Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration

Expanded hexanucleotide repeats in the chromosome 9 open reading frame 72 (C9orf72) gene are the most common genetic cause of ALS and frontotemporal degeneration (FTD). Here, we identify nuclear RNA foci containing the hexanucleotide expansion (GGGGCC) in patient cells, including white blood cells,...

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Detalhes bibliográficos
Main Authors: Lagier-Tourenne, Clotilde, Baughn, Michael, Rigo, Frank, Sun, Shuying, Liu, Patrick, Li, Hai-Ri, Jiang, Jie, Watt, Andrew T., Chun, Seung, Katz, Melanie, Qiu, Jinsong, Sun, Ying, Ling, Shuo-Chien, Zhu, Qiang, Polymenidou, Magdalini, Drenner, Kevin, Artates, Jonathan W., McAlonis-Downes, Melissa, Markmiller, Sebastian, Hutt, Kasey R., Pizzo, Donald P., Cady, Janet, Harms, Matthew B., Baloh, Robert H., Vandenberg, Scott R., Yeo, Gene W., Fu, Xiang-Dong, Bennett, C. Frank, Cleveland, Don W., Ravits, John
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2013
Assuntos:
PNAS Plus
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3839752/
https://ncbi.nlm.nih.gov/pubmed/24170860
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1318835110
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