TY - JOUR
T1 - VPA alleviates neurological deficits and restores gene expression in a mouse model of rett syndrome
AU - Guo, Weixiang
AU - Tsujimura, Keita
AU - Otsuka I, Maky
AU - Irie, Koichiro
AU - Igarashi, Katsuhide
AU - Nakashima, Kinichi
AU - Zhao, Xinyu
PY - 2014/6/26
Y1 - 2014/6/26
N2 - 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.
AB - 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.
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U2 - 10.1371/journal.pone.0100215
DO - 10.1371/journal.pone.0100215
M3 - Article
C2 - 24968028
AN - SCOPUS:84903398742
SN - 1932-6203
VL - 9
JO - PloS one
JF - PloS one
IS - 6
M1 - e100215
ER -