Gene therapy for blindness

06 February 2014

Interview with

Nell Barrie

Kat -  Now, the first story I wanted to talk about was a really exciting but a very small clinical trial of gene therapy for a particular type of blindness.  What have the scientists done here?  This is really exciting.

Nell -   Yes.  This is really cool, especially because I remember learning about gene therapy back at school many years ago and being told, "Yes, this is the future in 10 or 15 years.  We're going to be treating all kinds of diseases using gene therapy."  As we know now, it's proved a lot more difficult than that for a lot of complicated reasons.  This is really small trial with six patients, looking at a condition called choroideremia and it's a type of progressive blindness caused by faulty gene called CHM.  And what the researchers are actually doing is using a viral vector, so a virus, to introduce a new section of DNA into the eye.

Kat -   And the results seem quite promising.  What have they found so far?

Nell -   So, yes, it does look really exciting.  They've actually found that people's vision did get better.  It's not a kind of immediate, "Oh my God, I can suddenly see again."  They looked at the acuity, so the kind of acuteness or the clearness of these people's vision, which is what they measure when you're looking at those little lines of text on the sight charts at the opticians.  I'm very bad of this because I'm very short-sighted.  And they found that that acuity did go up.  And also, patients were able to see better in the dark so you're getting some real benefits here, but it's really important just to emphasize that this is a phase one trial, so all they were trying to find out was whether this was a safe way to use this treatment.

So it's promising they've got these results, but they need to do bigger studies before they can find out whether this is really going to help.  The researchers think that it could help with age-related macular degeneration, which is actually pretty common and that makes about 300,000 people blind every year in the U.K.  So, it's definitely possible that if we can spot these kinds of things earlier on, if we know that we can treat them out in earlier stage, then it could really be a useful way to stop people from getting to the point where they start to lose their vision in the first place, which should be great.

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