Discovery of a Biomarker and Lead Small Molecules to Target r(GGGGCC)-Associated Defects in c9FTD/ALS

A repeat expansion in C9ORF72 causes frontotemporal dementia and amyotrophic lateral sclerosis (c9FTD/ALS). RNA of the expanded repeat (r(GGGGCC)(exp)) forms nuclear foci or undergoes repeat-associated non-ATG (RAN) translation producing “c9RAN proteins”. Since neutralizing r(GGGGCC)(exp) could inhi...

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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Neuron
Prif Awduron: Su, Zhaoming, Zhang, Yongjie, Gendron, Tania F., Bauer, Peter O., Chew, Jeannie, Yang, Wang-Yong, Fostvedt, Erik, Jansen-West, Karen, Belzil, Veronique V., Desaro, Pamela, Johnston, Amelia, Overstreet, Karen, Oh, Seok-Yoon, Todd, Peter K., Berry, James D., Cudkowicz, Merit E., Boeve, Bradley F., Dickson, Dennis, Floeter, Mary Kay, Traynor, Bryan J., Morelli, Claudia, Ratti, Antonia, Silani, Vincenzo, Rademakers, Rosa, Brown, Robert H., Rothstein, Jeffrey D., Boylan, Kevin B., Petrucelli, Leonard, Disney, Matthew D.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: 2014
Pynciau:
Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC4232217/
https://ncbi.nlm.nih.gov/pubmed/25132468
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.neuron.2014.07.041
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