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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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: 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
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: National Academy of Sciences 2013
Pynciau:
PNAS Plus
Mynediad Ar-lein: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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