Shi KY, Mori E, Nizami ZF, Lin Y, Kato M, Xiang S, Wu LC, Ding M, Yu Y, Gall JG, McKnight SL. Toxic PRn poly-dipeptides encoded by the C9orf72 repeat expansion block nuclear import and export. Proc Natl Acad Sci U S A. 2017 Feb 14;114(7):E1111-E1117. Epub 2017 Jan 9 PubMed.
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This study examined the biochemical interaction between poly(PR) and the nuclear pore. They found that poly(PR) binds to polymeric forms of the FG repeat domain found in several nuclear pore proteins, providing molecular mechanistic insight into the toxicity of poly(PR). These interesting studies also suggest that blocking FG domain polymer formation may be a potential therapeutic approach for ALS/FTD with GGGGCC repeat expansion.
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