CRISPR/Cas9-loaded stealth liposomes effectively cleared established HPV16-driven tumours in syngeneic mice

PLoS One. 2021 Jan 7;16(1):e0223288. doi: 10.1371/journal.pone.0223288. eCollection 2021.

Abstract

Gene-editing has raised the possibility of being able to treat or cure cancers, but key challenges remain, including efficient delivery, in vivo efficacy, and its safety profile. Ideal targets for cancer therapy are oncogenes, that when edited, cause cell death. Here, we show, using the human papillomavirus (HPV) type 16 cancer cell line TC1, that CRISPR/Cas9 targeting the E7 oncogene and packaged in PEGylated liposomes cleared established tumours in immunocompetent mice. Treatment caused no significant toxicity in the spleen or liver. An ideal therapeutic outcome would be the induction of an immunogenic cell death (ICD), such that recurrent tumours would be eliminated by the host immune system. We show here for the first time that CRISPR/Cas9-mediated cell death via targeting E7 did not result in ICD. Overall, our data show that in vivo CRISPR/Cas targeting of oncogenes is an effective treatment approach for cancer.

MeSH terms

  • Animals
  • CRISPR-Cas Systems / genetics*
  • Cell Death / genetics
  • Cell Line, Tumor
  • Clustered Regularly Interspaced Short Palindromic Repeats / genetics*
  • Female
  • Gene Editing / methods
  • Genetic Therapy / methods
  • HeLa Cells
  • Human papillomavirus 16 / genetics*
  • Humans
  • Jurkat Cells
  • Liposomes / metabolism*
  • Mice
  • Mice, Inbred C57BL
  • Neoplasms / genetics*
  • Neoplasms / virology
  • Oncogene Proteins, Viral / genetics
  • Papillomavirus E7 Proteins / genetics
  • Papillomavirus Infections / genetics*
  • Papillomavirus Infections / virology
  • Uterine Cervical Neoplasms / genetics*
  • Uterine Cervical Neoplasms / virology

Substances

  • Liposomes
  • Oncogene Proteins, Viral
  • Papillomavirus E7 Proteins

Grants and funding

The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.