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Gene therapy restores hearing in toddlers and teenagers born with congenital deafness – new research


This is the first time such results have been achieved in both children and adult patients born with a specific type of congenital deafness.

But recent research my colleagues and I conducted has shown that a form of gene therapy can successfully restore hearing in toddlers and young adults born with congenital deafness. Unlike other types of genetic deafness, people with OTOF mutations have healthy hearing structures in their inner ear – the problem is simply that one crucial gene isn’t working properly. In our study, we used a modified virus as a delivery system to carry a working copy of the OTOF gene directly into the inner ear’s hearing cells.

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