Gene therapy could treat inherited blindness

September 6, 2007

DNA elements have been identified that control when and where genes linked to blindness are "switched on".

DNA elements have been identified that control when and where genes linked to blindness are "switched on", according to researchers at Washington University School of Medicine, St Louis, USA.

Using a computational analysis of DNA to search for elements that can turn on genes in the photoreceptor cells of the eye, the investigators have identified hundreds of potential cis-regulatory elements (DNA segments involved in activating and deactiviating genes). Nineteen of these elements have now been confirmed, more than doubling the number previously identified.

The results of this study will be used to design gene therapy vectors for a form of Leber's congenital amaurosis, an inherited disorder that leaves patients blind from birth.