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CRISPR-Cas9 targeted deletion of the C9orf72 repeat expansion mutation corrects cellular phenotypes in patient-derived iPS cells

2016-05-02

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The large hexanucleotide (GGGGCC) repeat expansion in the non-coding promoter region of C9orf72 is the leading cause of Frontotemporal Dementia (FTD) and Amyotrophic Lateral Sclerosis (ALS). Mechanisms underlying neurodegeneration are not clear, and both a C9orf72 loss of function and a gain of toxicity, in the form of RNA foci or dipeptide repeat deposition, are implicated. CRISPR (clustered regularly interspace...

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Literature Corpus work: e20760ad-524a-5f8f-bb11-bf74c84f37d1
DOI: 10.1101/051193

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CRISPR-Cas9 targeted deletion of the <i>C9orf72</i> repeat expansion mutation corrects cellular phenotypes in patient-derived iPS cells

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