JANUARY 24, 2024 MORNING EDITION, NPR, Rob Stein
An experimental gene therapy tested in young children with an inherited form of deafness restored some hearing for most of them.
For the first time, gene therapy is showing promise for treating inherited deafness, researchers reported on Jan. 24, 2024.
A study involving six children born with a genetic defect that left them profoundly deaf found that an experimental form of gene therapy restored at least some hearing for five of them.
"We are absolutely thrilled," says Zheng-Yi Chen, an associate scientist at Mass Eye and Ear's Eaton-Peabody Laboratories and associate professor of Otolaryngology–Head and Neck Surgery at Harvard Medical School in Boston. Chen led the research, which was published in the journal The Lancet.
"This is really the first time that hearing has been restored in any adult or children by a new approach — a gene therapy approach," Chen tells NPR in an interview.
He says the researchers plan to try the approach with other forms of genetic deafness, as well as possibly hearing loss caused by age and noise. "That's something we're really excited about," Chen says.
WHAT IT'S ABOUT: Restoring a protein needed for hearing: The study involved children born with rare genetic defect in a gene that produces otoferlin, a protein necessary for the transmission of the sound signals from the ear to the brain. The researchers modified a virus commonly used to ferry genes into the body known as an adeno-associated virus to carry a functioning form of the gene into the inner ear.
Within weeks, five of the six children, who were between the ages of 1 and 7, began to be able to hear and the oldest child has been able to say simple words, Chen says. The children were treated at the EYE & ENT Hospital of Fudan University in China.
"Before the treatment they couldn't hear a thing. You could put the loudest sound in the ear and they don't hear anything," Chen says. "And now they can hear."
The children's hearing isn't completely normal — they may still need hearing aids — but improved significantly, Chen says. The treatment appears safe. The children have been followed for between six months and a year so far.
"It worked as well as we imagined," Chen says. "This really was beyond our expectations."
Chen and his colleagues have continued to treat additional patients and will follow the study subjects in the hope that the improvement is permanent.
"This is a very big deal. It's a new dawn for hearing loss," Chen says.
A first for treatment of hereditary deafness Other researchers agreed.
Several other groups are pursuing similar gene therapies for genetic deafness and will report their findings Feb. 3 at the annual meeting of the Association for Research in Otolaryngology.
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