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.