Researchers at the University of Florida's Genetics Institute successfully used gene therapy in mice to treat Retinoschisis, a rare incurable genetic eye disease that affects boys.

Retinal cells secrete a protein called Retinoschisin (RS1), which acts like a glue to bind the layers of the retina. Boys with Retinoschisis lack the ability to secrete RS1. The absence of this protein causes the retinal layers to separate and leads to the formation of tiny cysts. The boys lose their sight gradually, often with devastating results.

The findings, published in the August issue of the journal Molecular Therapy, suggest this gene transfer method may eventually be used to treat retinoschisis and other eye diseases in humans caused by single gene defects.

Currently, there is no treatment for retinoschisis. The disease, which is first detected between the ages of 5 and 10, leads to blindness in about one of every 5,000 boys.