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AAV-OTOF Gene Therapy Restores Hearing in Patients Aged 1 to 24

Six-month results from a 10-participant trial in China show marked decibel gains with a favorable safety profile ahead of long-term monitoring.

Image
Audiologist doing impedance audiometry or diagnosis of hearing impairment. (© Dusko - stock.adobe.com)
© PeopleImages.com - Yuri A via Shutterstock
The results also show that the treatment was safe and well-tolerated. Credit: Neuroscience News

Overview

  • Ten patients aged 1 to 24 received a single inner-ear AAV-OTOF injection at five hospitals in China.
  • Measurable hearing recovery began within one month for most participants.
  • At six months, average pure-tone thresholds improved from 106 decibels to 52 decibels across all patients.
  • No serious adverse events occurred; the most common side effect was a temporary drop in neutrophil counts.
  • Researchers have started extended follow-up to evaluate durability and plan to adapt the approach to other deafness genes like GJB2 and TMC1.