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Gene therapy restores hearing to children with inherited deafness
The first clinical trial to administer gene therapy to both ears in one person has restored hearing function to 5 children born with a form of inherited
The first clinical trial to administer gene therapy to both ears in one person has restored hearing function to 5 children born with a form of inherited deafness, astounding the research team.. It is caused by mutations in the OTOF gene which prevent the production of functioning otoferlin protein, which can significantly reduce sound transmission from the inner ear hair cells to hearing nerves. Through minimally invasive surgery, Shu injected adeno-associated virus (AAV) engineered to carry and deliver functioning copies of the human OTOF transgene, into the children’s inner ears.
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