Fragile X syndrome (FXS) is the most frequent inherited form of intellectual disability and autism spectrum disorder. Loss of the fragile X mental retardation protein, FMRP, engenders molecular, behavioral, and cognitive deficits in FXS patients. Experiments using different animal models advanced our knowledge of the pathophysiology of FXS and led to the discovery of many targets for drug treatments. In this review, we discuss the potential of metformin, an antidiabetic drug approved by the US Food and Drug Administration, to correct core symptoms of FXS and other neurological disorders in humans. We summarize its mechanisms of action in different animal and cellular models and human diseases.

中文翻译：

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二甲双胍治疗脆性X综合征和其他神经系统疾病。

脆弱X综合征（FXS）是智力残疾和自闭症谱系障碍的最常见遗传形式。脆弱的X智力障碍蛋白FMRP的丢失会导致FXS患者出现分子，行为和认知缺陷。使用不同动物模型进行的实验提高了我们对FXS病理生理学的了解，并导致发现了许多药物治疗靶标。在这篇综述中，我们讨论了二甲双胍（一种由美国食品和药物管理局批准的抗糖尿病药）纠正人体内FXS和其他神经系统疾病的核心症状的潜力。我们总结了其在不同动物和细胞模型以及人类疾病中的作用机理。