An improved approach to gene therapy may one day treat some of the nearly 200 inherited forms of blindness, scientists at Washington University School of Medicine in St. Louis suggest this week.
In a paper published online by Public Library of Science ONE, researchers take initial steps toward filling a gap in the toolkit for treating blindness by identifying DNA elements that control when and where genes linked to blindness are turned on.
"In its most basic form, gene therapy requires three components," explains Joseph Corbo, M.D., Ph.D., assistant professor of pathology and immunology and senior author of the paper. "You need to identify the gene that's mutated in patients and obtain a healthy copy of that gene; you need an agent to deliver the healthy gene to a patient's cells, which is usually a weakened virus; and then you also need additional DNA elements that turn on the healthy gene in the right cells and at the right levels."
Corbo and colleagues used a computational analysis of DNA to look for elements that can turn on genes in the eye's light-sensing photoreceptor cells. Their search identified hundreds of potential cis-regulatory elements, which are segments of DNA involved in turning genes on and off. They confirmed 19 of these elements, more than doubling the number known to scientists. The newly discovered elements can be used as switches to activate blindness therapies.
Washington University in St. Louis News & Information (07/25/07)
Science Daily (07/27/07)
Medical News Today (07/27/07)