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Translational medicine

Mice and men show the way

Nature volume 491, pages 196–197 (2012)Cite this article

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Disorders caused by single genes, such as fragile X syndrome, share symptoms with the genetically complex autism spectrum disorders. It emerges that effective drugs for the former may lead to therapies for the latter.

The translation of findings from genomics and animal studies into therapies for neurodevelopmental disorders, such as autism, is still in its infancy. One promising approach is to align animal studies with clinical trials, as Henderson et al.1 and Berry-Kravis et al.2 report in Science Translational Medicine. Their data suggest that the drug arbaclofen (STX209) reverses symptoms related to fragile X syndrome in a mouse model of the disorder. It also promotes social engagement in patients with fragile X syndrome who show marked social impairment.

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Figure 1: From lab to clinic.

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Authors and Affiliations

  1. Department of Pediatrics, Evdokia Anagnostou is at the Bloorview Research Institute, University of Toronto, Toronto, Ontario M4G 1R8, Canada.,

    Evdokia Anagnostou

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  1. Evdokia Anagnostou
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Correspondence to Evdokia Anagnostou.

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Anagnostou, E. Mice and men show the way. Nature 491, 196–197 (2012). https://doi.org/10.1038/491196a

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