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A blood–brain penetrant RNA-targeted small molecule triggers elimination of r(G(4)C(2))(exp) in c9ALS/FTD via the nuclear RNA exosome

A hexanucleotide repeat expansion in intron 1 of the C9orf72 gene is the most common genetic cause of amyotrophic lateral sclerosis and frontotemporal dementia, or c9ALS/FTD. The RNA transcribed from the expansion, r(G(4)C(2))(exp), causes various pathologies, including intron retention, aberrant tr...

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Detalles Bibliográficos
Autores principales:	Bush, Jessica A., Meyer, Samantha M., Fuerst, Rita, Tong, Yuquan, Li, Yue, Benhamou, Raphael I., Aikawa, Haruo, Zanon, Patrick R. A., Gibaut, Quentin M. R., Angelbello, Alicia J., Gendron, Tania F., Zhang, Yong-Jie, Petrucelli, Leonard, Heick Jensen, Torben, Childs-Disney, Jessica L., Disney, Matthew D.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	National Academy of Sciences 2022
Materias:	Biological Sciences
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9860304/ https://www.ncbi.nlm.nih.gov/pubmed/36409902 http://dx.doi.org/10.1073/pnas.2210532119

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