Article
Topoisomerase inhibitors unsilence the dormant allele of Ube3a in neurons.
Nature - 21 Dec 2011
Huang Hsien-Sung, Allen John A, Mabb Angela M, King Ian F, Miriyala Jayalakshmi, Taylor-Blake Bonnie, Sciaky Noah, Dutton J Walter, Lee Hyeong-Min, Chen Xin, Jin Jian, Bridges Arlene S, Zylka Mark J, Roth Bryan L, Philpot Benjamin D
Abstract excerpt
Angelman syndrome is a severe neurodevelopmental disorder caused by deletion or mutation of the maternal allele of the ubiquitin protein ligase E3A (UBE3A). In neurons, the paternal allele of UBE3A is intact but epigenetically silenced, raising the possibility that Angelman syndrome could be treated by activating this silenced allele to restore functional UBE3A protein. Using an unbiased, high-content screen in...
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