High-resolution characterization of gene function using single-cell CRISPR tiling screen

Nat Commun. 2021 Jul 1;12(1):4063. doi: 10.1038/s41467-021-24324-0.

Abstract

Identification of novel functional domains and characterization of detailed regulatory mechanisms in cancer-driving genes is critical for advanced cancer therapy. To date, CRISPR gene editing has primarily been applied to defining the role of individual genes. Recently, high-density mutagenesis via CRISPR tiling of gene-coding exons has been demonstrated to identify functional regions in genes. Furthermore, breakthroughs in combining CRISPR library screens with single-cell droplet RNA sequencing (sc-RNAseq) platforms have revealed the capacity to monitor gene expression changes upon genetic perturbations at single-cell resolution. Here, we present "sc-Tiling," which integrates a CRISPR gene-tiling screen with single-cell transcriptomic and protein structural analyses. Distinct from other reported single-cell CRISPR screens focused on observing gene function and gene-to-gene/enhancer-to-gene regulation, sc-Tiling enables the capacity to identify regulatory mechanisms within a gene-coding region that dictate gene activity and therapeutic response.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • CRISPR-Cas Systems*
  • Clustered Regularly Interspaced Short Palindromic Repeats / genetics*
  • Drug Screening Assays, Antitumor
  • Gene Editing
  • Gene Expression Regulation, Neoplastic
  • Genetic Testing
  • Genome, Human
  • Histone-Lysine N-Methyltransferase / chemistry
  • Histone-Lysine N-Methyltransferase / genetics
  • Histones
  • Humans
  • Models, Molecular
  • Mutagenesis
  • Neoplasms / genetics*
  • Phenotype*
  • Transcriptome

Substances

  • Histones
  • DOT1L protein, human
  • Histone-Lysine N-Methyltransferase