Research ResourceDRUG DEVELOPMENT

Hyperactive locomotion in a Drosophila model is a functional readout for the synaptic abnormalities underlying fragile X syndrome

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Sci. Signal.  02 May 2017:
Vol. 10, Issue 477, eaai8133
DOI: 10.1126/scisignal.aai8133

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Spurring drug development for FXS

Fragile X syndrome (FXS) is an autistic intellectual disability disorder caused by loss of the RNA binding protein FMRP. Treating FXS has been challenging because of the lack of a reliable in vivo drug screening model. Kashima et al. found that the hyperactive locomotion observed in a fly model of FXS was a reliable behavioral marker for the neurological abnormalities underlying the disease. The kinase LIMK1 is implicated in the pathogenesis of FXS, and high-throughput (rapid, quantitative, and time- and cost-effective) drug screening in the fly FXS model confirmed that LIMK1 inhibitors ameliorated both the neurological and behavioral phenotypes of this model. LIMK1 inhibitors also reduced hyperactivity in a mouse model of FXS. Thus, this method may aid in future drug development for FXS patients.