Article

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

2017-04-21

Abstract excerpt

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 neurodegeneration. Studies in model organisms, including yeast and flies have converged upon nucleocytoplasmic transport as one...

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Literature Corpus work: a940ae8f-372e-5ba3-a623-62260da8e4b8
DOI: 10.1101/129254

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