| First Author | Gantois I | Year | 2017 |
| Journal | Nat Med | Volume | 23 |
| Issue | 6 | Pages | 674-677 |
| PubMed ID | 28504725 | Mgi Jnum | J:251854 |
| Mgi Id | MGI:6103471 | Doi | 10.1038/nm.4335 |
| Citation | Gantois I, et al. (2017) Metformin ameliorates core deficits in a mouse model of fragile X syndrome. Nat Med 23(6):674-677 |
| abstractText | 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. |
