Breaking Barriers: Gene Therapy Shows Promising Results in Restoring Hearing for Deaf Children with OTOF Mutations

In Cambridge, a trial of Opal gene therapy has shown promising results, with one child achieving near-normal hearing restoration. Though it is not yet known whether she can fully understand speech, the child can hear soft sounds almost normally for her age. Another child has also received the treatment and has shown positive results six weeks after surgery. This trial provides valuable information on the effectiveness of gene therapy for cases of deafness caused by specific genetic mutations.

Professor Bance estimates that there are around 20,000 people in the US, Germany, France, Italy, Spain, and the UK who have auditory neuropathy due to OTOF mutations. The National Deaf Children’s Society believes that this trial will provide valuable information on the effectiveness of gene therapy for cases where deafness has a specific genetic cause. With the right support, deafness should never be a barrier to happiness or fulfillment.

Opal and her sister Nora enjoy making noise and playing together. Opal has started talking in the last six weeks and both sisters like to see who can make the most noise. Opal enjoys playing with her musical instrument set while Nora likes reading to her and dancing with her in the kitchen. Despite their differences, the sisters enjoy spending time together.

Meanwhile, in Baltimore, results from the Chord trial were presented at the American Society of Gene and Cell Therapy conference showing promising outcomes for those with auditory neuropathy caused by genetic mutations. The potential for gene therapy to cure deafness caused by genetic mutations is an exciting development in the field of hearing restoration.