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Gene Therapy Trial Restores Legally Blind Boy's Sight

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9-year-old Corey Hass figured he would never see again: suffering from an inherited disease called Leber’s congenital amaurosis, Corey was classified as legally blind. Naturally, Corey’s family was willing to try anything to give him a chance so that he could participate in normal childhood activities. Thus, when doctors at The Children’s Hospital of Philadelphia offered Corey an opportunity to be part of an experimental gene therapy trial, Corey and his parents decided to take the chance. Miraculously, Corey can see again. In fact, he started seeing results just four days after the procedure.

Corey received a procedure in which scientists used DNA from a DNA bank to create a missing gene in Corey’s DNA. The gene was injected into Corey’s eye with a thin needle and will now make the missing protein inside Corey’s retina to restore his vision.

While Corey admits he was scared before the procedure, he now says he finds it “amazing” that he can see again. Doctors at the Foundation Fighting Blindness, the group that helped support the research for Corey’s procedure, is hopeful that the procedure will become more widely available. With many clinical trials currently ongoing, the doctors are also hopeful that similar procedures could help treat other retinal and eye diseases.