More children gain hearing as gene therapy for profound deafness advances

Enlarge / Opal Sandy (center), who was born completely deaf because of a rare genetic condition, can now hear unaided for the first time after receiving gene therapy at 11-months-old. She is shown with her mother, father, and sister at their home in Eynsham, Oxfordshire, on May 7, 2024. (credit: Getty | Andrew Matthews)

There are few things more heartwarming than videos of children with deafness gaining the ability to hear, showing them happily turning their heads at the sound of their parents' voices and joyfully bobbing to newly discovered music. Thanks to recent advances in gene therapy, more kids are getting those sweet and triumphant moments—with no hearing aids or cochlear implants needed.

At the annual conference of the American Society for Gene & Cell Therapy held in Baltimore this week, researchers showed many of those videos to their audiences of experts. On Wednesday, Larry Lustig, an otolaryngologist at Columbia University, presented clinical trial data of two children with profound deafness—the most severe type of deafness—who are now able to hear at normal levels after receiving an experimental gene therapy. One of the children was 11 months old at the time of the treatment, marking her as the youngest child in the world to date to receive gene therapy for genetic deafness.

On Thursday, Yilai Shu, an otolaryngologist at Fudan University in Shanghai, provided a one-year progress report on six children who were treated in the first in-human trial of gene therapy for genetic therapy. Five of the six had their hearing restored.