London specialists preserve sight in children with rare genetic eye condition in world first

National World - LocalTV

Specialists from University College London and Moorfields Eye Hospital have treated children with a rare genetic eye condition in a world first.

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00:00This is a particularly severe condition.

00:02Children born with very little sight at all,

00:05typically they can only distinguish light and dark,

00:08and that little sight they will lose within the first few years of life.

00:12So it's really devastating in terms of its impact on their sight

00:15and, of course, on their general development.

00:18So without treatment, that was the expectation,

00:21and that's why the parents were so concerned for their kids.

00:24Some children are born with very little sight, essentially blind,

00:28because they lack one of the many genes that are needed

00:31for their eyes to work properly.

00:33Thanks to world-leading institutions here in London,

00:36specialists from both Moorfields Eye Hospital and University College London

00:42have successfully preserved sight in young children through gene therapy.

00:48Four children were selected for the gene therapy,

00:52and given the rarity of the condition,

00:55all from abroad and taken to London for this experimental treatment.

01:01The genes are delivered to the eye in an operation

01:04that takes about an hour to do.

01:07The genes are packed in harmless viruses,

01:11which are delivered in a suspension to the retina

01:13during keyhole surgery under general anaesthetic.

01:17And the effect of that is to kick-start, if you like,

01:22the sensitivity of the retina,

01:24and the expectation is that they might have some benefit to their sight

01:27within a few weeks or months.

01:30And indeed, we were delighted to see that that was indeed the case.

01:35Parents of one of the young patients

01:37have hailed the results of the gene therapy as amazing.

01:42It's been pretty amazing.

01:43I think when we went into the surgery,

01:45we knew the risks for Jace and for us,

01:49and we had no expectations that this was a long-term treatment.

01:54I think nobody really knew what the outcome was going to be.

01:57I think for us at the time,

01:59the best hope was that this would have some positive effect on Jace's vision

02:05in some capacity, in any capacity,

02:07just to help him navigate the world and learn the world a little bit better.

02:11However amount of time that was, that was a positive for us and for him.

02:17But since then, and I hate to use the word progression,

02:20but in a way it really is Jace's progress in his vision,

02:24specifically in the eye that was treated,

02:27he's picking up small things off the floor,

02:29he's calling out toys,

02:32he's doing things that are really driven by his sight.

02:37All of these children had severe retinal dystrophy,

02:41known as Leber congenital amaurosis,

02:44an inherited condition that causes vision loss.

02:48We understand the patients who were essentially blind at birth

02:53can now see shapes, find toys, faces,

02:56and even in some cases, read and write.

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