University of Florida scientists 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 UF 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. “Currently there is no treatment,” said William Hauswirth, Ph.D., the Rybaczki-Bullard 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.” Scientists say the gene transfer method eventually could be applied to many eye diseases caused by single gene defects, including a host of retinal disorders.
