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Updated: Sep 28, 2026
Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
Published on: August 25, 2021
Introduction to cancer gene therapy
James C Cusack1, Kenneth K Tanabe
1Division of Surgical Oncology, Massachusetts General Hospital, Harvard Medical School, Cox Building, Room 626, 100 Blossom Street, Boston, MA 02114, USA. jcusack@partners.org
Abstract:
Over the past decade, the unprecedented growth in science and technology has fueled the development of novel treatment strategies to combat disease. The creative and innovative efforts of scientists and clinicians to overcome the multitude of unforeseen obstacles to success is no better exemplified than in the field of cancer gene therapy. Since its inception, developers of cancer gene therapy have been charged with the challenge of altering basic tumor biology or, alternatively, the host responses for the purpose of tumor eradication and prevention. Several major therapeutic strategies have emerged from preclinical studies, and results from these early studies hold promise for altering the clinical outcome in a variety of malignancies. These strategies may be broadly subcategorized and range in intent from alteration of the tumor cell phenotype by replacement of defective cellular response genes (e.g., mutated or deleted tumor suppressor genes) to the enhancement of the immunological response to cancer (e.g., amplification of the cell surface antigen signature or modulation of the host response). Not surprisingly, the increasingly intricate nature of tumor biology revealed over the past several years has effectively raised the bar of success for those involved in the development of effective molecular and cancer gene therapy strategies. This, in turn, has led to the development of more complex therapies that frequently draw upon multiple disciplines in an effort to optimize treatment response.
Insights
Cancer gene therapy advances novel treatments by altering tumor biology or host responses. These innovative strategies show promise for improving outcomes in various cancers.
Area of Science:
- Oncology
- Molecular Biology
- Immunology
Background:
- Rapid advancements in science and technology drive the development of new disease treatments.
- Cancer gene therapy aims to modify tumor biology or host immune responses for cancer eradication.
- Overcoming obstacles in cancer gene therapy requires innovative approaches from scientists and clinicians.
Purpose of the Study:
- To review emerging therapeutic strategies in cancer gene therapy.
- To highlight the evolution of cancer gene therapy from preclinical studies to clinical promise.
- To discuss the complexity and multidisciplinary nature of developing effective cancer gene therapies.
Main Methods:
- Review of preclinical studies and emerging therapeutic strategies in cancer gene therapy.
- Categorization of strategies based on their intent (e.g., altering tumor cell phenotype, enhancing immune response).
- Analysis of the impact of increasing knowledge of tumor biology on therapy development.
Main Results:
- Several major therapeutic strategies have emerged from preclinical research.
- Promising results suggest potential to alter clinical outcomes in various malignancies.
- Therapeutic strategies include replacing defective tumor suppressor genes and enhancing anti-cancer immune responses.
Conclusions:
- Cancer gene therapy holds significant promise for treating a range of cancers.
- The complexity of tumor biology necessitates sophisticated and multidisciplinary therapeutic approaches.
- Continued innovation is crucial for optimizing treatment response in molecular and cancer gene therapy.
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