Topoisomerase inhibitors unsilence the dormant allele of Ube3a in neurons

@inproceedings{Huang2011TopoisomeraseIU,
  title={Topoisomerase inhibitors unsilence the dormant allele of Ube3a in neurons},
  author={Hsien-Sung Huang and John A. Allen and Angela M. Mabb and Ian F. G. King and Jayalakshmi Miriyala and Bonnie Taylor-Blake and Noah Sciaky and J. Walter Dutton and Hyeong-min Lee and Xin Jane Chen and Jian Jin and Arlene S. Bridges and Mark J. Zylka and Bryan L. Roth and Benjamin D Philpot},
  booktitle={Nature},
  year={2011}
}
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 primary cortical neurons from mice, we identify twelve topoisomerase I inhibitors… CONTINUE READING
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