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Neurodegenerative disease

Two–for–one on potential therapies

Nature volume 544, pages 302–303 (2017)Cite this article

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Molecules that inhibit the synthesis of the ataxin 2 protein can ameliorate the effects of two neurodegenerative diseases in mouse models, raising hopes for the success of this approach in clinical trials. See Letters p.362 & p.367

Neurodegenerative disorders can be devastating, and many lack effective treatments. One possible therapeutic strategy involves the use of antisense oligonucleotides (ASOs) — short stretches of nucleotides that bind to a messenger RNA and inhibit its translation into protein. Support for this idea is provided by two papers in this issue1,2, which report that ASOs that target mRNA translated into the ataxin 2 protein can ameliorate movement defects in mouse models of neurodegenerative disease.

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Figure 1: A shared strategy for treating two neurodegenerative diseases.

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Authors and Affiliations

  1. Ke Zhang and Jeffrey D. Rothstein are at the Brain Science Institute, Johns Hopkins University, Baltimore, Maryland 21205, USA.,

    Ke Zhang & Jeffrey D. Rothstein

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  1. Ke Zhang
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  2. Jeffrey D. Rothstein
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Correspondence to Ke Zhang or Jeffrey D. Rothstein.

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Zhang, K., Rothstein, J. Two–for–one on potential therapies. Nature 544, 302–303 (2017). https://doi.org/10.1038/nature21911

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