Aptazyme-mediated direct modulation of post-transcriptional sgRNA level for conditional genome editing and gene expression

Hao Chen, Yanqing Li, Chunhua Du, Yan Li, Junli Zhao, Xiaojing Zheng, Qinwen Mao, Haibin Xia*

*Corresponding author for this work

Research output: Contribution to journalArticle

4 Scopus citations

Abstract

RNA-guided endonuclease Cas9 derived from microbial CRISPR-Cas adaptive immune systems is a powerful tool for genome editing, which has been widely used in eukaryotic systems, prokaryotic systems, and plants. However, the off-target effects caused by Cas9/sgRNA remain a major concern. Currently, the efforts to reduce the off-target effects mainly focus on improving the targeting specificity of sgRNA/Cas9, regulating the activity of the Cas9 protein or the sgRNA, and controlling the time window of their expression. In this study, a novel system was established to regulate the post-transcriptional sgRNA level by small molecule-controlled aptazyme. This system was shown to reduce the off-target effects caused by Cas9/sgRNA, while enabling precise temporal control over gene editing and regulatory activity. This new system could provide a potentially safer and more powerful tool for genome editing and therapeutic application.

Original languageEnglish (US)
Pages (from-to)23-29
Number of pages7
JournalJournal of Biotechnology
Volume288
DOIs
StatePublished - Dec 20 2018

Keywords

  • Aptazyme
  • Cas9
  • Genome editing
  • Off-target effect
  • Theophylline
  • sgRNA

ASJC Scopus subject areas

  • Biotechnology
  • Bioengineering
  • Applied Microbiology and Biotechnology

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