What risks are associated with current gene therapy trials?

Dr. Jeanne Morrison, PhD
Family Practitioner

Viruses can usually infect more than one type of cell. Thus, when viral vectors are used to carry genes into the body, they might infect healthy cells as well as cancer cells. 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.

In addition, when viruses or liposomes 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 inflammation or an immune reaction; and that the virus could be transmitted from the patient to other individuals or into the environment. Scientists use animal testing and other precautions to identify and avoid these risks before any clinical trials are conducted in humans.

This answer is based on source information from the National Cancer Institute.

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