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kwilliams
Apr 28th, 2008, 12:04 PM
ITN - Monday, April 28 07:32 amBreakthrough 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 before the operation, but one patient benefited significantly.

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," he said.

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

The team is confident that the technique is safe and say it will test the technique on other patients with LCA. It hopes to begin trials for other forms of retinal disease in the future.

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.

Steven Howarth, 18, experienced significant improvement after the operation.

"When I woke up afterwards, my eyes felt uncomfortable - like sandpaper - at first I could not see anything in the eye that was operated on, but it got much better after a week, then gradually even better until it was back to normal.

"Now, my sight when it's getting dark or it's badly lit is definitely better. It's a small change but it makes a big difference to me.

"Before the operation, I used to rush home from college when it started to get dark because I was worried about getting around. Now I can take my time and stay later at college if I need to, for band rehearsals and things like that."

kwilliams
Apr 28th, 2008, 12:04 PM
It's amazing what people can do these days. I wonder how they 'inserted' the genes into their eyes though.

It must be so disheartening for the other two patients though.

Yasmine
Apr 28th, 2008, 12:37 PM
well from what I know about genes and all that my guess is they would have used solution in which the gene would have been in lipids that when it all comes in contact with the cells allows the gene to be released within the cell. (and I think just putting drops on top of the eyes would work, no injection or anything would be required).

kwilliams
Apr 28th, 2008, 02:34 PM
well from what I know about genes and all that my guess is they would have used solution in which the gene would have been in lipids that when it all comes in contact with the cells allows the gene to be released within the cell. (and I think just putting drops on top of the eyes would work, no injection or anything would be required).


Wow, as simple as eyedrops. Very cool.

Yasmine
Apr 28th, 2008, 02:50 PM
pretty much;) unless they have to be very specifically targetted and in that case it much have been a pain to insert them directly in the cells they wanted (which I doubt was necessary)

Apoleb
Apr 28th, 2008, 03:09 PM
pretty much;) unless they have to be very specifically targetted and in that case it much have been a pain to insert them directly in the cells they wanted (which I doubt was necessary)

My guess is that they have to get them to get in contact with the retina. I'm not sure eye drops can get the job done.

Apoleb
Apr 28th, 2008, 03:13 PM
Check this webpage: http://news.bbc.co.uk/2/hi/health/7369740.stm

They have a step by step animation if you scroll down. It's basically a surgical operation, and the gene gets inserted into the eye with a needle. Oh, and basically a virus carrying the gene is injected, so that's how the gene is spread in the cells of the retina.

Yasmine
Apr 28th, 2008, 03:38 PM
Check this webpage: http://news.bbc.co.uk/2/hi/health/7369740.stm

They have a step by step animation if you scroll down. It's basically a surgical operation, and the gene gets inserted into the eye with a needle. Oh, and basically a virus carrying the gene is injected, so that's how the gene is spread in the cells of the retina.
Yes viruses or lipids can be used for gene therapy. Principle is virus gets into the cells without destroying them and inserts the DNA in the cell with the repair gene without causing any damage. For some genes drops might be enough but in that case obviously the cells have to be precisely targeted which is why an injection was necessary.

And that was just me assuming, I hadn't looked for it.