JDFI Gene Therapy Center for Diabetes and Diabetic Complications at UF and UM


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Project 5

Investigators: Kenneth Berns, William Hauswirth, Sergei Zolotukhin

Diabetes-associated vision loss is the most frequent cause of blindness among the working-age population. The formation of new blood vessels in the eye and their leakage into the light- sensing retina is the cause of this blindness. Unfortunately no current treatment is successful in very many patients for a significant length of time. The need therefore clearly exists to develop treatments that can prevent formation of these unwanted blood vessels.

PEDF (pigment epithelium-derived factor) is the molecule that controls the number of normal blood vessels in the eye by stopping the creation of new vessels soon after birth. PEDF therefore holds promise for treating diabetic adults to prevent this form of blindness.Our central aim is to develop such treatment using non-toxic viruses, engineered in the lab to carry the gene for PEDF into the eye. We plan to test this idea on a type of mouse that has been developed to make abnormally high numbers of blood vessels in the eye. These mice go blind in the same way humans with diabetes lose vision. Using these mice, we will examine various forms of PEDF that lead to different amounts of PEDF in the eye, testing each for its ability to prevent or slow the formation of unwanted vessels.

We will also determine the best part of the eye from which PEDF should be produced to be most effective in preventing blindness in this mouse model of human disease. This project is aimed at carefully examining the ability of one promising gene therapy to treat vision loss with the long-term aim of finding an effective prevention for the blindness that frequently accompanies diabetes

UF Center for Immunology and Transplatation

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August 16, 2004
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