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Gene Therapy Restores Hearing in 90% of Congenital Deafness Patients

4/24/2026

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Story summary
  • A single-dose viral gene therapy targeting the OTOF gene restored hearing in participants with years-long congenital deafness.
  • Researchers treated 42 Chinese participants and confirmed hearing gains over a 2.5-year follow-up.
  • Approximately 90% of treated ears improved and half of the participants reached near-normal hearing.
  • The trial reported no serious treatment-related adverse events.
  • Younger children and participants treated in both ears showed the greatest speech and language improvements.