Child With Congenital Blindness Can See After Gene Therapy

Born with a retinal disease that made him legally blind, and would eventually leave him totally sightless, Corey Haas used to sit in the back of the classroom, relying on the large print on an electronic screen and assisted by teacher aides. Now, after a single injection of genes that produce light-sensitive pigments in the back of his eye, he sits in front with classmates and participates in class without extra help.

Harry Smith spoke to a Corey Haas and his family about how the experimental genetic surgery has restored his sight.


Harry Smith: Corey, Nancy and Ethan Haas join us this morning, along with Dr. Steve Rose, Chief of Research of Office for the Foundation of Fighting Blindness. Good morning to everybody.

Guests: Good morning

Harry Smith: First, help me understand what this disease is exactly.

Dr. Steve Rose: This is a disease where the back of the eye, the retina, does not function correctly, doesn’t see light, and therefore, leads to blindness.

Harry Smith: And so, this is relatively rare and it’s a genetic — is it a malfunction?

Dr. Steve Rose: It, in fact, is a missing gene. And in Corey’s particular case, this is a totally missing gene, one that doesn’t work right, isn’t there, and therefore, his retina is not seeing light and is not working.

Harry Smith: So, where did the gene come from then that was placed in his eye?

Dr. Steve Rose: It came from normal human DNA. The researchers at Children’s Hospital of Philadelphia actually took that DNA and were able to put it back into Corey’s eye and make the retina work.

Harry Smith: I need to talk to mom and dad here for a minute. Ethan and Nancy, what has this been like before the injection to realize you’ve got a child with a congenital problem that heretofore there isn’t much of an answer for?

Ethan Haas: Right, heartbreaking

Harry Smith: Yeah, difficult. And, how did you then find out about this … about this opportunity?

Ethan Haas: From a doctor we were seeing in Boston. She learned about the study and asked if we would be possible in participating.

Harry Smith: Yeah. And what was your reaction when you heard about this, that maybe there might be something that might be able to help you a little bit?

Corey Haas: If there’s something that was going to help me see, then I’m all for it.

Harry Smith: All for it, yeah, absolutely. Were you scared when you … because you have to go … it was an operation, right?  Is it an operation?

Corey Haas: Yeah

Harry Smith: Yeah. Were you kind of scared a little bit?

Corey Haas: Yeah

Harry Smith: Yeah, and then how long was it before you could … you knew there was a change?

Corey Haas: Oh, four days

Harry Smith: About four days?

Corey Haas: Yeah

Harry Smith: Yeah. I want to show some tape of Corey going through this obstacle course before, right, he’s got the patch over one eye and it’s difficult, it’s difficult for him to get through this obstacle course, right? And it took … how long did it take mom?

Nancy Haas: I believe this one took almost over four minutes.

Harry Smith: Over four minutes. You’ve got the best attitude; I’ll tell you what, you know, for having gone through all of this stuff. So, it takes four minutes and then, we see you with the new gene therapy, and let’s take a look at this. How long does it take this time around?

Nancy Haas: A matter of seconds

Harry Smith: Look at this, look at that.

Nancy Hass: He zips right through.

Harry Smith: You are motoring through there man. Dude.

Corey Haas: It isn’t dark.

Harry Smith: What’s that?

Corey Haas: They can’t even Photoshop me; it’s that dark. [inaudible]

Ethan Haas: Yeah, the [inaudible] was pretty light.

Harry Smith: Oh, OK, yeah. So, how big a difference is it? You went on the tour bus yesterday, can you see? Can you see things you couldn’t see before?

Corey Haas: Probably

Harry Smith: Yeah, yeah. And what has it been like for you?

Corey Haas: Good

Ethan Haas: Good, really good. His independence has increased and he’s able to play like a normal child now.

Harry Smith: Oh, my God

Ethan Haas: Just run around, play, and I don’t have to worry about him tripping over everything.

Harry Smith: Holy cow. Wow. How widely available might this be? And what do you think it means in the larger sense, doctor?

Dr. Steve Rose: The whole point of this, of the foundation Fighting Blindness and supporting this research has been to make this widely available. So, there are lots of clinical trials ongoing, and this really sets the stage for many more diseases, retinal diseases and eye diseases, going forward.

Harry Smith: Corey, rocking through that, rocking through that obstacle course, dude. Thanks very much. Good to have you on the program, mom and dad, Dr. Rose. Appreciate it. Alright, for more information on this, all you need to do is go to our website, it’s

Related Centers and Programs: Division of Ophthalmology