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Sunday, April 11, 2021

UF researchers have begun a study that could cure a type of blindness by intentionally injecting viruses into patients ' eyes.

Patients with inherited blindness will be injected with viruses engineered to provide a missing protein to the light-collecting cells in their eyes.

UF is one of the leaders of the multi-university study.

The clinical trial, which began earlier this month, will study six adults and three children afflicted with inherited blindness, a UF press release stated.

"The idea of the therapy is simple," wrote William Hauswirth, a UF professor, in the press release. "If cells are missing a gene for a vital function, such as vision, the therapy is to replace that gene."

The process was pioneered by Nicholas Muzyczka, a UF professor of molecular genetics and microbiology. Muzyczka said viruses have the unique ability to penetrate the nucleus of living cells and, in normal circumstances, leave their own DNA to replicate more viruses.

During this procedure, molecular geneticists remove a virus's DNA and reprogram it to deliver a different DNA, Muzyczka said.

Once injected into the patient's retina, the virus invades a cell and leaves the modified DNA in the nucleus. The DNA makes the nucleus produce a missing protein, which could restore sight to the patient's eye.

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