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Neuron. 2013 Oct 16;80(2):257-8. doi: 10.1016/j.neuron.2013.10.010.

C9orf72-associated FTD/ALS: when less is more.

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1
Department of Neurology, University of Michigan Medical Center, Ann Arbor, MI 48109, USA.

Abstract

Hexanucleotide repeat expansions in C9ORF72 cause neurodegeneration in FTD and ALS by unknown mechanisms. A new report, by Donnelly et al. (2013), finds that these repeats trigger a pathogenic gain-of-function cascade that can be corrected by suppressing expression of the repeat transcript, paving the way for therapeutic strategies aimed at eliminating the toxic RNA.

PMID:
24139028
PMCID:
PMC4982762
DOI:
10.1016/j.neuron.2013.10.010
[Indexed for MEDLINE]
Free PMC Article
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