Journal
GENOME BIOLOGY
Volume 19, Issue -, Pages -Publisher
BMC
DOI: 10.1186/s13059-018-1482-5
Keywords
Gene editing; Base editing; Exon skipping; Alternative splicing; Synthetic biology; CRISPR-Cas9; RELA; PIK3CA; BRCA2; Gene isoform
Funding
- ZJU-Illinois Institute Research Program
- American Heart Association Scientist Development Grant [17SDG33650087]
- NIH [R01CA163336]
- NSF [0822613]
- National Science Foundation Graduate Research Fellowship Program [DGE-1746047]
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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.
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