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May 10, 2024 | News


First time gene therapy restores the hearing of a toddler


A girl born deaf is now able to hear without any assistance after receiving a groundbreaking gene therapy treatment.


Opal Sandy, a woman from Oxfordshire in England, was born afflicted with auditory neuropathy, a genetic disorder. Auditory neuropathy is caused by a variation of a single gene called the OTOF gene. This gene produces a protein known as otoferlin, which allows inner hair cells to communicate with hearing nerves. A mutation in the OTOF genes is responsible for the deafness of approximately 20,000 people in the UK, Germany France, Spain, and Italy.


Opal is the world’s first patient to undergo a gene therapy trial. The results were “mind-blowing”.


“Gene Therapy has been the future of otology for many years, and I am so excited that it’s finally here,” said Prof Manohar Balce, an ear specialist at Cambridge University Hospitals NHS Foundation. This is the beginning of a new age for gene therapy for the inner ear, and for many types of hearing impairment.

Jo Sandy , , Opal’s mom, said: “When Opal heard us clapping without assistance, it was mind-blowing. We were so happy.”



Image: Opal, Jo, and James, her father. Credit: Cambridge University Hospitals

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