Gene Therapy Restores Hearing In Both Ears
A groundbreaking study reveals that gene therapy has successfully restored hearing in both ears of children born deaf. The research, conducted by scientists at the Eaton-Peabody Laboratories at Mass Eye and Ear, found that all five children in the study experienced significant improvements in speech perception and sound localization after receiving the therapy.
According to co-senior researcher Zheng-Yi Chen, the results are remarkable, demonstrating not only enhanced hearing abilities but also newfound appreciation for music in two of the children. This achievement marks a significant breakthrough in treating genetic hearing conditions, particularly DFNB9, which is caused by mutations in the OTOF gene and affects millions worldwide.
DFNB9 hampers the production of otoferlin protein crucial for auditory and nerve functions related to hearing. Currently, there are no pharmacological treatments available for inherited deafness conditions like DFNB9. However, gene therapy has emerged as a promising solution, as evidenced by previous successful trials restoring hearing in one ear for five children.
In this study, researchers aimed to assess the efficacy of gene therapy in restoring hearing in both ears simultaneously. Lead researcher Dr. Yilai Shu from the Diagnosis and Treatment Center of Genetic Hearing Loss at Fudan University spearheaded the administration of functioning copies of the OTOF gene via virus injections into the inner ears of the children.
Despite concerns about potential immune responses to the double dose of the virus, the treatment showed no serious toxicity or adverse effects. The study, published in Nature Medicine, underscores the potential of gene therapy in addressing various forms of deafness, both genetic and non-genetic.
Chen emphasizes the importance of expanding this approach to treat other forms of deafness and ultimately aims to help individuals regain their hearing regardless of the underlying cause. This breakthrough holds promise for improving the quality of life for millions affected by hearing loss worldwide.
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