Fragile X syndrome (FXS) is caused by loss of the fragile X mental retardation 1 gene product, a repressor of mRNA translation. It is thought that excessive protein synthesis downstream of metabotropic glutamate receptor 5 activation leads to many of the neuropsychiatric symptoms of FXS. The authors show that lovastatin, a widely prescribed cholesterol-lowering drug, can normalize protein synthesis in the hippocampus of a mouse model of FXS and prevent epileptogenesis. Although the effects of the drug on the neurocognitive phenotypes of these mice remain to be investigated, lovastatin could be a promising therapy for FXS.
ORIGINAL RESEARCH PAPER
Osterweil, E. K. et al. Lovastatin corrects excess protein synthesis and prevents epileptogenesis in a mouse model of fragile X syndrome. Neuron 77, 243–250 (2013)
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Flight, M. Lovastatin as fragile X therapy. Nat Rev Neurosci 14, 156 (2013). https://doi.org/10.1038/nrn3459