Most babies born with otoferlin deafness get cochlear implants in infancy and are hence ineligible for your demo. The implants somewhat alter the cochlea, which could hamper the interpretation of gene therapy benefits.

The mutated otoferlin gene destroys a protein within the interior ear’s hair cells essential to transmit sound towards the brain. With lots of the other mutations that bring about deafness, hair cells die in the course of infancy or perhaps at the fetal phase.

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The here goal of the gene therapy is to replace the mutated otoferlin gene in patients’ ears with a functional gene.

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