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Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...

Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...

Targeted Cancer Therapies

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...

In-vitro Mutagenesis

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.

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Related Experiment Video

Updated: Jun 2, 2026

Genome-Wide CRISPR Screen for Unveiling Radiosensitive and Radioresistant Genes

Genome-Wide CRISPR Screen for Unveiling Radiosensitive and Radioresistant Genes

Published on: May 23, 2025

Radioprotective gene therapy.

Patrick Maier¹, Marlon R Veldwijk, Frederik Wenz

¹Heidelberg University, University Medical Centre Mannheim, Department of Radiation Oncology, Theodor-Kutzer-Ufer 1-3, 68167 Mannheim, Germany. patrick.maier@medma.uni-heidelberg.de

Expert Opinion on Biological Therapy

|May 3, 2011

Summary

Radioprotective gene therapy offers a promising approach to mitigate radiation-induced damage in normal tissues, potentially improving cancer treatment outcomes and patient quality of life by reducing side effects like myelosuppression and mucositis.

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Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Published on: February 15, 2019

Area of Science:

Radiation oncology
Gene therapy
Molecular biology

Background:

Radiotherapy can cause myelosuppression and mucositis, limiting treatment effectiveness.
Gene transfer into normal tissues could reduce radiation-induced toxicity.
This approach aims to protect normal cells from radiation damage.

Purpose of the Study:

To review the potential of radioprotective gene therapy.
To discuss gene transfer methods and radioprotective genes.
To evaluate the feasibility of this therapeutic strategy.

Main Methods:

Evaluation of genes like MDR1, SNAI2, and superoxide dismutases in preclinical models.
Use of viral vectors such as adenoviral, AAV, and retroviral vectors for gene transfer.
Discussion of cytoprotective agents and small-molecule protectors.

Main Results:

Several genes demonstrated radioprotective potential in preclinical studies.
Various viral vectors have been employed for gene delivery.
The feasibility of radioprotective gene therapy is supported by current research.

Conclusions:

Radioprotective gene therapy shows significant promise for reducing normal tissue toxicity.
Further optimization of vectors for targeted transduction and transgene expression is needed.
This therapy could enhance radiotherapy success and improve patient quality of life.