Gene Therapy Could Restore Failing Sight

Article excerpt


BRITISH scientists have used gene therapy to restore a patient's sight in a pioneering operation that will bring hope to tens of thousands of partially blind people.

Robert Johnson, 23, hopes to see fully again following the surgery at Moorfields Eye Hospital in London.

Mr Johnson, who was born with a degenerative disorder that means he can only see outlines during the day and nothing at night, had copies of genes inserted into one of his eyes.

The hope is this will correct a genetic fault that stops his retina detecting light properly. It will be several months before it is known if the operation was a success. If it is, it offers hope to tens of thousands of people with genetic eye problems.

Scientists warn it is more likely the procedure will only work on children and young adults because older people's eyesight may have deteriorated too much.

But they say it could lead on to advances in the treatment of age-related macular degeneration, which affects 400,000 people in the UK. Lead researcher Professor Robin Ali, of University College London's institute of ophthalmology, said the clinical trial was 'exciting'.

He said: 'We have been developing gene therapy for eye disease for almost 15 years but until now we have been evaluating the technology only in the laboratory.

'Testing it for the first time in patients is very important and exciting, and represents a huge step towards establishing gene therapy for the treatment of many different eye conditions.' He added: 'There are many forms of retinal degeneration, meaning the use of gene therapy treatments must be individually developed and then tested in a separate clinical trial specifically for that disease.

'However, the results from this first human trial are likely to provide an important basis for many more gene therapy protocols in the future, as well as potentially leading to an effective treatment for a rare but debilitating disease.' Mr Johnson's disorder is caused by a faulty gene called RPE65, which stops the layer of cells at the back of the eyes, behind the retina, from working.

It results in severely impaired vision from a very young age and there are currently no effective treatments available.

Before the operation, Mr Johnson could see outlines during the day, but very little at night. …