Department of Bioengineering, University of Illinois at Urbana-Champaign, 1406 West Green Street, Urbana, 61801-2918, IL, USA.
Department of Physics, University of Illinois at Urbana-Champaign, 1110 West Green Street, Urbana, IL, 61801-3080, USA.
Genome Biol. 2018 Aug 15;19(1):107. doi: 10.1186/s13059-018-1482-5.
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.
CRISPR 基因编辑通过提供一种简单的方法,在活细胞的基因组 DNA 中靶向双链断裂,从而彻底改变了生物医学和生物技术。然而,鉴于细胞 DNA 修复机制的随机性和潜在的脱靶突变,人们更喜欢能够以更高的精度引入靶向变化的技术,如单碱基编辑器。我们提出了一种称为 CRISPR-SKIP 的多功能方法,该方法利用胞嘧啶脱氨酶单碱基编辑器通过突变剪接受体位点内的靶 DNA 碱基来编程外显子跳跃。鉴于其简单性和精确性,CRISPR-SKIP 将在基因治疗和合成生物学中得到广泛应用。