Gene therapy restored hearing to five children with congenital hereditary deafness, two of whom gained the ability to appreciate music. The research team was also surprised by the results, published in the journal Nature Medicine. The clinical trial was conducted at the Eye, Ear, Nose and Throat Hospital affiliated to Fudan University and targeted patients with hereditary deafness DFNB9. The research team published the preliminary results of the study in the journal The Lancet earlier this year. DFNB9 is caused by mutations in the OTOF gene and the inability to produce a functioning otoferlin protein, which is required to transmit sound signals from the ear to the brain. The researchers observed six children with DFNB9 for 26 weeks. They used an adeno-associated virus (AAV) carrying the human OTOF gene and carefully introduced the gene into the patients' inner ears through a special surgical procedure. A single injection of the viral vector was used with different doses. Clinical trials are still continuing, and researchers say this gene therapy is “feasible, safe and effective.”
https://cosmosmagazine.com/science/biology/gene-therapy-restores-hearing-to-children-with-inherited-deafness/
https://www.nature.com/articles/s41591-024-03023-5
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