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Updated: Dec 6, 2025

CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy
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In vivo CRISPR/Cas9 targeting of fusion oncogenes for selective elimination of cancer cells

M Martinez-Lage1, R Torres-Ruiz2,3, P Puig-Serra1

  • 1Molecular Cytogenetics and Genome Editing Unit, Human Cancer Genetics Program, Centro Nacional de Investigaciones Oncológicas (CNIO), 28029, Madrid, Spain.

Nature Communications
|October 9, 2020
PubMed

Insights

Scientists developed a new gene-editing method to target fusion oncogenes (FOs) in cancer. This approach uses CRISPR/Cas9 to disrupt FOs specifically in cancer cells, offering a promising strategy for cancer therapy.

Area of Science:

  • Oncology
  • Gene Editing
  • Molecular Biology

Background:

  • Fusion oncogenes (FOs) are key drivers in many cancers.
  • Targeting FOs is challenging due to their chimeric nature.
  • Cancer cell-specific expression and apoptosis induction make FOs attractive targets.

Purpose of the Study:

  • To develop a novel, efficient, and non-patient-specific gene-editing strategy for targeting fusion oncogenes.
  • To demonstrate the efficacy of intron-based targeting of FOs in preclinical cancer models.

Main Methods:

  • Utilized CRISPR/Cas9 gene-editing technology.
  • Designed a strategy targeting specific introns of genes involved in fusion oncogene rearrangement.
  • Validated the approach in in vivo models for transcription factor and tyrosine kinase FOs.

Main Results:

  • Achieved robust disruption of fusion oncogenes specifically in cancer cells.
  • Demonstrated reduction in tumor burden and mortality in preclinical models.
  • Proof-of-concept for intron-based targeting of FOs.

Conclusions:

  • The intron-based gene-editing strategy offers a new method for selective cancer cell elimination.
  • This approach holds potential for developing novel cancer therapeutics targeting fusion oncogenes.
  • Opens new avenues for treating FO-driven cancers.

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