🧏 Curing deafness in children with gene therapy - trail begins

• A novel gene therapy trial aims to restore quality hearing in children with auditory neuropathy.
• Treatment focuses on rectifying a fault in the OTOF gene, which is pivotal for sound transmission.
• Initial results from the multinational trial expected by next February.

A first-of-its-kind gene therapy trial has commenced, with the objective of curing a rare form of genetic deafness in children. The trial zeroes in on children afflicted with auditory neuropathy, a genetic condition that hampers the transmission of sound signals from the ear to the brain, despite normal sound detection.

Bridging the communication gap

At the root of auditory neuropathy is a malfunction in the OTOF gene, crucial for the production of otoferlin protein. This protein is a key player in enabling the cells within the ear to communicate with the hearing nerve. A defect in the OTOF gene hampers this communication, thereby reducing sound transmission from the inner hair cells to the hearing nerves. The gene therapy on trial is designed to introduce a functional copy of the OTOF gene to rectify this defect.

Employing a neutralised virus as a delivery vehicle, the treatment is administered by injecting it into the inner ear, under general anaesthesia. The methodology is broken down into a three-phase approach: an initial dose in one ear, followed by a higher dose in one ear post confirming safety, culminating with treatment in both ears.

Unlocking potential for broader applications

The therapy, although experimental, has the potential to furnish better quality hearing compared to cochlear implants. Furthermore, timely intervention is crucial as the young brain rapidly evolves, making early treatment more effective.

Success in this venture could unfold new horizons for tackling hearing loss caused by other genetic conditions, thereby amplifying the potential impact of gene therapy.

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