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

I Gantois, A Khoutorsky, J Popic, A Aguilar-Valles, E Freemantle, R Cao, V Sharma, T Pooters, A Nagpal, A Skalecka, VT Truong, S Wiebe, IA Groves, Seyed Mehdi Jafarnejad Shourkaei, Clément Chapat, Elizabeth A McCullagh, Karine Gamache, Karim Nader, Jean-Claude Lacaille, Christos G GkogkasN Sonenberg

Research output: Contribution to journalArticlepeer-review

76 Citations (Scopus)

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
Pages (from-to)1-7
JournalNature Medicine
Early online date15 May 2017
DOIs
Publication statusPublished - 06 Jun 2017
Externally publishedYes

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