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Gene Therapy for Safer Treatment of Type 1 Diabetes

标题

Delaying the Progression of Diabetes

详细技术说明

None

*Abstract

Reduces Blood Glucose Levels in Patients to Prevent the Onset of Type 1 Diabetes

This gene therapy boosts pancreatic function to better manage Type 1 diabetes. Formerly called "juvenile diabetes," diabetes mellitus Type 1 occurs when the body begins attacking itself, destroying the pancreas' ability to produce insulin. A healthy person's pancreas makes insulin and glucagon, which lower and raise blood glucose levels, respectively. Glucose, a simple sugar that fuels every cell in the body, comes from food that is broken down by the digestive system. Insulin and glucagon counterbalance each other to ensure that tissues are supplied with a steady source of energy. Type 1 diabetics lose their ability to produce insulin, which creates dangerous blood-glucose imbalances. University of Florida researchers have discovered a protein present within the islets of the pancreas that can regulate these two important pancreatic molecules. Expression of this newly named protein, now referred to as Islet Homestasis Protein (IHoP), can be manipulated to alter the balance of insulin and glucagon in a patient's body. In the case of a pre-diabetic patient, IHoP expression can be decreased using small interfering RNA (siRNA) technology, which causes the decreases in glucagon expression, lowering the patient's overall blood-glucose levels. In the United States, approximately 3 million people have Type 1 diabetes, and another 30,000 are diagnosed every year. This technology has the potential to keep those with insulin resistance from developing diabetes. It also reduces the need for painful and invasive pancreatic transplants.

Application

Gene therapy for the reduction of blood glucose levels in pre-diabetic patients

Advantages

• Aids pancreas islet homeostasis, possibly preventing the progression of diabetes in pre-diabetic individuals
• Less expensive and less risky than pancreatic transplant, providing a competitive advantage in the marketplace
• Naturally reduces the production of glucagon, safely lowering blood glucose levels while raising insulin levels

Technology

University of Florida researchers have discovered a new functional protein called IHoP that is expressed in the glucagon-expressing cells of the pancreatic islets. Glucagon is a peptide hormone that alpha cells in the pancreas secrete in response to low blood glucose (sugar) levels. When the liver detects glucagon, it releases stored glucose into the bloodstream to restore normal blood sugar levels. The body also releases insulin - particularly after large meals - to prevent blood sugar levels from climbing too high. Hypoglycemia (low blood sugar) can lead to headaches, dizziness, blurred vision and confusion, while prolonged hyperglycemia (high blood sugar) damages blood vessel walls, causing heart disease, strokes and kidney failure. The newly discovered protein regulates two important pancreatic molecules that affect the delicate balance between insulin and glucagon. In addition to regulating the secretion of important hormones, IHoP also may play a role in programmed cell death (apoptosis) within the islets.

*IP Issue Date

Aug 18, 2015

*IP Publication Date

Oct 9, 2014

*Principal Investigation

Name: Bryon Petersen

Department:

Name: Houda Darwiche

Department:

Name: Seh-hoon Oh

Department:

Name: Thomas Shupe

Department:

申请日期

May 8, 2012

申请号码

9,109,042

其他

国家/地区

美国