Journal Article

CRISPR-SKIP: programmable gene splicing with single base editors

Perez-Pinera, Pablo, Luu, Alan, Woods, Wendy S., Shiva, Nikhil, Kostan, Kurt A., Gapinske, Michael, Winter, Jackson, Song, Jun S.

2018

Overview

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.

• Publisher: BioMed Central,Springer Nature B.V,BMC
• Subject: Alternative splicing / Animal Genetics and Genomics / Base editing / Base Pairing - genetics / Base Sequence / Bioinformatics / Biomedical and Life Sciences / Biotechnology / Cell Line / Cloning / Clustered Regularly Interspaced Short Palindromic Repeats - genetics / Consensus Sequence - genetics / CRISPR / CRISPR-Cas9 / Cytidine deaminase / Deoxyribonucleic acid / DNA / DNA repair / Efficiency / Evolutionary Biology / Exon skipping / exons / Exons - genetics / Gene Editing / Gene expression / Gene therapy / Genome / Genomes / High-Throughput Nucleotide Sequencing / Human Genetics / Humans / Insights from Genome Editing / Life Sciences / medicine / Method / Microbial Genetics and Genomics / Muscular dystrophy / Mutation / Penicillin / Plant Genetics and Genomics / Plasmids / Proteins / RNA Splice Sites - genetics / Splicing / Stochasticity / Synthetic biology