Vanderbilt Department of Pharmacology and Vanderbilt Center for Neuroscience Drug Discovery, Vanderbilt University, Nashville, TN, USA.
Vanderbilt Department of Pharmacology and Vanderbilt Center for Neuroscience Drug Discovery, Vanderbilt University, Nashville, TN, USA; Vanderbilt Institute of Chemical Biology, Vanderbilt University, Nashville, TN, USA; Vanderbilt Kennedy Center, Vanderbilt University Medical Center, Nashville, TN, USA.
Trends Pharmacol Sci. 2019 Apr;40(4):233-236. doi: 10.1016/j.tips.2019.02.007.
Rett syndrome (RTT) is a neurodevelopmental disorder caused by mutations in the Methyl CpG binding protein 2 (MeCP2) gene. This Science & Society article focuses on pharmacological strategies that attack RTT treatment from multiple angles, including drug repurposing and de novo discovery efforts, and discusses the impacts of preclinical study design and translationally relevant outcome measures.
雷特综合征（RTT）是一种神经发育障碍，由甲基化CpG 结合蛋白 2（MeCP2）基因突变引起。本期《科学与社会》聚焦于从多个角度攻克 RTT 治疗的药理学策略，包括药物再利用和从头发现努力，并讨论了临床前研究设计和转化相关结果衡量指标的影响。
