Article
Bromodomain inhibitors regulate the C9ORF72 locus in ALS.
Experimental neurology - 1 Sept 2015
Zeier Zane, Esanov Rustam, Belle Kinsley C, Volmar Claude-Henry, Johnstone Andrea L, Halley Paul, DeRosa Brooke A, Khoury Nathalie, van Blitterswijk Marka, Rademakers Rosa, Albert Jeffrey, Brothers Shaun P, Wuu Joanne, Dykxhoorn Derek M, Benatar Michael, Wahlestedt Claes
Abstract excerpt
A hexanucleotide repeat expansion residing within the C9ORF72 gene represents the most common known cause of amyotrophic lateral sclerosis (ALS) and places the disease among a growing family of repeat expansion disorders. The presence of RNA foci, repeat-associated translation products, and sequestration of RNA binding proteins suggests that toxic RNA gain-of-function contributes to pathology while C9ORF72...
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