Gain of toxicity from ALS/FTD-linked repeat expansions in C9ORF72 is alleviated by antisense oligonucleotides targeting GGGGCC-containing RNAs

Hexanucleotide expansions in C9ORF72 are the most frequent genetic cause of amyotrophic lateral sclerosis and frontotemporal dementia. Disease mechanisms were evaluated in mice expressing C9ORF72 RNAs with up to 450 GGGGCC repeats or with one or both C9orf72 alleles inactivated. Chronic 50% reductio...

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Publicado no:Neuron
Main Authors: Jiang, Jie, Zhu, Qiang, Gendron, Tania F., Saberi, Shahram, McAlonis-Downes, Melissa, Seelman, Amanda, Stauffer, Jennifer E., Jafar-nejad, Paymaan, Drenner, Kevin, Schulte, Derek, Chun, Seung, Sun, Shuying, Ling, Shuo-Chien, Myers, Brian, Engelhardt, Jeffery, Katz, Melanie, Baughn, Michael, Platoshyn, Oleksandr, Marsala, Martin, Watt, Andy, Heyser, Charles J, Ard, M. Colin, De Muynck, Louis, Daughrity, Lillian M., Swing, Deborah A., Tessarollo, Lino, Jung, Chris J., Delpoux, Arnaud, Utzschneider, Daniel T., Hedrick, Stephen M., de Jong, Pieter J., Edbauer, Dieter, Van Damme, Philip, Petrucelli, Leonard, Shaw, Christopher E., Bennett, C. Frank, Da Cruz, Sandrine, Ravits, John, Rigo, Frank, Cleveland, Don W., Lagier-Tourenne, Clotilde
Formato: Artigo
Idioma:Inglês
Publicado em: 2016
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4860075/
https://ncbi.nlm.nih.gov/pubmed/27112497
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.neuron.2016.04.006
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