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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...
| Autores principales: | , , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
National Academy of Sciences
2022
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| 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 |