Ontology highlight
ABSTRACT:
SUBMITTER: Su Z
PROVIDER: S-EPMC4232217 | biostudies-literature | 2014 Sep
REPOSITORIES: biostudies-literature
Su Zhaoming Z Zhang Yongjie Y Gendron Tania F TF Bauer Peter O PO Chew Jeannie J Yang Wang-Yong WY Fostvedt Erik E Jansen-West Karen K Belzil Veronique V VV Desaro Pamela P Johnston Amelia A Overstreet Karen K Oh Seok-Yoon SY Todd Peter K PK Berry James D JD Cudkowicz Merit E ME Boeve Bradley F BF Dickson Dennis D Floeter Mary Kay MK Traynor Bryan J BJ Morelli Claudia C Ratti Antonia A Silani Vincenzo V Rademakers Rosa R Brown Robert H RH Rothstein Jeffrey D JD Boylan Kevin B KB Petrucelli Leonard L Disney Matthew D MD
Neuron 20140814 5
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 inhibit these potentially toxic events, we sought to identify small-molecule binders of r(GGGGCC)exp. Chemical and enzymatic probing of r(GGGGCC)8 indicate that it adopts a hairpin structure in equilibrium w ...[more]