First Gene Therapy Cure for Blindness

First Gene Therapy Cure for Blindness

Leber congenital amaurosis is basically
an inherited disorder that causes some blindness that occurs shortly after birth.
And this is first noticed by the family fairly rapidly. And typically what the
patient has, what the child has, is decreased night vision and also
decreased central vision. Creed did not make eye contact with me. And we also noticed that if there wasn’t
direct sunlight Creed was very emotional. Well I could not see a rainbow. And also I needed a flashlight to see my books and
everything else. We found out that he was RPE 65
which is one of the rarest mutations. But one of the ones they have a cure for. So the treatment that Creed
received is a sub-retinal injection. So we go inside the eye, we detach the
retina. And in that area of the detachment we do it with a medication.
So we are infecting the retina, the sub-retinal space with that gene that
he’s missing. So he can start seeing again. And Sarah had sent me recently a
picture when she was in the bath with him and she said this is the first time
Creed has looked at me in the eyes. And I said, mom this is the most powerful moment. I see everything right now, after the gene replacement. Our life has changed.
It’s actually harder in a lot of ways. Creed doesn’t just sit still anymore.
But it’s changed for the better. It’s fun to teach him different things.
And it’s fun to see him succeed in all these things
that he missed out on in life. I think it’s exciting because it’s the very first
time that we can even treat such patients. But I think more importantly, it probably
offers hope to other patients that we have other research, other studies, that
could eventually also apply to them. We are fixing the genetic makeup of
somebody to allow them to do something that they couldn’t do before.

5 Replies to “First Gene Therapy Cure for Blindness”

  1. My cousin also has the same issue with his eyes since his childhood, is this treatment successful . how should i contact doctors regarding this, kindly help.

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