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Natl. Acad. Sci. U.S.A."],"published-print":{"date-parts":[[2018,7,31]]},"abstract":"<jats:title>Significance<\/jats:title>\n                  <jats:p>\n                    Rett syndrome (RTT) is a devastating neurodevelopmental disorder with no cure. RTT typically affects females and is caused by mutations in one of the two copies of the X-linked gene\n                    <jats:italic>MECP2<\/jats:italic>\n                    . Due to X chromosome inactivation, the random silencing of one X chromosome, females with RTT have a mixture of cells that express either normal or mutant\n                    <jats:italic>MECP2<\/jats:italic>\n                    . Importantly, cells expressing mutant\n                    <jats:italic>MECP2<\/jats:italic>\n                    retain a normal copy of\n                    <jats:italic>MECP2<\/jats:italic>\n                    on the inactive X chromosome (Xi), the reactivation of which represents a potential therapeutic approach for RTT. Here we use small-molecule inhibitors to reactivate the Xi-linked\n                    <jats:italic>Mecp2<\/jats:italic>\n                    gene in brain neurons of living mice. Our results further establish the feasibility of Xi-linked\n                    <jats:italic>MECP2<\/jats:italic>\n                    reactivation-based therapeutics for treatment of RTT.\n                  <\/jats:p>","DOI":"10.1073\/pnas.1803792115","type":"journal-article","created":{"date-parts":[[2018,7,16]],"date-time":"2018-07-16T15:05:50Z","timestamp":1531753550000},"page":"7991-7996","update-policy":"https:\/\/doi.org\/10.1073\/pnas.cm10313","source":"Crossref","is-referenced-by-count":56,"title":["Pharmacological reactivation of inactive X-linked\n                    <i>Mecp2<\/i>\n                    in cerebral cortical neurons of living mice"],"prefix":"10.1073","volume":"115","author":[{"given":"Piotr","family":"Przanowski","sequence":"first","affiliation":[{"name":"Department of Biochemistry and Molecular Genetics, University of Virginia School of Medicine, Charlottesville, VA 22908;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Urszula","family":"Wasko","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Molecular Genetics, University of Virginia School of Medicine, Charlottesville, VA 22908;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zeming","family":"Zheng","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Molecular Genetics, University of Virginia School of Medicine, Charlottesville, VA 22908;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jun","family":"Yu","sequence":"additional","affiliation":[{"name":"Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA 01605;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robyn","family":"Sherman","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Molecular Genetics, University of Virginia School of Medicine, Charlottesville, VA 22908;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lihua Julie","family":"Zhu","sequence":"additional","affiliation":[{"name":"Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA 01605;"},{"name":"Programs in Molecular Medicine and Bioinformatics and Integrative Biology, University of Massachusetts Medical School, Worcester, MA 01605;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael J.","family":"McConnell","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Molecular Genetics, University of Virginia School of Medicine, Charlottesville, VA 22908;"},{"name":"Department of Neuroscience, University of Virginia School of Medicine, Charlottesville, VA 22908;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jogender","family":"Tushir-Singh","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and 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