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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Udgivet i: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
Format: Artigo
Sprog:Inglês
Udgivet: 2016
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Article
Online adgang: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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