CRISPR-Cas9 screens identify the RNA helicase DDX3X as a repressor of C9ORF72 (GGGGCC)n repeat-associated non-AUG translation

Hexanucleotide GGGGCC repeat expansion in C9ORF72 is the most prevalent genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). One pathogenic mechanism is the aberrant accumulation of dipeptide repeat (DPR) proteins produced by the unconventional translation of expan...

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Veröffentlicht in:Neuron
Hauptverfasser: Cheng, Weiwei, Wang, Shaopeng, Zhang, Zhe, Morgens, David W., Hayes, Lindsey R., Lee, Soojin, Portz, Bede, Xie, Yongzhi, Nguyen, Baotram V., Haney, Michael S., Yan, Shirui, Dong, Daoyuan, Coyne, Alyssa N., Yang, Junhua, Xian, Fengfan, Cleveland, Don W., Qiu, Zhaozhu, Rothstein, Jeffrey D., Shorter, James, Gao, Fen-Biao, Bassik, Michael C., Sun, Shuying
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2019
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Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6895427/
https://ncbi.nlm.nih.gov/pubmed/31587919
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.neuron.2019.09.003
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