Gene therapy treatment works for inherited blindness

May 5, 2008

One of the first clinical trial's of a revolutionary gene therapy for Leber's congenital amaurosis (LCA), a type of inherited blindness, showed that the treatment can improve vision and cause no side effects.

London-One of the first clinical trial's of a revolutionary gene therapy for Leber's congenital amaurosis (LCA), a type of inherited blindness, showed that the treatment can improve vision and cause no side effects.

The trial, which was carried out on three patients suffering from LCA caused by an abnormality in gene RPE5, involved inserting healthy copies of the missing RPE65 genes into the cells of the retina to help them function normally. The healthy genes were delivered to the retina using a vector.

The trial began in February 2007 and resulted in one of the patients benefiting from significantly improved night vision. He was able to negotiate a simulation of a night-time scene quickly and without mistakes, which he could not have done before the surgery.

"Showing for the first time that gene therapy can work in patients with eye disease is a very significant milestone," said Robin Ali, PhD, University College London, Institute of Ophthalmology who led the trial. "This trial paves the way for the development of gene therapy approaches for a broad range of eye disorders."