000041889 001__ 41889 000041889 005__ 20240417091813.0 000041889 0247_ $$2doi$$a10.6083/bpxhc41889 000041889 037__ $$aIR 000041889 041__ $$aeng 000041889 245__ $$aTargeted RNA editing in brainstem alleviates respiratory dysfunction in a mouse model of rett syndrome 000041889 260__ $$bOregon Health and Science University 000041889 269__ $$a2023-09-13 000041889 336__ $$aAbstract 000041889 520__ $$aRett syndrome is a neurological disease due to loss-of-function mutations in the transcription factor, Methyl CpG binding protein 2 (MECP2). Because overexpression of endogenous MECP2 also causes disease, we have exploited a targeted RNA-editing approach to repair patient mutations where levels of MECP2 protein will never exceed endogenous levels. 000041889 540__ $$fCC BY 000041889 542__ $$fIn copyright - joint owners 000041889 650__ $$aRett Syndrome$$028468 000041889 650__ $$aNervous System Diseases$$022809 000041889 650__ $$aMutation$$022555 000041889 650__ $$aRNA Editing$$029934 000041889 650__ $$aDevelopmental Disabilities$$016472 000041889 650__ $$aHumans$$020376 000041889 650__ $$aNervous System Diseases$$022809 000041889 650__ $$aNeurodevelopmental Disorders$$040742 000041889 650__ $$aMethyl-CpG-Binding Protein 2$$037001 000041889 6531_ $$aMECP2 000041889 6531_ $$atargeted RNA editing 000041889 692__ $$aVollum Institute$$041509 000041889 7001_ $$aSinnamon, John R.$$uOregon Health and Science University$$041354 000041889 7001_ $$aJacobson, Michael E.$$uOregon Health and Science University$$041354 000041889 7001_ $$aYung, John F.$$uOregon Health and Science University$$041354 000041889 7001_ $$aFisk, Jenna R.$$uOregon Health and Science University$$041354 000041889 7001_ $$aJeng, Sophia$$uOregon Health and Science University$$041354 000041889 7001_ $$aMcWeeney, Shannon K.$$uOregon Health and Science University$$041354 000041889 7001_ $$aParmelee, Lindsay K.$$uOregon Health and Science University$$041354 000041889 7001_ $$aChan, Chi Ngai$$uOregon Health and Science University$$041354 000041889 7001_ $$aYee, Siu-Pok$$uOregon Health and Science University$$041354 000041889 7001_ $$aKragness, Sabrina$$uOregon Health and Science University$$041354 000041889 7001_ $$aMandel, Gail$$uOregon Health and Science University$$041354 000041889 711__ $$aResearch Week$$uOregon Health and Science University$$d2023 000041889 8564_ $$916e6450b-c4d5-464f-b92f-8f99cd1df29c$$s300492$$uhttps://digitalcollections.ohsu.edu/record/41889/files/ResearchWeek.2023.Yung.John.pdf 000041889 980__ $$aResearch Week 000041889 981__ $$aPublished$$b2023-09-13