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Gene Therapy Restores Hearing In Children

Gene Therapy Restores Hearing In Children

A groundbreaking new therapy has restored hearing in children who were born deaf.

As detailed in a new study this week, six children were participants in the research project. After receiving an experimental gene therapy treatment, hearing was restored in five of the six participants. All of the children completed follow-up visits, which showed no dose-limiting toxicity or serious adverse events.

The study took place at Fudan University in Shanghai and was funded by Chinese science organizations and biotech company Shanghai Refreshgene Therapeutics.

As the Associated Press (AP) reported, researchers observed the participants for roughly six months. Though they are unable to determine why the therapy did not work for one participant, the five for whom it did work can now hear conversations and are able to talk to others.

Zheng-Yi Chen of Boston’s Mass Eye and Ear, a senior author of the study, told the AP that the children now hear at a level around 60 to 70 percent of normal.

Other recent studies have also shown considerable improvement among children who have undergone the procedure.

The first participant in one study, an 11-year-old at the time the treatment was administered, had profound hearing loss from birth, but had restored hearing within 30 days.

On Jan. 30, the Children’s Hospital of Philadelphia announced improvements in the boy’s hearing. Though the sound is muffled, as if wearing foam earplugs, he is now able to hear his father’s voice and road traffic.

"What we have really accomplished is exciting because it's the first time hearing has been restored in a child — really in a human being — for a hereditary type of hearing loss," Dr. John Germiller of the Children's Hospital of Philadelphia told CBS News. "We've always wanted to be able to restore natural hearing, and we've been waiting 30 years for this ... and its finally here and that's restoring a defective gene you're born with."

The treatment is a minimally invasive endoscope that allows gene therapy to be placed directly into the inner ear.

"The idea here is to replace a defective gene that a child is born with, restore that gene to the inner ear by literally injecting it into the inner ear," Germiller says. "It's a surgical procedure to do that, but it's exciting because if we can get the gene back in and get it functioning, it should restore hearing."

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