Gene therapy

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By Mayo Clinic staff

The possibilities of gene therapy hold much promise. To date, however, that promise has not yet 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.

MY00105

Sept. 19, 2008

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