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By Mayo Clinic staffThe possibilities of gene therapy hold much promise. To date, however, that promise has not been realized. Some clinical trials have recorded small successes for a few participants. But, several significant barriers stand in the way of gene therapy becoming a reliable form of treatment. The barriers include developing reliable vectors, consistently ensuring safety, targeting the correct cells, and preventing genetic changes from being passed on from parents to children.
Although at this time no clinical trial has been seen as a breakthrough in overcoming all these barriers, gene therapy is a very active area of research. Researchers hope that with continued study, advances in gene therapy will eventually make it a practical approach to treating disease.
- Gene therapy. Centre for Genetics Education. http://www.genetics.edu.au/pdf/factsheets/fs27.pdf. Accessed Sept. 7, 2010.
- Gene therapy for cancer: Questions and answers. National Cancer Institute. http://www.cancer.gov/cancertopics/factsheet/Therapy/gene. Accessed Sept. 7, 2010.
- Gene therapy. Human Genome Project Information. http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml. Accessed Sept. 7, 2010.
- Genetics home reference. National Library of Medicine. http://ghr.nlm.nih.gov/handbook. Accessed Sept. 7, 2010.
- Wieben ED (expert opinion). Mayo Clinic, Rochester, Minn. Sept. 18, 2010
- About clinical trials. American Society of Gene & Cell Therapy. http://www.asgct.org/educational_resources/clinical_trials.php. Accessed Sept. 24, 2010.
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