Gene Therapy May Alleviate Blindness, Study Shows

Results of a clinical trial supported by the Foundation Fighting Blindness published in the New England Journal of Medicine reveal gene therapy may reverse loss of sight.

Imagine a future where you don’t have to be concerned with the threat of blindness. Though ten years ago the idea would have been completely unrealistic, today, through research, scientists have found a way to restore sight to a group of individuals who were once nearly blind. Their findings provide hope that researchers can, one day, improve the eyesight of those who suffer from other degenerative diseases, including age-related macular degeneration (AMD).

According to two new studies, researchers restored partial vision in a small group of young adults with a form of retinal degeneration by using gene therapy. The subjects had a form of retinal degeneration known as Leber congenital amaurosis (LCA). LCA causes blindness by damaging the retina so that it no longer processes light.

Scientists utilized a normal version of a gene known as RPE65, which is mutated in one form of LCA. Researchers injected one eye with the gene and left the other eye untreated.

Funded in part by the Foundation Fighting Blindness, two studies were conducted; one took place in the United States and the other in Britain. In the American study, the patients, one aged 19 and two aged 26, were treated at The Children’s Hospital of Philadelphia. Within two weeks, they noticed their vision improve in the treated eye. Over a six-month period, researchers found continued improvement.

“Our clinical trial results represent an important first step in developing therapies and treatments that will reverse blindness in people with a variety of retinal degenerative diseases,” said Jean Bennett, M.D., Ph.D., who is the study’s lead researcher at The Children’s Hospital of Philadelphia.

“I believe that this is one of the most significant advances in human history in the field of medicine, to be able to treat a complicated retinal disease that we thought only ten years ago was untreatable,” said Dr. Robert Koenekoop of Montreal Children’s Hospital.

Hope for AMD Sufferers
With these findings, researchers hope that gene therapy could one day treat the more than 10 million people in the United States affected by retinal degenerative diseases, including macular degeneration, retinitis pigmentosa, and Usher syndrome.

AMD alone affects as many as 10 million people and is the leading cause of severe vision loss in Americans ages 50 and older. AMD is a permanent condition involving damage to the central part of the retina, the lining at the back of the eye that is crucial to vision.

This results in severe vision loss that makes activities such as reading, driving, and watching television extremely difficult. Though there is no cure for AMD, modifying risks such as protecting your eyes from the sunlight, improving your diet and avoiding smoking can delay the onset of the disease.

To learn more about preventative treatments of AMD visit http://www.visivite.com/visivite.html.