RNA Toxicity from the ALS/FTD C9ORF72 Expansion Is Mitigated by Antisense Intervention

SUMMARY: A hexanucleotide GGGGCC repeat expansion in the noncoding region of the C9ORF72 gene is the most common genetic abnormality in familial and sporadic amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The function of the C9ORF72 protein is unknown, as is the mechanism by...

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Autori principali: Donnelly, Christopher J., Zhang, Ping-Wu, Pham, Jacqueline T., Heusler, Aaron R., Mistry, Nipun A., Vidensky, Svetlana, Daley, Elizabeth L., Poth, Erin M., Hoover, Benjamin, Fines, Daniel M., Maragakis, Nicholas, Tienari, Pentti J., Petrucelli, Leonard, Traynor, Bryan J., Wang, Jiou, Rigo, Frank, Bennett, C. Frank, Blackshaw, Seth, Sattler, Rita, Rothstein, Jeffrey D.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2013
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4098943/
https://ncbi.nlm.nih.gov/pubmed/24139042
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.neuron.2013.10.015
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