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Toddler born with hearing loss can now hear thanks to new gene therapy trial

Therapy offers hope for a potential cure for certain types of hearing loss

09-May-2024

Key points from article :

An 18-month-old British toddler, Opal Sandy, born with profound deafness due to a faulty gene, had her hearing almost fully restored through a pioneering gene therapy trial.

The groundbreaking surgery at Addenbrooke's Hospital, part of Cambridge University Hospitals, involved a 16-minute infusion of a working copy of the OTOF gene, responsible for producing a protein essential for hearing.

This world-first trial, called Chord, is recruiting more deaf children from the UK, Spain, and the US to assess the long-term effectiveness and safety of the gene therapy.

The treatment uses a harmless virus to deliver the working gene to the inner ear, potentially offering a cure for children with this specific type of genetic deafness.

Early results from the trial have been exceptionally promising, with Opal now able to hear near-perfectly and enjoy activities like playing with toy drums.

While this gene therapy marks a significant advancement in treating deafness, further research and longer-term follow-up are necessary to determine its full potential and applicability to other forms of hearing loss.

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Cambridge University Hospitals NHS Foundation Trust (CUH)

Family of hospitals comprising Addenbrooke’s and The Rosie.

Topics mentioned on this page:
Gene Therapy, Hearing Loss
Toddler born with hearing loss can now hear thanks to new gene therapy trial