Gene therapy may cure boys' eye disease

GAINESVILLE, Fla., Aug 03, 2005 (United Press International via COMTEX) -- University of Florida scientists say they used a healthy human gene to prevent blindness in mice with a form of an incurable eye disease that strikes boys.

Writing in the August issue of Molecular Therapy, scientists from the university's Genetics Institute describe how they successfully used gene therapy in mice to treat retinoschisis, a rare genetic disorder that is passed from mothers, who retain their sight, to their sons.

. Retinoschisis is usually detected in boys between 5 and 10 years of age when vision problems cause reading difficulties.

"Currently there is no treatment," said William Hauswirth, a professor of ophthalmic molecular genetics. "These children lose their sight gradually, often with devastating results.

"What happens is the retina actually begins to split in the middle, causing loss of central vision -- that's the vision that you need to be able to read or walk around."

Researchers say the gene transfer method eventually help many eye diseases caused by single gene defects, including retinitis pigmentosa, which affects about 200,000 people in the United States. It is one of the most common inherited causes of blindness in people between the ages of 20 and 60.