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PLoS One. 2014 Jun 26;9(6):e100215. doi: 10.1371/journal.pone.0100215. eCollection 2014.

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

Author information

1
Department of Neuroscience and Waisman Center, School of Medicine and Public Health, University of Wisconsin, Madison, Wisconsin, United States of America.
2
Department of Stem Cell Biology and Medicine, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan.
3
Life Science Tokyo Advanced Research center (L-StaR), Hoshi University School of Pharmacy and Pharmaceutical Science, Tokyo, Japan.

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.

PMID:
24968028
PMCID:
PMC4072629
DOI:
10.1371/journal.pone.0100215
[Indexed for MEDLINE]
Free PMC Article

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