Article
Targeted RNA editing in brainstem alleviates respiratory dysfunction in a mouse model of Rett syndrome.
Proceedings of the National Academy of Sciences of the United States of America - 16 Aug 2022
Sinnamon John R, Jacobson Michael E, Yung John F, Fisk Jenna R, Jeng Sophia, McWeeney Shannon K, Parmelee Lindsay K, Chan Chi Ngai, Yee Siu-Pok, Mandel Gail
Abstract excerpt
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. Here, we have constructed adeno-associated viruses coexpressing a...
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