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Deaf Siblings Hear for First Time Via Gene Therapy
16 Oct
Summary
- Gene therapy allows deaf children to hear without devices.
- Two siblings from Uzbekistan received breakthrough treatment.
- The therapy targets a rare OTOF gene variant causing deafness.
A revolutionary gene therapy is enabling deaf children to hear, a development previously considered unexpected by medical professionals. Cincinnati Children's Hospital in Ohio is at the forefront of this medical advancement.
Two young siblings from Uzbekistan, 3-year-old Abdu and his 1-year-old sister Sumaya, traveled to Ohio to participate in a clinical trial for a rare form of congenital deafness. Their condition stemmed from a biallelic variant in the OTOF gene, which is crucial for hearing.
The experimental gene therapy, known as Otarmeni, was administered to both children. This treatment works by delivering a gene therapy product directly to the cochlea.
Following the procedure, Abdu began detecting sounds within four weeks and is now hearing within the normal range, simultaneously developing speech. His sister, Sumaya, also underwent the treatment with similarly positive outcomes.
Published results in The New England Journal of Medicine indicate that nine out of the first 12 participants in the trial achieved hearing levels down to at least 70 decibels or less within six months post-surgery. Researchers describe this success as a "medical miracle" and a significant step forward for gene therapy in treating deafness.
