| Literature DB >> 28504725 |
Ilse Gantois1,2, Arkady Khoutorsky1,2,3,4, Jelena Popic1,2, Argel Aguilar-Valles1,2,5,6, Erika Freemantle5,6, Ruifeng Cao1,2,7, Vijendra Sharma1,2, Tine Pooters8,9, Anmol Nagpal1,2, Agnieszka Skalecka1,2, Vinh T Truong1,2, Shane Wiebe1,2, Isabelle A Groves8,9, Seyed Mehdi Jafarnejad1,2, Clément Chapat1,2, Elizabeth A McCullagh10, Karine Gamache11, Karim Nader11, Jean-Claude Lacaille5,6, Christos G Gkogkas8,9, Nahum Sonenberg1,2.
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.Entities:
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Year: 2017 PMID： 28504725 DOI： 10.1038/nm.4335
Source DB: PubMed Journal: Nat Med ISSN： 1078-8956 Impact factor: 53.440