The blind can see after gene therapy

Multiple newspapers have reported a small trial of a revolutionary new gene therapy that has cured a form of inherited blindness within days.

Three young adult volunteers who had LCA, a particular form of blindness, and had severe vision loss since childhood were recruited. The researchers used a technique whereby the DNA of the virus AAV was removed and then replaced with DNA containing the desired gene (RPE65 in this case). The virus carrying the RPE65 gene was then injected into the retina of one eye in each of the volunteers.

The researchers found that all three volunteers had an improvement in their vision 30 days after the injection, with two volunteers reporting improvements as early as 7 - 10 days after the treatment.

Critics say it is important to note that this particular gene therapy would only work for this type of LCA, more "studies are needed to fully understand the process and its longer-term effects and safety before it can be used more widely".

Source: "Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics". PNAS USA 2008; Sep 22 [Epub ahead of print]