CHOP helps restore vision in 2 children using experimental gene editing treatment

PHILADELPHIA (CBS) — A first-of-its-kind genetic treatment at Children's Hospital of Philadelphia is restoring and improving vision, according to a study released on Monday.

The treatment was tested on 12 adults and two children who have a genetic form of vision loss. According to the results published, the two children at CHOP had significant improvements. 

"It was just an amazing experience," father Andrew Peckham said. 

Andrew Peckham's 11-year-old son Jacob was one of the two children who received the experimental treatment. 

"It's really cool just being one of the first to experience the treatment," Jacob, of Waren, Michigan, said. 

Jacob was born with a genetic condition that causes vision loss. He said his vision is better following the treatment received in one eye using gene editing to correct the DNA deficiency. 

"We jumped right on it," Andrew Peckham said. 

The two children in the clinical trial were treated at CHOP and Penn. 

"The study basically delivers this genetic treatment underneath the retina after a surgical procedure, it's an injection," Dr. Tomas S. Aleman said. 

Aleman is a retina specialist who led the trial in Philadelphia. 

"Both patients improved their vision," Aleman said. "We were pleasantly surprised that there was a major improvement to this very new therapy never before used in the eye." 

Since he had the treatment two years ago, Jacob said he enjoys going out more with friends and is playing baseball. 

"It's really cool," Jacob said. "Before I couldn't see that far away. Now it's like very clear. It's a lot clearer than before... I can see a lot better."

If approved by the FDA, Jacob hopes to have the treatment on his other eye. The family is already thrilled with the improvements so far. 

"Just the day-to-day stuff that he was struggling with before, it's not as a struggle as now. So, it's just an amazing process," Andrew Peckham said. 

Doctors are hoping once the treatment is approved they'll be able to use it on younger patients when the results are expected to be even better. 

In:
• Health
• Children's Hospital of Philadelphia
• CRISPR
• Philadelphia
• Children

Stephanie Stahl

Stephanie Stahl is an Emmy Award-winning health reporter. She can be seen daily on CBS News Philadelphia and Philly57.