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Targeted RNA editing in brainstem alleviates respiratory dysfunction in a mouse model of Rett syndrome

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 M...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: National Academy of Sciences 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9388114/
https://www.ncbi.nlm.nih.gov/pubmed/35939700
http://dx.doi.org/10.1073/pnas.2206053119