Gene therapy hope for blind

Breakthrough gene therapy could improve the eyesight of hundreds of thousands of people who suffer from inherited blindness.

A medical trial, which began last February, involved inserting genes into patient's eyes to correct a genetic fault that stops their retinas detecting light properly.

After treatment, the three patients involved experienced vision at least equivalent to that before the operation, but one patient benefited significantly.

Known as Leber's congenital amaurosis (LCA), the inherited disorder causes progressive deterioration in vision and can lead to blindness in teenagers.

It occurs when faulty genes, called RPE65, stop the layer of cells at the back of the eye working.

The research was carried out by the University College London (UCL) Institute of Ophthalmology and Moorfields Eye Hospital, who received £1 million from the Department of Health

The team conducting the trial was led by Professor Robin Ali and includes eye surgeon James Bainbridge and retinal specialist Professor Tony Moore.

Prof Ali said: "Showing for the first time that gene therapy can work in patients with eye disease is a very significant milestone.

"This trial establishes proof of principle of gene therapy for inherited retinal disease and paves the way for the development of gene therapy approaches for a broad range of eye disorders."

Prof Moore said: "It is very encouraging to see that this treatment can work, even in young adults who have severely advanced disease."

Share this article

Send this article to a friend »

• Del.icio.us
• Digg
• Reddit
• Facebook
• StumbleUpon

What are these?