. Poly-dipeptides encoded by the C9orf72 repeats bind nucleoli, impede RNA biogenesis, and kill cells. Science. 2014 Sep 5;345(6201):1139-45. Epub 2014 Jul 31 PubMed.


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  1. McKnight and colleagues report that C9ORF72 RANT dipeptide products can physically interact with the nucleolus and impair rRNA processing. This is a new pathway leading to nucleolar defects in addition to the previously reported pathway in which C9ORF72 repeat RNA disrupts the nucleolus and impairs its rRNA processing functions. The study highlights the possibility that the nucleolus is a hub for C9ORF72 pathogenic cascades. With insights from the current work and rapid advances in understanding C9ORF72 diseases, treatment targets may be directed at the major sites of cellular defects or, perhaps more effectively, at upstream molecular events.

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  1. C9ORF72 Killer Dipeptides Clog the Nucleolus