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A groundbreaking gene therapy has enabled six deaf children, aged one to 11, to experience sound for the first time in their lives. These children, part of experimental groups in China and the US, were born with a gene mutation hindering the production of a crucial protein for hearing. The researchers, who came from multiple institutions across the two countries, injected a virus that could pass on a modified version of the gene, called OTOF, directly into the inner ear. The cells of the ear were able to incorporate the gene and begin producing the crucial protein, otoferlin. After 26 weeks of therapy, the children’s hearing levels improved by up to 70%, with progress evident as early as six weeks. Videos documenting their progress reveal heartwarming moments, such as a one-year-old responding to his name and a girl uttering words like ‘father’ and ‘I love you’ for the first time. While the specific scope of this research was aimed at a rare genetic defect, the broader breakthrough of being able to functionally cure hereditary, genetic deafness has been likened to the introduction of the cochlear implant in the 1970s.
