CRISPR-SKIP: Programmable gene splicing with single base editors | DuchenneXchange

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Scientific Articles

CRISPR-SKIP: Programmable gene splicing with single base editors

key information

source: Genome Biology

year: 2018

authors: Gapinske M, Luu A, Winter J, Woods WS, Kostan KA, Shiva N, Song JS, Perez-Pinera P

summary/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.

organisation: University of Illinois at Urbana-Champaign, USA; Carle Illinois College of Medicine, USA

DOI: 10.1186/s13059-018-1482-5

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