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Natl. Acad. Sci. U.S.A."],"published-print":{"date-parts":[[2017,10,31]]},"abstract":"<jats:title>Significance<\/jats:title>\n                  <jats:p>\n                    Rett syndrome (RTT) is a neurological disease caused by mutations in the gene encoding the global transcriptional regulator, Methyl CpG Binding Protein 2 (MECP2). We exploit a strategy to repair mutant\n                    <jats:italic>Mecp2<\/jats:italic>\n                    mRNA that if successful should reverse symptoms. The strategy utilizes the catalytic activity of a naturally occurring enzyme, Adenosine Deaminase Acting on RNA (ADAR2), which in brain alters the mRNA sequence and function of proteins. In cultured RTT neurons co-expressing a modified ADAR2 protein and an appropriate RNA guide, a human mutation in\n                    <jats:italic>Mecp2<\/jats:italic>\n                    mRNA is repaired efficiently. RNA repair restores MeCP2 function, consistent with reversal of the pathological consequences of the RTT mutation. Our strategy holds promise for new therapeutic approaches to RTT and other neurological diseases.\n                  <\/jats:p>","DOI":"10.1073\/pnas.1715320114","type":"journal-article","created":{"date-parts":[[2017,10,16]],"date-time":"2017-10-16T15:21:03Z","timestamp":1508167263000},"update-policy":"https:\/\/doi.org\/10.1073\/pnas.cm10313","source":"Crossref","is-referenced-by-count":103,"title":["Site-directed RNA repair of endogenous Mecp2 RNA in neurons"],"prefix":"10.1073","volume":"114","author":[{"given":"John R.","family":"Sinnamon","sequence":"first","affiliation":[{"name":"Vollum Institute, Oregon Health and Science University, Portland, OR 97239;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan Y.","family":"Kim","sequence":"additional","affiliation":[{"name":"Vollum Institute, Oregon Health and Science University, Portland, OR 97239;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Glen M.","family":"Corson","sequence":"additional","affiliation":[{"name":"Vollum Institute, Oregon Health and Science University, Portland, OR 97239;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhen","family":"Song","sequence":"additional","affiliation":[{"name":"Department of Molecular and Medical Genetics, Oregon Health and Science University, Portland, OR 97239"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hiroyuki","family":"Nakai","sequence":"additional","affiliation":[{"name":"Department of Molecular and Medical Genetics, Oregon Health and Science University, Portland, OR 97239"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John P.","family":"Adelman","sequence":"additional","affiliation":[{"name":"Vollum Institute, Oregon Health and Science University, Portland, OR 97239;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gail","family":"Mandel","sequence":"additional","affiliation":[{"name":"Vollum Institute, Oregon Health and Science University, Portland, OR 97239;"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"341","published-online":{"date-parts":[[2017,10,16]]},"reference":[{"key":"e_1_3_3_1_2","doi-asserted-by":"publisher","DOI":"10.1038\/13810"},{"key":"e_1_3_3_2_2","doi-asserted-by":"publisher","DOI":"10.1002\/ana.22124"},{"key":"e_1_3_3_3_2","doi-asserted-by":"publisher","DOI":"10.1002\/ana.22154"},{"key":"e_1_3_3_4_2","doi-asserted-by":"publisher","DOI":"10.1111\/j.1399-0004.2008.01005.x"},{"key":"e_1_3_3_5_2","doi-asserted-by":"publisher","DOI":"10.1038\/85899"},{"key":"e_1_3_3_6_2","doi-asserted-by":"publisher","DOI":"10.1038\/nature10214"},{"key":"e_1_3_3_7_2","doi-asserted-by":"publisher","DOI":"10.1038\/85906"},{"key":"e_1_3_3_8_2","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0401626101"},{"key":"e_1_3_3_9_2","first-page":"4389","article-title":"Exclusive expression of MeCP2 in the nervous system distinguishes between brain and peripheral Rett syndrome-like phenotypes","volume":"25","author":"Ross PD","year":"2016","unstructured":"PD Ross, , Exclusive expression of MeCP2 in the nervous system distinguishes between brain and peripheral Rett syndrome-like phenotypes. 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