Good Virus Helps Researchers Slow Progression of Eye Disorders

Scientists are analyzing engineered adeno-associated virus (AAV), the benign virus, to compensate for the missing protein that causes vision problems. The findings are carried out by West Virginia University School of Medicine researchers.

“Eighty-five percent of Americans are seropositive for AAV. However, the virus has never been associated with any pathological effect,” said Wen Tao Deng — an assistant professor in the Department of Ophthalmology and Visual Sciences — who is leading the effort. “We engineered the virus to use it as a vehicle to deliver the genes we are interested in. We use it as a tool to actually benefit us. So, this is a good virus.”

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

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The National Eye Institute has awarded the five-year project $1.9 million.

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, basically you lose visual acuity; you lose your ability to read; you lose your color vision,” Deng said. “It severely, severely affects your daily function.”

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

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They will analyze — on a molecular level — the unique mechanisms that underlie the disease.

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

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

Research reported in this publication was supported by the National Eye Institute of the National Institutes of Health under Award Number 7R01EY030056-03. The content is solely the responsibility of the authors and does not necessarily represent the official views of NIH.

Source-Eurekalert