Gene Therapy May Help Against Rare Blinding Disease

According to researchers, a new gene therapy that successfully treated a rare eye disease in clinical trials could prove the key to preventing more common inherited causes of blindness.

According to the clinical trial report published in The Lancet, Doctors used a virus to repair a defective gene that causes choroideremia, a degenerative eye disease that can lead to complete blindness by middle age.

Lead author Robert MacLaren of the Nuffield Laboratory of Ophthalmology at the University of Oxford, and a consultant surgeon at the Oxford Eye Hospital, in England said that “Vision improved for all the patients following the gene therapy, and particularly for two patients with advanced choroideremia”

In a university news release, MacLaren further stated that “In truth, we did not expect to see such dramatic improvements in visual acuity and so we contacted both patients' home opticians to get current and historical data on their vision in former years, long before the gene therapy trial started.”

While the initial research had focused on choroideremia which is a relatively simple disease caused by just one defective gene, now that it has been proven that this type of treatment can work, MacLaren and his team of doctors can investigate it to treat more complicated genetic eye disorders in which multiple genes are malfunctioning.

For details you should read the original article by Dennis Thompson, HealthDay Reporter which was published on webmd.com.

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