ISD Team
03 Jul 2025
A young boy undergoes a routine ear examination by a medical professional using an otoscope.

A recent study has found that gene therapy can greatly enhance hearing in both children and adults with congenital deafness linked to mutations in the OTOF gene. The treatment involves using a synthetic virus to deliver a functional copy of the gene directly into the inner ear. All ten participants showed signs of hearing improvement within the first month.

The youngest patients showed the most dramatic progress—one seven-year-old recovered nearly normal hearing and conversational skills. The therapy was deemed safe and well-tolerated, with no serious side effects reported during the 6 to 12 months of follow-up.

Key Facts:

  • Targeted Gene: Replaces the faulty OTOF gene associated with hearing loss.
  • Quick Results: Most participants experienced hearing gains within a month.
  • Safe and Promising: No major side effects observed; best results noted in younger children.

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