A girl who was born deaf can hear unaided after receiving groundbreaking gene therapy treatment.
Opal Sandy from Oxfordshire, England, was born with a genetic condition known as auditory neuropathy. Auditory neuropathy can be due to a variation in a single gene, known as the OTOF gene, which produces a protein called otoferlin that allows the inner hair cells in the ear to communicate with the hearing nerve. An estimated 20,000 people across the UK, Germany, France, Spain and Italy are deaf due to a mutation in the OTOF gene.
Opal is the first patient to be treated in a global gene therapy trial, which showed “mind-blowing” results.
“Gene therapy has been the future in otology and audiology for many years and I’m so excited that it is now finally here,” said trial leader Prof Manohar Bance, an ear surgeon at Cambridge University Hospitals NHS Foundation. “This is hopefully the start of a new era for gene therapies for the inner ear and many types of hearing loss.”
Jo Sandy, Opal’s mother, added: “When Opal could first hear us clapping unaided it was mind-blowing – we were so happy.”
Image: Opal with mother, Jo, and Father, James. Credit: Cambridge University Hospitals
