Another danger is that the new gene might be inserted in the wrong location in the DNA, possibly causing harmful mutations to the DNA or even cancer. This has occurred in clinical trials for X-linked severe combined immunodeficiency (X-SCID) patients, in which hematopoietic stem cells were transduced with a corrective transgene using a retrovirus, and this led to the development of T cell leukemia in 4 of 20 patients. See reports for first patient, second patient and third patient.
In addition, when viruses are used to deliver DNA to cells inside the patient’s body, there is a slight chance that this DNA could unintentionally be introduced into the patient’s reproductive cells. If this happens, it could produce changes that may be passed on if a patient has children after treatment.
Other concerns include the possibility that transferred genes could be overexpressed, producing so much of the missing protein as to be harmful; that the viral vector could cause an immune reaction; and that the virus could be transmitted from the patient to other individuals or into the environment.
However, this basic mode of gene introduction currently shows much promise and doctors and scientists are working hard to fix any potential problems that could exist. They use animal testing and other precautions to identify and avoid these risks before any clinical trials are conducted in humans.
- Alliance for Cancer Gene Therapy
- American Cancer Society
- Bioscience Explained
- Gene Therapy and Children, KidsHealth
- Genetic Science Learning Center
- Human Genome Project Information
- ICTVdB, The Universal Virus Database
- Molecular Medicine in Action
- National Cancer Institute
- Understanding the Human Genome Project - Online education kit
- Viral Vectors and Gene Therapy, All the Virology on the www
- Images of gene therapy
- PowerPoint presentations on gene therapy
- YouTube videos about gene therapy