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.
Original language | English (US) |
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Article number | 107 |
Journal | Genome biology |
Volume | 19 |
Issue number | 1 |
DOIs | |
State | Published - Aug 15 2018 |
Keywords
- Alternative splicing
- BRCA2
- Base editing
- CRISPR-Cas9
- Exon skipping
- Gene editing
- Gene isoform
- PIK3CA
- RELA
- Synthetic biology
ASJC Scopus subject areas
- Ecology, Evolution, Behavior and Systematics
- Genetics
- Cell Biology