title: VPA alleviates neurological deficits and restores gene expression in a mouse model of Rett syndrome.
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.
authors: Department of Neuroscience and Waisman Center, School of Medicine and Public Health, University of Wisconsin, Madison, Wisconsin, United States of America.
authors: Weixiang
authors: W
authors: Guo
authors: Department of Stem Cell Biology and Medicine, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan.
authors: Keita
authors: K
authors: Tsujimura
authors: Life Science Tokyo Advanced Research center (L-StaR), Hoshi University School of Pharmacy and Pharmaceutical Science, Tokyo, Japan.
authors: Maky
authors: M
authors: Otsuka I
authors: Department of Stem Cell Biology and Medicine, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan.
authors: Koichiro
authors: K
authors: Irie
authors: Life Science Tokyo Advanced Research center (L-StaR), Hoshi University School of Pharmacy and Pharmaceutical Science, Tokyo, Japan.
authors: Katsuhide
authors: K
authors: Igarashi
authors: Department of Stem Cell Biology and Medicine, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan.
authors: Kinichi
authors: K
authors: Nakashima
authors: Department of Neuroscience and Waisman Center, School of Medicine and Public Health, University of Wisconsin, Madison, Wisconsin, United States of America.
authors: Xinyu
authors: X
authors: Zhao
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D005260:Female; D005786:Gene Expression Regulation / Q000187:drug effects*; D055786:Gene Knockout Techniques; D051783:Methyl-CpG-Binding Protein 2 / Q000172:deficiency / Q000235:genetics; D051379:Mice; D008813:Mice, Inbred ICR; D009462:Neurology; D015518:Rett Syndrome / Q000188:drug therapy* / Q000235:genetics* / Q000473:pathology / Q000503:physiopathology; D013997:Time Factors; D014635:Valproic Acid / Q000494:pharmacology* / Q000627:therapeutic use
chemical_list: D051783:Methyl-CpG-Binding Protein 2; D014635:Valproic Acid