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Updated: Nov 22, 2025
In Vivo CRISPR/Cas9 Screening to Simultaneously Evaluate Gene Function in Mouse Skin and Oral Cavity
Published on: November 2, 2020
CRISPR/Cas9-loaded stealth liposomes effectively cleared established HPV16-driven tumours in syngeneic mice
Luqman Jubair1,2, Alfred K Lam3, Sora Fallaha1
1School of Medical Sciences, Griffith University, Gold Coast, Queensland, Australia.
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.
Insights
CRISPR/Cas9 gene editing effectively cleared tumors in mice by targeting the E7 oncogene. This cancer therapy showed no significant toxicity, though it did not induce immunogenic cell death.
Area of Science:
- Oncology
- Gene Therapy
- Immunology
Background:
- Gene editing offers potential cancer treatments, but challenges in delivery, efficacy, and safety persist.
- Oncogenes are ideal targets for cancer therapy, as their editing can induce cell death.
Purpose of the Study:
- To evaluate the efficacy and safety of CRISPR/Cas9 targeting the E7 oncogene in established tumors.
- To determine if CRISPR/Cas9-mediated E7 targeting induces immunogenic cell death (ICD).
Main Methods:
- CRISPR/Cas9 targeting the E7 oncogene was delivered via PEGylated liposomes.
- Treatment efficacy and toxicity were assessed in TC1 tumor-bearing immunocompetent mice.
- Immunogenic cell death was evaluated post-treatment.
Main Results:
- Established tumors were cleared in mice treated with CRISPR/Cas9 targeting E7.
- No significant spleen or liver toxicity was observed.
- CRISPR/Cas9-mediated E7 targeting did not induce immunogenic cell death.
Conclusions:
- In vivo CRISPR/Cas9 targeting of oncogenes represents a viable and safe therapeutic strategy for cancer.
- While effective in tumor clearance, this approach does not currently induce an immune response via ICD.
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