Vector engineering, strategies and targets in cancer gene therapy
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Updated: Nov 9, 2025

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
Published on: August 25, 2021
Vijayata Singh1, Nusrat Khan1, Giridhara R Jayandharan2,3
1Department of Biological Sciences and Bioengineering, Indian Institute of Technology, Kanpur, UP, India.
Abstract:
Understanding the molecular basis of disease and the design of rationally designed molecular therapies has been the holy grail in the management of human cancers. Gene-based therapies are an important avenue for achieving a possible cure. Focused research in the last three decades has provided significant clues to optimize the potential of cancer gene therapy. The development of gene therapies with a high potential to kill the target cells at the lowest effective dose possible, the development of vectors with significant ability to target cancer-associated antigen, the application of adjunct therapies to target dysregulated microRNA, and embracing a hybrid strategy with a combination of gene therapy and low-dose chemotherapy in a disease-specific manner will be pivotal. This article outlines the advances and challenges in the field with emphasis on the biology and scope of vectors used for gene transfer, newer targets identified, and their outcome in preclinical and clinical studies.
Insights
Cancer gene therapy offers a potential cure by targeting cancer cells effectively. Advances focus on optimized gene delivery vectors, novel targets, and combination strategies for improved outcomes.
Area of Science:
- Oncology
- Molecular Biology
- Gene Therapy
Background:
- Cancer management seeks molecular understanding and targeted therapies.
- Gene therapy presents a promising avenue for cancer cure.
- Decades of research have advanced cancer gene therapy potential.
Purpose of the Study:
- To outline advances and challenges in cancer gene therapy.
- To emphasize vector biology and scope in gene transfer.
- To review newer targets and their outcomes in studies.
Main Methods:
- Review of preclinical and clinical studies on cancer gene therapy.
- Analysis of vector development for targeting cancer antigens.
- Exploration of adjunct therapies and hybrid strategies.
Main Results:
- Development of highly potent gene therapies for target cell killing.
- Improved vector targeting of cancer-associated antigens.
- Emerging strategies combining gene therapy with chemotherapy.
Conclusions:
- Optimized gene therapies, targeted vectors, and combination approaches are pivotal.
- Further research in vector biology and novel targets is crucial.
- Disease-specific hybrid strategies hold significant therapeutic promise.
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