Gene Therapy Breakthrough Enables Hearing in Children with Profound Deafness

Recent clinical trials show promising results in restoring hearing through gene therapy, potentially revolutionizing treatment for genetic deafness.

Opal Sandy, an 11-month-old from the UK, gained the ability to hear without aids following a pioneering gene therapy treatment.
Clinical trials in the US and China demonstrate significant hearing restoration in children with a rare genetic mutation affecting auditory signals.
The gene therapy targets mutations in the OTOF gene, crucial for sound signal transmission to the brain, using virus vectors for delivery.
Researchers express optimism about the therapy's long-term effectiveness and its potential application to more common types of deafness.
The advancements were highlighted at the American Society for Gene & Cell Therapy's annual conference, underscoring a potential new era in treating genetic hearing loss.