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Gene therapy prevents retinoschisis in mice
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GAINESVILLE, Fla. — Gene therapy using a healthy human gene was successful in preventing blindness in mice with retinoschisis, according to researchers in Florida.
William Hauswirth, PhD, and colleagues at the University of Florida injected a healthy version of the human Rs1 gene into the subretinal space of the right eyes of 15-day-old mice with retinoschisis. The mice were roughly equivalent in age to a 10-year old boy, according to a press release from the university.
After 6 months, the untreated left eyes had cyst formation, but the treated right eyes were healthy, the researchers reported. The rods and cones in the treated eyes showed no signs of the disease, and the connections between the layers of the retina were intact.
“We now have proof of principle that gene therapy can basically prevent retinoschisis,” said Stephen Rose, PhD, chief research officer for the Foundation Fighting Blindness, an underwriter of the research.