Gene Therapy Restores Hearing in Toddlers and Teenagers Born with Congenital Deafness

Strategic Insights -

Gene therapy has shown remarkable results in restoring hearing in toddlers and young adults born with congenital deafness, specifically in those with OTOF-related deafness. A study conducted by researcher Maoli Duan and their team has demonstrated that the therapy can be safely administered to patients of various ages, including those as young as one year old. The treatment involves injecting a modified virus carrying a working copy of the OTOF gene into the inner ear, which successfully restores hearing in patients.

Key Takeaways:

  • The study involved 10 participants aged 1-24 years with OTOF-related deafness, all of whom received the gene therapy and were closely monitored for 12 months.
  • The virus therapy was found to be safe, with only mild to moderate side effects, and no serious adverse events were observed.
  • The treatment resulted in significant hearing improvements, with an average total hearing improvement of 62% on objective brainstem response tests and 78% on behavioural hearing assessments.
  • Two participants achieved near-normal speech perception, and one participant's parent reported that their child could hear sounds just three days after treatment.
  • The study revealed that children between the ages of five and eight showed the most pronounced benefit from the treatment, but younger children and older participants also experienced improvement.
  • The brain's ability to process newly restored sounds may vary at different ages, but the exact reasons for this are not yet understood.

Statistics:

  • 10 participants aged 1-24 years with OTOF-related deafness received the gene therapy.
  • The average total hearing improvement reached 62% on objective brainstem response tests and 78% on behavioural hearing assessments within the first month of treatment.
  • 2 participants achieved near-normal speech perception.
  • 1 participant's parent reported that their child could hear sounds just three days after treatment.
  • The most common adverse effect was a decrease in white blood cells.
  • No serious adverse events were observed over the 12-month study period.

Sources:

  • The Conversation UK -- UK -- By Maoli Duan, Associate Professor, Senior Consultant, Karolinska Institutet
  • MIL OSI Analysis (http://milnz.co.nz/mil-osi-aggregation/) - Copyright 2025 Multimedia Investments Ltd, distributed by Contify.com