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Gene therapy helps doctors restore hearing for children born deaf
Doctors say the treatment may last a lifetime and is limited to children with a specific genetic cause of deafness.
- Dr. John Greinwald, an otolaryngology attending at Cincinnati Children, treated two young siblings from Uzbekistan for congenital deafness using gene therapy after the family sought his molecular genetics expertise.
- A new study published in Nature Medicine reports researchers at Karolinska Institutet and Chinese hospitals treated ten patients born with OTOF gene mutations; all participants showed hearing improvements.
- Delivered via a synthetic AAV injection, the therapy enables the body to produce otoferlin protein. Sound detection improved from an average of 106 decibels to 52 decibels.
- Patients aged 1 to 24 tolerated the treatment well with no serious adverse reactions during the 6 to 12 month follow-up. "This is a huge step forward," says Maoli Duan, consultant and docent at Karolinska Institutet.
- Expanding beyond OTOF, researchers are targeting other common genes like GJB2 and TMC1. Greinwald believes this breakthrough will lead to therapies for a wide range of genetic hearing loss conditions.
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Deafness reversed: One injection restores hearing in just weeks
A new gene therapy is giving people born deaf the chance to hear, often within just weeks. In a small but groundbreaking study, researchers delivered a working copy of a key hearing gene directly into the inner ear using a single injection. All ten patients, ranging from young children to adults, experienced improved hearing, with some showing rapid gains in just one month.
·Encinitas, United States
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