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Two therapeutic CRISPR/Cas9 gene editing approaches revert FTD/ALS cellular pathology caused by a C9orf72 repeat expansion mutation in patient derived cells

2022-05-21

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CRISPR gene editing holds promise to cure or arrest genetic disease, if we can find and implement curative edits reliably, safely and effectively. Expansion of a hexanucleotide repeat in C9orf72 is the leading known genetic cause of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). We evaluated three approaches to editing the mutant C9orf72 gene for their ability to correct pathology in neur...

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Literature Corpus work: ff45d554-5431-5c0b-97c5-f38f442546a7
DOI: 10.1101/2022.05.21.492887

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Two therapeutic CRISPR/Cas9 gene editing approaches revert FTD/ALS cellular pathology caused by a <i>C9orf72</i> repeat expansion mutation in patient derived cells

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