Related Experiment Video
Updated: Apr 24, 2026

A Nonviral Approach to Generate Transient Chimeric Antigen Receptor T Cells Using mRNA for Cancer Immunotherapy
Published on: February 21, 2025
Gene Therapies for Cancer: Strategies, Challenges and Successes
Swadesh K Das1,2,3, Mitchell E Menezes1, Shilpa Bhatia1
1Department of Human and Molecular Genetics, Virginia Commonwealth University, Richmond, Virginia.
Abstract:
Gene therapy, which involves replacement of a defective gene with a functional, healthy copy of that gene, is a potentially beneficial cancer treatment approach particularly over chemotherapy, which often lacks selectivity and can cause non-specific toxicity. Despite significant progress pre-clinically with respect to both enhanced targeting and expression in a tumor-selective manner several hurdles still prevent success in the clinic, including non-specific expression, low-efficiency delivery and biosafety. Various innovative genetic approaches are under development to reconstruct vectors/transgenes to make them safer and more effective. Utilizing cutting-edge delivery technologies, gene expression can now be targeted in a tissue- and organ-specific manner. With these advances, gene therapy is poised to become amenable for routine cancer therapy with potential to elevate this methodology as a first line therapy for neoplastic diseases. This review discusses recent advances in gene therapy and their impact on a pre-clinical and clinical level.
Insights
Gene therapy offers a promising cancer treatment by replacing faulty genes. Advances in delivery and vector design are overcoming challenges, paving the way for its use in treating neoplastic diseases.
Area of Science:
- Oncology
- Molecular Biology
- Biotechnology
Background:
- Gene therapy presents a targeted alternative to chemotherapy for cancer treatment.
- Chemotherapy's lack of selectivity and non-specific toxicity remain significant clinical challenges.
- Gene therapy has shown pre-clinical promise but faces hurdles like non-specific expression, delivery efficiency, and biosafety.
Purpose of the Study:
- To review recent advancements in gene therapy for cancer treatment.
- To discuss the impact of these advances on pre-clinical and clinical applications.
- To highlight the potential of gene therapy as a first-line cancer treatment.
Main Methods:
- Review of current literature on gene therapy strategies for cancer.
- Analysis of innovative genetic approaches and delivery technologies.
- Evaluation of pre-clinical and clinical progress in gene therapy.
Main Results:
- Development of safer and more effective vectors and transgenes.
- Improved tissue- and organ-specific targeting of gene expression.
- Overcoming key hurdles in delivery efficiency and biosafety.
Conclusions:
- Gene therapy is advancing towards routine clinical application for cancer.
- Targeted delivery technologies enhance the safety and efficacy of gene therapy.
- Gene therapy holds potential to become a primary treatment for neoplastic diseases.
More Related Videos
07:33Enhancing Chimeric Antigen Receptor-Extracellular Vesicles (CAR-EV) Technology: The Future of Cancer Therapy
Published on: September 19, 2025
09:37Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
Published on: August 25, 2021
Related Concept Videos
Targeted Cancer Therapies
There are several types of targeted therapies against...
Targeted Cancer Therapies
Combination Therapies and Personalized Medicine
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
Combination Therapies and Personalized Medicine
Cancer Therapies
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...
Cancer Therapies