http://www.digitaljournal.com/article/253900

Gene therapy improves eyesight by replacing faulty gene

Posted Apr 28, 2008 by Paul Wallis
From progressively losing his eyesight to gaining better vision has taken Stephen Howarth a few months, and he doesn’t mind a bit. It’s certainly not wasted time. Gene therapy has taken another big step forward, against a truly grim medical condition.
DNA strand
DNA strand
The BBC describes the process:
His condition was due to a faulty gene that meant that the light-detecting cells at the back of his eye were damaged and slowly degenerating further.
But, in a delicate operation, surgeons at Moorfields injected working copies of the gene into the back of Stephen's eye.
After a few months, doctors detected some improvements.
But Stephen did not notice these changes until he confidently strode through a dimly-lit maze designed to test his vision.
Until then he had kept walking into walls - and it would take him nearly a minute to walk a few feet.
His doctors were shocked at the improvement.
Extremely significant about this is the fact that the replacement genes took over from the faulty ones.
It’s not a natural assumption that the new genes would do that.
The possibility was that they wouldn’t. Two other patients have been given this treatment, and their vision hasn’t improved, although there’s reason to hope it won’t deteriorate further.
Obviously, something has gone very right with Stephen’s treatment.
Professor Ali (Professor Robin Ali, of the Institute for Ophthalmology)
said that the team now hoped to treat children: "The next stage is to increase the dose of the gene which we anticipate will improve the outcome - and it's also to treat younger patients, who have better residual vision and in whom we expect to see a much greater benefit."
As one of the consulting surgeons says, another very important point is that this treatment has shown that gene therapy can work for a particular gene disorders.
The economics of that are impressive. This was a relatively simple, straightforward, procedure in terms of principles and methods. (See BBC article graphic.)
That it’s worked against a progressive degenerative disease is almost indescribably important. Conditions like that include some of the nastiest diseases on Earth.
The method could take a lot of strain off the medical profession. Particularly likely to benefit is the long suffering therapeutic management part of the health system, which has been burdened with palliative treatments and massive use of resources, time and money.
“Just add gene” could easily revolutionize the treatment of a lot of pernicious, vicious, diseases which have been literally getting away with murder for centuries.
It’s too soon to say that this is the guiding principle of future gene therapy, but it’s definitely a big, bright, beacon.