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CRISPR-SKIP: programmable gene splicing with single base editors.

ABSTRACT: CRISPR gene editing has revolutionized biomedicine and biotechnology by providing a simple means to engineer genes through targeted double-strand breaks in the genomic DNA of living cells. However, given the stochasticity of cellular DNA repair mechanisms and the potential for off-target mutations, technologies capable of introducing targeted changes with increased precision, such as single-base editors, are preferred. We present a versatile method termed CRISPR-SKIP that utilizes cytidine deaminase single-base editors to program exon skipping by mutating target DNA bases within splice acceptor sites. Given its simplicity and precision, CRISPR-SKIP will be broadly applicable in gene therapy and synthetic biology.

SUBMITTER: Gapinske M 

PROVIDER: S-EPMC6092781 | biostudies-literature | 2018 Aug

REPOSITORIES: biostudies-literature

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CRISPR-SKIP: programmable gene splicing with single base editors.

Gapinske Michael M   Luu Alan A   Winter Jackson J   Woods Wendy S WS   Kostan Kurt A KA   Shiva Nikhil N   Song Jun S JS   Perez-Pinera Pablo P  

Genome biology 20180815 1

CRISPR gene editing has revolutionized biomedicine and biotechnology by providing a simple means to engineer genes through targeted double-strand breaks in the genomic DNA of living cells. However, given the stochasticity of cellular DNA repair mechanisms and the potential for off-target mutations, technologies capable of introducing targeted changes with increased precision, such as single-base editors, are preferred. We present a versatile method termed CRISPR-SKIP that utilizes cytidine deami  ...[more]

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