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Gene therapy restores hearing in children with hereditary deafness
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Gene Therapy Successfully Reversifies Hereditary Deafness in Kids
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HMS Scientist Co-Leads Study to Correct Inner Ear Gene Mutation
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<p>A novel gene therapy for hearing loss was administered to six children in China in a clinical trial co-led by investigators at Mass Eye and Ear in Boston and Eye & ENT Hospital of Fudan University in Shanghai, China. Each child had an inherited deafness caused by mutations in the <em>OTOF </em>gene<em>,</em> called DFNB9. The researchers report in a new study in <em>The Lancet</em>, after 26 weeks, five children demonstrated hearing recovery and dramatic improvements in speech perception and the restored ability to conduct normal conversation. With its first patient treated in December 2022, this research represents the first human clinical trial to administer gene therapy for treating this form of hearing loss, with the most patients treated and longest follow-up conducted to date.</p>