TY - GEN AB - Rett 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. AD - Oregon Health and Science University AD - Oregon Health and Science University AD - Oregon Health and Science University AD - Oregon Health and Science University AD - Oregon Health and Science University AD - Oregon Health and Science University AD - Oregon Health and Science University AD - Oregon Health and Science University AD - Oregon Health and Science University AD - Oregon Health and Science University AD - Oregon Health and Science University AU - Sinnamon, John R. AU - Jacobson, Michael E. AU - Yung, John F. AU - Fisk, Jenna R. AU - Jeng, Sophia AU - McWeeney, Shannon K. AU - Parmelee, Lindsay K. AU - Chan, Chi Ngai AU - Yee, Siu-Pok AU - Kragness, Sabrina AU - Mandel, Gail DA - 2023-09-13 DO - 10.6083/bpxhc41889 DO - doi ID - 41889 KW - Rett Syndrome KW - Nervous System Diseases KW - Mutation KW - RNA Editing KW - Developmental Disabilities KW - Humans KW - Nervous System Diseases KW - Neurodevelopmental Disorders KW - Methyl-CpG-Binding Protein 2 KW - MECP2 KW - targeted RNA editing L1 - https://digitalcollections.ohsu.edu/record/41889/files/ResearchWeek.2023.Yung.John.pdf L2 - https://digitalcollections.ohsu.edu/record/41889/files/ResearchWeek.2023.Yung.John.pdf L4 - https://digitalcollections.ohsu.edu/record/41889/files/ResearchWeek.2023.Yung.John.pdf LA - eng LK - https://digitalcollections.ohsu.edu/record/41889/files/ResearchWeek.2023.Yung.John.pdf N2 - Rett 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. PB - Oregon Health and Science University PY - 2023-09-13 T1 - Targeted RNA editing in brainstem alleviates respiratory dysfunction in a mouse model of rett syndrome TI - Targeted RNA editing in brainstem alleviates respiratory dysfunction in a mouse model of rett syndrome UR - https://digitalcollections.ohsu.edu/record/41889/files/ResearchWeek.2023.Yung.John.pdf Y1 - 2023-09-13 ER -