‘Good Virus’ May Help Scientists Slow Progression of Incurable Eye Disorders | WVU today


Wen Tao Deng, a researcher at the WVU School of Medicine, is studying how a benign virus (a modified adeno-associated virus) can be used in gene therapy to treat vision problems associated with specific photoreceptors. His work is relevant to a range of eye disorders, including the color vision impairments shown here (clockwise from the upper left corner: normal vision, deuteranopia, blue cone monochromacy, protanopia ).
(Photo illustration WVU / Jennifer Shephard / Aira Burkhart)

Viruses get a bad rap for good reason. But some viruses don’t hurt people. Some can even help them.

Researchers with the University of West Virginia Medicine School are studying how a benign virus can make possible new treatments for eye disease. They are exploring how to use a modified adeno-associated virus, or AAV, to make up for the missing protein or swap out genetic mutations that cause vision problems and replace them with DNA that works as it should.

“Eighty-five percent of Americans are HIV positive for AAV. However, the virus has never been associated with any pathological effect, ”said Wen Tao Deng-an assistant professor at the Department of Ophthalmology and Visual Sciences– who is leading the effort. “We designed the virus to use it as a vehicle to pass on the genes that interest us. We use it as a tool for us to benefit from it. So, this is a good virus.

The National Eye Institute has allocated $ 1.9 million for the project over five years.

The project focuses on genetic mutations that affect specific photoreceptors in the eye, called L and M cones.

“When you lose your L and M cones, you basically lose your visual acuity; you lose your ability to read; you lose your color vision, ”Deng said. “It seriously, seriously affects your daily function. “

Deng and his colleagues will use mouse models that they genetically modified to lose their L and M cones in a way that mimics the experience of humans inheriting this mutation.

They will analyze, at the molecular level, the unique mechanisms underlying the disease.

They will take advantage of the AAV ‘Trojan horse’ ability to sneak into a photoreceptor nucleus and either replace its missing protein or remove a troublesome mutation while installing healthy DNA in its place.

Deng is also interested in developing new treatments that could delay the onset or slow the progression of a range of eye diseases, from vision defects from red-green colors (the most common form of color deficiency) to blue cone monochromatism (a much rarer condition) and other forms of cone dystrophy.

“We are also interested in delaying degeneration,” Deng said. “Some patients gradually lose their vision. So if you could delay the degeneration of their photoreceptor cells for 5-10 years, that could also give them an extended treatment window. It’s especially important for children to buy time until we identify a treatment to reverse it. “

The research reported in this publication was supported by the National Eye Institute of the National Institutes of Health under award number 7R01EY030056-03. The contents are the sole responsibility of the authors and do not necessarily represent the official opinions of the NIH.

-WVU-

see 15/12/21

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