Gene delivery of a mutant form of erythropoietin preserves neuronal cell bodies, axons, and function.
Description:
The Problem
Glaucoma is a leading cause of blindness worldwide and affects approximately 4 million people in the United States. Currently, there is no cure for glaucoma and any vision loss that occurs is permanent. Current treatments include daily drops and pills aimed at lowering the intraocular pressure (IOP). New treatments are needed to 1) address the issue of noncompliance with current treatments, 2) preserve cells and ocular functions, and 3) benefit patients that do not respond to current treatments aimed at lowering IOP.
The Technology Solution
Researchers at the University of Tennessee have developed a novel mutant of erythropoietin, which can be delivered systemically without causing an unsafe increase in hematocrit. Further, if packaged with a recombinant adeno-associated virus, a single intramuscular injection provides long-term therapy. Preliminary results in glaucomatous mice show that this mutant provides protection of retinal ganglion cells (RGCs) from cell death, protection of retinal ganglion cell axons from degeneration, and preserves visual function, all independent of IOP. Although neuroprotection is a known characteristic of EPO, this is the first time that any form of EPO or any other neuroprotective agent has been shown to protect both the cell soma and axon and retain visual function without increasing the hematocrit, all without performing an intraocular injection. These results indicate that this mutant could possibly be a new treatment that would alleviate the limitations of current treatments. The researchers continue their studies to test this mutant form of EPO in other models of neurodegenerative disease.


Related Publications:
•Neurochem Res. 2010 Oct 6. Epub.
•Exp Eye Res. 2009 Nov;89(5):735-40
Benefits
· Potential one-time treatment for glaucoma
· EPO safety profile already established
· No unsafe increase in hematocrit
Patents
U.S. Provisional Patent Application 61/433,186- Filed January 2011
PCT Patent Application PCT/US2012/21247- Filed January 2012
The Inventor
Dr. Tonia Rex is an Assistant Professor in Ophthalmology. Her research interests include neuroprotective gene therapy in mouse models of retinal degenerative diseases (including retinitis pigmentosa and glaucoma) and development, characterization, and treatment of a novel model of ocular trauma.
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