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

C9orf72-associated FTD/ALS: when less is more

Peter K Todd  1 , Henry L Paulson
Affiliations
Editorial

C9orf72-associated FTD/ALS: when less is more

Peter K Todd et al. Neuron. .

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.

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Comment on

  • RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention.
    Donnelly CJ, Zhang PW, Pham JT, Haeusler AR, Mistry NA, Vidensky S, Daley EL, Poth EM, Hoover B, Fines DM, Maragakis N, Tienari PJ, Petrucelli L, Traynor BJ, Wang J, Rigo F, Bennett CF, Blackshaw S, Sattler R, Rothstein JD. Donnelly CJ, et al. Neuron. 2013 Oct 16;80(2):415-28. doi: 10.1016/j.neuron.2013.10.015. Neuron. 2013. PMID: 24139042 Free PMC article.

References

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