Children with deafness are gaining the ability to hear thanks to recent advances in gene therapy, presenting a groundbreaking development in the field of medicine. Researchers from Columbia University and Fudan University shared their findings at the American Society for Gene & Cell Therapy conference, showcasing videos of children with profound deafness who have regained their hearing without the need for traditional aids like hearing aids or cochlear implants.
Larry Lustig from Columbia University presented data from a clinical trial, revealing that two children with profound deafness now have normal levels of hearing after receiving experimental gene therapy. Yilai Shu from Fudan University reported on the progress of six children who underwent gene therapy for genetic deafness, with five of them experiencing restored hearing.
Initially, the trials focused on treating one ear for safety measures but have since advanced to bilateral treatment, resulting in all five additional patients achieving hearing restoration in both ears. The gene therapy targets mutations in the OTOF gene, allowing for the production of the essential protein otoferlin in the inner ear to restore hearing.
Patients showed improvements in their hearing within weeks to months after the treatment, although the need for additional therapies in the future remains uncertain. This gene therapy specifically targets a type of deafness known as auditory neuropathy caused by recessive mutations in a single gene, impacting a small percentage of individuals with deafness worldwide.
Researchers are optimistic about the success of this gene therapy, believing it could pave the way for the development of treatments for more common types of deafness in the future. This groundbreaking achievement offers hope for children with deafness and their families, transforming their lives by restoring the precious gift of hearing.
