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Islets, located in the pancreas, are the body’s natural insulin-producing cells. Insulin promotes the absorption of sugar (glucose) by various organs and lowers glucose levels in the blood. In people with Type 1 diabetes, the body’s immune system malfunctions, producing an “autoimmune” response that destroys the islets. Transplanting healthy islets from a donor is an experimental method that diabetes researchers are using to attempt to restore normal insulin production in Type 1 patients. However, islet cell transplantation faces several major problems.

Because of a shortage of organ donors, the supply of pancreatic islets available for transplantation is very limited. For the transplant of one patient, three to four donor pancreases are required. In addition, the immune system of any patient who receives transplants rejects the foreign cells, unless the recipient’s immune response is suppressed. Unfortunately, current therapies that suppress the immune system target not only the transplanted organ or cells but also generally reduce immune responsiveness, thereby greatly increasing the recipient’s risk of infection and cancer. Also, the drugs currently available are particularly toxic to islets and can in themselves cause diabetes. Finally, the particular autoimmune response of Type 1 diabetes patients, which causes Type 1 diabetes, can also attack and destroy the transplanted islet cell tissue that is meant to cure the disease.

Gene therapists treat or prevent disease by replacing, altering or supplementing a patient’s genetic material. To do so, they must not only determine what genetic material a patient needs, they must also find or develop “vectors.” Vectors carry genes or genetic material to a target cell and transfer the therapeutic genes to that cell. Viruses are often used because of their ability to enter cells and to introduce DNA into them.

The goal of the JDRF Center is to develop new gene therapy techniques that will increase the number of viable islets for transplantation, protect these transplanted cells from immune system attack and prevent the diabetes-related complication, peripheral neuropathy, in which nerves are injured. In at least the first three years of the Pittsburgh Center, all of the projects’ research and testing will be on laboratory animals. Eventually, investigators expect that findings from these projects will form the basis of clinical trials with humans.

Six of the seven projects at the JDF Center will use ex vivo gene therapy; that is, cells will be taken from a living animal, treated with genetic material, then returned to the animal. In the project to prevent diabetic nerve damage, gene therapy will be used in vivo; that is, genetic material will be introduced directly into a living animal.

Last Update
February 17, 2014
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Last Update
February 17, 2014