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.
Details
Title
Targeted RNA editing in brainstem alleviates respiratory dysfunction in a mouse model of rett syndrome
Creator
Sinnamon, John R. : Oregon Health and Science University Jacobson, Michael E. : Oregon Health and Science University Yung, John F. : Oregon Health and Science University Fisk, Jenna R. : Oregon Health and Science University Jeng, Sophia : Oregon Health and Science University McWeeney, Shannon K. : Oregon Health and Science University Parmelee, Lindsay K. : Oregon Health and Science University Chan, Chi Ngai : Oregon Health and Science University Yee, Siu-Pok : Oregon Health and Science University Kragness, Sabrina : Oregon Health and Science University Mandel, Gail : Oregon Health and Science University
Meeting Name
Research Week, Oregon Health and Science University, 2023
