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Beyond expectations: Groundbreaking gene therapy fully restores hearing in deaf UK toddler

Mother with children reading a book

In the UK, a clinical trial of a new groundbreaking treatment helped a toddler to hear for the first time in her life. An 18-month-old girl Opal Sandy was born with auditory neuropathy – a condition that disrupts nerve impulses travelling from the inner ear to the brain. The condition can be caused by a faulty gene and as a result, Opal couldn’t hear anything – until now.

The British girl from Oxfordshire had her hearing restored thanks to the new gene therapy treatment DB-OTO from biotech company Regeneron trialed by the University of Cambridge, The Guardian reported.

Auditory neuropathy is associated with mutations in the OTOF gene that encodes a protein called otoferlin which enables cells in the ear to communicate with the hearing nerve. Therefore, fixing the otoferlin production is instrumental to hearing restoration efforts.

The clinical trial aims to assess different doses of DB-OTO in up to 18 children (24 months of age or younger) to see how effective the treatment is and whether it is safe. As the University of Cambridge explains:

“DB-OTO is a gene therapy that has been created to deliver a working version of the OTOF gene to the inner ear. The DB-OTO injection is given into the inside of the ear during a surgical procedure that takes place under general anesthesia. This surgery is similar to cochlear implant surgery, which is well-established and has been used since the 1960s to treat deafness in babies.”

While Opal can hear “almost perfectly” following the surgery that took only 16 minutes, The Guardian reports that in the meantime, a second child was treated with DB-OTO with “positive results.”

Professor Manohar Bance, an ear surgeon and chief investigator for the trial, was heard saying that the initial results were better than he hoped or expected and went as far as claiming that the success “marks a new era in the treatment for deafness.”

It is worth mentioning that science and technology offer many different approaches to treating severe conditions, some of which we previously couldn’t cure at all. Among those approaches are BCIs, or brain-computer interfaces developed by Neuralink and Synchron, among others.

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