Article

A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTD

2023-12-12

Abstract excerpt

<h4>ABSTRACT</h4> An abnormal expansion of a GGGGCC hexanucleotide repeat in the C9ORF72 gene is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), two debilitating neurodegenerative disorders driven in part by gain-of-function mechanisms involving transcribed forms of the repeat expansion. By utilizing a Cas13 variant with reduced collateral effects, we develo...

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Literature Corpus work: 26b1c9c8-bac9-57cb-95c9-b7f7f0beeeaa
DOI: 10.1101/2023.12.12.571328

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