Diana Kwon⁰

Diana Kwon
1. Diana Kwon is a freelance science journalist based in Berlin.
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4 year old Evie walks up a set of stone steps in the garden followed by her father and mother — Evie Lewis with her parents, Elliot and Janell. Evie receives a dose of a genetic therapy every few months to treat spinal muscular atrophy. Credit: Kim Raff for *Nature*

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Update: Since this story was published, the Roche and Wave Life Sciences trials mentioned in the feature have been halted, owing to the drugs’ disappointing performance.

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Nature 592, 180-183 (2021)

doi: https://doi.org/10.1038/d41586-021-00870-x

References

Stephenson, M. L. & Zamecnik, P. C. Proc. Natl Acad. Sci. USA 75, 285–288 (1978).
Article PubMed Google Scholar
Hua, Y., Vickers, T. A., Okunola, H. L., Bennett, F. & Krainer, A. R. Am. J. Hum. Genet. 82, 834–848 (2008).
Article PubMed Google Scholar
Finkel, R. S. et al. N. Engl. J. Med. 377, 1723–1732 (2017).
Article PubMed Google Scholar
Tabrizi, S. J. et al. N. Engl. J. Med. 380, 2307–2316 (2019).
Article PubMed Google Scholar
Qian, H. et al. Nature 582, 550–556 (2020).
Article PubMed Google Scholar