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C9orf72 associated FTD/ALS: When less is more

Hexanucleotide repeat expansions in C9ORF72 cause neurodegeneration in FTD and ALS by unknown mechanisms. A new report 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 strat...

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Publicat a:Neuron
Autors principals: Todd, Peter K., Paulson, Henry L.
Format: Artigo
Idioma:Inglês
Publicat: 2013
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4982762/
https://ncbi.nlm.nih.gov/pubmed/24139028
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.neuron.2013.10.010
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