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

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In the UK, a clinical trial of a groundbreaking new treatment helped a toddler hear for the first time in her life. 18-month-old Opal Sandy was born with auditory neuropathy – a condition that disrupts nerve impulses traveling 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 restored her hearing thanks to the new DB-OTO gene therapy treatment from the biotech company Regeneron tested by the University of Cambridge, the guardian report.

Auditory neuropathy is associated with mutations in the OTOF gene that codes for a protein called otoferlin that allows cells in the ear to communicate with the auditory nerve. Therefore, correcting autoperlin production is instrumental in hearing restoration efforts.

The clinical trial is designed to evaluate different doses of DB-OTO in up to 18 children (aged 24 months and under) to see how effective the treatment is and whether it is safe. As the University of Cambridge explains:

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

While Opal can hear “almost perfectly” following the operation, which lasted just 16 minutes, the Guardian reports that in the meantime, a second boy has been treated with DB-OTO with “positive results”.

Professor Manohar Bans, an ear surgeon and principal investigator in the trial, was heard saying that the initial results were better than he had hoped or expected and went as far as to claim that the success “signals a new era in the treatment of deafness”.

It is worth mentioning that science and technology offer many different approaches to treating serious conditions, some of which in the past we could not cure at all. Among these approaches are BCIs, or brain-computer interfaces developed by Neuralink and Synchron, among others.

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