Gene Transfer Helps Reverse Blindness

POSTED: Monday, October 26, 2009 - 8:01am

UPDATED: Thursday, June 3, 2010 - 11:59pm

Watching 9 year-old Cory Haas walk to the podium at the Children's Hospital of Philadelphia, without using a cane, thrilled the audience of journalists and doctors. Cory was born with Leber's Congenital Amaurosis.

It is a genetic disease that had robbed most of the 4th graders vision and would eventually leave him completely blind. It's the result of damaged genes he inherited from his parents. Last September doctors from Children's Hospital and Penn Medicine injected healthy genes into Cory’s worst eye.

There is research video of Cory before the gene transfer. He attempts to walk through an obstacle course on the videotape. You can see him bump into a stop sign obstacle that is only inches from his face.

In another part of the video, he's feeling along the wall for a doorknob. There is another video of Cory walking through the same course after he received the healthy gene. It's clear he can see where he's going. Children's Hospital Dr. Katherine High is one of the researchers involved in the trial.

"I don't think I'll see anything as amazing in the whole span of my career…" High said. Corey's parents say it was tearing them up, watching their little boy slip into darkness. They say they knew the gene therapy was working when Cory suddenly noticed the sun. "He would stare at lights...” Cory’s Mom Nancy Haas said.

“He would stare at the sun and it wouldn't effect his vision... Or he wouldn't see anything... So when he said the sun is really bright I said wow!" Doctors don't know if the treatment will be permanent. They say it should last based on animal studies. 11 others in the study report improved vision. The best success has been seen in children. Cory says what he loves most about being able to see are. "Colors definitely colors." Said Cory. When asked what his favorite color was? "Red."

He says he now loves playing baseball. Even though this New Yorker admits to being a Mets fan, he plans to watch the World Series.