Metformin ameliorates core deficits in a mouse model of fragile X syndrome

Ilse Gantois, Arkady Khoutorsky, Jelena Popic, Argel Aguilar-Valles, Erika Freemantle, Ruifeng Cao, Vijendra Sharma, Tine Pooters, Anmol Nagpal, Agnieszka Skalecka, Vinh T. Truong, Shane Wiebe, Isabelle A. Groves, Seyed Mehdi Jafarnejad, Clément Chapat, Elizabeth A. McCullagh, Karine Gamache, Karim Nader, Jean Claude Lacaille, Christos G. GkogkasNahum Sonenberg

Research output: Contribution to journalArticle

70 Scopus citations

Abstract

Fragile X syndrome (FXS) is the leading monogenic cause of autism spectrum disorders (ASD). Trinucleotide repeat expansions in FMR1 abolish FMRP expression, leading to hyperactivation of ERK and mTOR signaling upstream of mRNA translation. Here we show that metformin, the most widely used drug for type 2 diabetes, rescues core phenotypes in Fmr1-/y mice and selectively normalizes ERK signaling, eIF4E phosphorylation and the expression of MMP-9. Thus, metformin is a potential FXS therapeutic.

Original languageEnglish (US)
Pages (from-to)674-677
Number of pages4
JournalNature Medicine
Volume23
Issue number6
DOIs
StatePublished - Jun 1 2017

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    Gantois, I., Khoutorsky, A., Popic, J., Aguilar-Valles, A., Freemantle, E., Cao, R., Sharma, V., Pooters, T., Nagpal, A., Skalecka, A., Truong, V. T., Wiebe, S., Groves, I. A., Jafarnejad, S. M., Chapat, C., McCullagh, E. A., Gamache, K., Nader, K., Lacaille, J. C., ... Sonenberg, N. (2017). Metformin ameliorates core deficits in a mouse model of fragile X syndrome. Nature Medicine, 23(6), 674-677. https://doi.org/10.1038/nm.4335