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Ouch Q&A #16: Blindness cure?

by Rob Crossan

25th July 2007

Q: A cure for blindness - how many times have we heard this before?
A: Well, lots of times I would imagine. This one is worth paying some attention to though, as it's using the rather advanced sounding technique of 'gene therapy'.

Q: Gene therapy? Please explain. Does it hurt?

Human eye
A: Gene therapy is, in layman's terms, a way of inserting working genes, instead of mis-firing ones, straight into the retina. This gets rid of the faulty genes which prevent light collecting cells at the back of someone's eye from developing properly. It's worked already on mice and dogs, and trials are taking place over the next year at Moorfields Eye Hospital in London on 12 people with Leber's congenital amaurosis - a condition that causes progressive sight loss.

Q: So when will we know if it works on humans?

A: The results won't be announced until well into next year, as the trials are taking place over 12 months. Even after that, the details will stay strictly under wraps until experts at Moorfields are happy they can release them to the public accurately.

Q: Is such gene therapy likely to be dangerous?

A: The process involves producing copies of the healthy gene - which can be created in a harmless virus. This virus is then injected into the eye which, experts hope, will be absorbed by the retina and not repelled. The belief is that this will happen as the retina has little immunity and would find it hard to defend a virus.

Q: Can the treatment work on anyone?

Moorfields Eye Hospital
A: Potentially, yes. But the research team, led by Professor Robert McClaren, a consultant at Moorfields, believe that the chances of success at first are going to be higher on younger people who have Leber's.

Q: So will this incredible treatment only apply to people with Leber's congenital amaurosis?

A: At the moment, yes. However, hopes are that in time up to 30,000 visually impaired and blind people could benefit from the treatment in the UK alone. More common eye conditions which cause blindness - such as age-related macular degeneration - are very much in the Moorfield team's, er, sights.
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