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CRISPR-SKIP: programmable gene splicing with single base editors

Authors :
Nikhil Shiva
Wendy S. Woods
Pablo Perez-Pinera
Alan Luu
Jun S. Song
Michael Gapinske
Kurt A. Kostan
Jackson Winter
Source :
Genome Biology, Genome Biology, Vol 19, Iss 1, Pp 1-11 (2018)
Publication Year :
2018
Publisher :
Springer Science and Business Media LLC, 2018.

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. Electronic supplementary material The online version of this article (10.1186/s13059-018-1482-5) contains supplementary material, which is available to authorized users.

Details

ISSN :
1474760X
Volume :
19
Database :
OpenAIRE
Journal :
Genome Biology
Accession number :
edsair.doi.dedup.....77a90eab7f30ae715d9d1fe40adfd197