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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
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