Gene therapy for carcinoma of the breast: Genetic ablation strategies

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Looking for Driver Pathways of Acquired Resistance to Targeted Therapy: Drug Resistant Subclone Generation and Sensitivity Restoring by Gene Knock-down
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Looking for Driver Pathways of Acquired Resistance to Targeted Therapy: Drug Resistant Subclone Generation and Sensitivity Restoring by Gene Knock-down

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D T Curiel1

  • 1Gene Therapy Clinic, The University of Alabama at Birmingham, 1824 6th Ave. South, Birmingham, AL 35294-3300, USA. david.curiel@ccc.uab.edu

Breast Cancer Research : BCR
|March 16, 2001
PubMed

Insights

Gene therapy corrects cancer-causing mutations by disabling oncogenes. This approach, targeting gene expression or proteins, has been applied to breast cancer, paving the way for human trials.

Area of Science:

  • Oncology
  • Molecular Biology
  • Gene Therapy

Background:

  • Neoplastic transformation arises from molecular lesions.
  • Dominant oncogenes drive cancer through overexpressed oncoproteins.
  • Gene therapy offers a strategy to compensate for these mutations.

Purpose of the Study:

  • To explore gene therapy for correcting cancer-causing mutations.
  • To investigate methods for functionally knocking out dysregulated pathways driven by oncogenes.
  • To provide a rationale for human trials based on preclinical findings.

Main Methods:

  • Targeting gene expression via antisense or ribozymes.
  • Utilizing intracellular single chain antibodies (intrabodies) for post-transcriptional targeting.
  • Applying these methods to breast cancer-associated oncogenes (erbB-2, cyclin D1) and the estrogen receptor.

Main Results:

  • Demonstrated neoplastic revision in model systems.
  • Validated gene therapy approaches for specific breast cancer targets.
  • Established a basis for translating these findings into clinical applications.

Conclusions:

  • Mutation compensation via gene therapy is a viable strategy for cancer treatment.
  • Targeting oncogenes at transcriptional or post-transcriptional levels shows promise.
  • Preclinical success supports the advancement of these gene therapy approaches to human trials for breast cancer.

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