CRISPR-SKIP: Programmable gene splicing with single base editors

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

Research output: Contribution to journalArticle

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 languageEnglish (US)
Article number107
JournalGenome biology
Volume19
Issue number1
DOIs
StatePublished - 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

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