RNA Therapeutics Institute; Program in Bioinformatics and Integrative Biology; Wellstone Muscular Dystrophy Program, Department of Neurology; Program in Molecular Medicine; Department of Biochemistry and Molecular Pharmacology; Department of Molecular, Cell and Cancer Biology
Biochemistry, Biophysics, and Structural Biology | Bioinformatics | Computational Biology | Integrative Biology | Systems Biology
CRISPR is widely used to disrupt gene function by inducing small insertions and deletions. Here, we show that some single-guide RNAs (sgRNAs) can induce exon skipping or large genomic deletions that delete exons. For example, CRISPR-mediated editing of beta-catenin exon 3, which encodes an autoinhibitory domain, induces partial skipping of the in-frame exon and nuclear accumulation of beta-catenin. A single sgRNA can induce small insertions or deletions that partially alter splicing or unexpected larger deletions that remove exons. Exon skipping adds to the unexpected outcomes that must be accounted for, and perhaps taken advantage of, in CRISPR experiments.
CRISPR/Cas9 genome editing, single-guide RNAs, sgRNAs, exon skipping
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Copyright © The Author(s). 2017.
DOI of Published Version
Genome Biol. 2017 Jun 14;18(1):108. doi: 10.1186/s13059-017-1237-8. Link to article on publisher's site
Mou H, Smith JL, Peng L, Moore J, Zhang X, Song C, Sheel A, Ozata DM, Li Y, Emerson CP, Sontheimer EJ, Moore MJ, Weng Z, Xue W. (2017). CRISPR/Cas9-mediated genome editing induces exon skipping by alternative splicing or exon deletion. Program in Bioinformatics and Integrative Biology Publications. https://doi.org/10.1186/s13059-017-1237-8. Retrieved from https://escholarship.umassmed.edu/bioinformatics_pubs/111
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