Molecular medicine of fragile X syndrome: based on known molecular mechanisms

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• 作者：Shi-Yu Luo ; Ling-Qian Wu ; Ran-Hui Duan
• 关键词：clinical trial ; fragile X syndrome ; mechanism ; based ; treatment
• 刊名：World Journal of Pediatrics
• 出版年：2016
• 出版时间：February 2016
• 年：2016
• 卷：12
• 期：1
• 页码：19-27
• 全文大小：608 KB
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• 作者单位：Shi-Yu Luo (1)
  Ling-Qian Wu (1)
  Ran-Hui Duan (1)
  
  1. State Key Laboratory of Medical Genetics & School of Life Sciences, Central South University, Changsha, China
  
• 刊物主题：Pediatrics; Pediatric Surgery; Maternal and Child Health; Intensive / Critical Care Medicine; Surgery; Imaging / Radiology;
• 出版者：Springer Berlin Heidelberg
• ISSN：1867-0687

文摘

Background Extensive research on fragile X mental retardation gene knockout mice and mutant Drosophila models has largely expanded our knowledge on mechanism-based treatment of fragile X syndrome (FXS). In light of these findings, several clinical trials are now underway for therapeutic translation to humans.