A gene therapy called DB-OTO has shown promising results in improving hearing to normal levels in two children with congenital deafness. Conducted as part of an ongoing early-stage trial, the therapy targeted otoferlin-related deafness, with one child showing improvement within 6 months and the other within 6 weeks. Researchers plan to continue monitoring the participants for the next 5 years to assess the therapy’s long-term effectiveness. The success of this gene therapy highlights the potential for gene therapy in treating genetic deafness and the importance of genetic testing to explore alternative treatment options for pediatric patients with hearing loss.
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‘Astounding’ success for gene therapy in otoferlin-related deafness
