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Gene Therapy & Diabetes

- Summary
- About gene therapy
- Role in treating diabetes
- Benefits and risks
- Alternatives and variations
- Ongoing research
- Questions for your doctor

Reviewed By:
Robert Cooper, M.D., FACE

Ongoing research on gene therapy

The research surrounding gene therapy is still in early stages of development, and scientists are exploring many potential uses of gene therapy to treat diabetes and other conditions. It is important to emphasize that years of additional research will be required before these techniques might be used as medical treatments. The areas of study include:

  • Stem cells. Stem cells are immature cells that have the ability to develop into a variety of mature cells. Stem cells may be used to create insulin-producing beta cells or islet cells.

  • Vaccines. A priority of genetic diabetic research is stopping the autoimmune process that kills beta cells. Scientists are researching the possibility of using DNA-based vaccines to increase the immune system’s tolerance.

  • Identification of all the genetic factors involved in type 1 diabetes, type 2 diabetes and other forms of the disease. Scientists have linked a number of genes to the various forms of diabetes. They hope one day to identify all the specific genes that may be responsible.

  • Improvement of vector alternatives. Although many alternatives to viral vectors have been found, there are still several drawbacks to some variations. Researchers are attempting to resolve some of the issues related to the toxicity of viral vectors in addition to creating new and effective non-viral vectors.

  • Delivery of insulin that can respond to glucose (blood sugar). Researchers are developing cellular depots to store insulin and secrete it in response to an increase in glucose.

Researchers have reported preliminary success in using gene therapy to treat diabetic rodents. Although these findings have been widely reported in the media, such advances might or might not translate to success in treating humans with diabetes. Scientists have cautioned, for example, that mice do not process glucose like humans do. Extensive testing is required before any studies completed on animals can be undertaken on human subjects.

Gene therapy is a growing field of research that will likely have many advances and setbacks before a cure for diabetes may be found. Although it offers promise, people with diabetes should understand that any research breakthrough reported in the media may take years to develop as a medical treatment and might never become feasible.

In the meantime, it is important that patients control their diabetes by exercising, eating healthily, performing glucose monitoring, taking medication and following the other steps in a physician’s treatment plan.

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Review Date: 06-27-2007
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