CRISPR-Cas9 screens in human cells and primary neurons identify modifiers of C9orf72 dipeptide repeat protein toxicity

Hexanucleotide repeat expansions in the C9orf72 gene are the most common cause of amyotrophic lateral sclerosis and frontotemporal dementia (c9FTD/ALS). The nucleotide repeat expansions are translated into dipeptide repeat (DPR) proteins, which are aggregation-prone and may contribute to neurodegene...

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Vydáno v:Nat Genet
Hlavní autoři: Kramer, Nicholas J., Haney, Michael S., Morgens, David W., Jovičić, Ana, Couthouis, Julien, Li, Amy, Ousey, James, Ma, Rosanna, Bieri, Gregor, Tsui, C. Kimberly, Shi, Yingxiao, Hertz, Nicholas T., Tessier-Lavigne, Marc, Ichida, Justin K., Bassik, Michael C., Gitler, Aaron D.
Médium: Artigo
Jazyk:Inglês
Vydáno: 2018
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5893388/
https://ncbi.nlm.nih.gov/pubmed/29507424
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41588-018-0070-7
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