🧏 Trying to treat deafness in children with gene therapy

• A new gene therapy is currently being tested with the goal of restoring hearing in children with auditory neuropathy.
• Treatment focuses on correcting an error in the OTOF gene, which is critical for sound transmission.
• Results of the multinational study are expected in February.

The first gene therapy trial has begun, aiming to treat a rare form of genetic deafness in children. The trial focuses on children with auditory neuropathy, a genetic disease that prevents the transmission of sound signals from the ear to the brain, despite normal sound perception.

Bridging the communication gap

At the heart of auditory neuropathy lies an error in the OTOF gene, which is crucial for the production of the protein otoferlin. This protein plays a key role in enabling communication between ear cells and the auditory nerve. A fault in the OTOF gene prevents this communication, thus reducing the transmission of sound from the inner hair cells to the auditory nerve. The gene therapy being tested is designed to introduce a working copy of the OTOF gene to correct this defect.

Using a neutralizing virus as a delivery vehicle, the treatment is given by injection into the inner ear. The method is divided into a three-stage strategy: an initial dose in one ear, followed by a higher dose in one ear, and finally treatment in both ears.

It opens the potential for wider applications

Although the treatment is experimental, it has the potential to provide better hearing quality compared to a cochlear implant. In addition, early intervention is crucial because the young brain develops rapidly, making early treatment more effective.

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