VPA alleviates neurological deficits and restores gene expression in a mouse model of rett syndrome

Weixiang Guo, Keita Tsujimura, Maky Otsuka I, Koichiro Irie, Katsuhide Igarashi, Kinichi Nakashima, Xinyu Zhao

Research output: Contribution to journalArticle

10 Citations (Scopus)

Abstract

Rett syndrome (RTT) is a devastating neurodevelopmental disorder that occurs once in every 10,000-15,000 live female births. Despite intensive research, no effective cure is yet available. Valproic acid (VPA) has been used widely to treat mood disorder, epilepsy, and a growing number of other disorders. In limited clinical studies, VPA has also been used to control seizure in RTT patients with promising albeit somewhat unclear efficacy. In this study we tested the effect of VPA on the neurological symptoms of RTT and discovered that short-term VPA treatment during the symptomatic period could reduce neurological symptoms in RTT mice. We found that VPA restores the expression of a subset of genes in RTT mouse brains, and these genes clustered in neurological disease and developmental disorder networks. Our data suggest that VPA could be used as a drug to alleviate RTT symptoms.

Original languageEnglish
Article numbere100215
JournalPloS one
Volume9
Issue number6
DOIs
Publication statusPublished - Jun 26 2014

All Science Journal Classification (ASJC) codes

  • Biochemistry, Genetics and Molecular Biology(all)
  • Agricultural and Biological Sciences(all)
  • General

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