World’s First Gene Therapy Trial Successfully Cures Deaf Girl

A groundbreaking gene therapy trial has successfully restored the hearing of an 18-month-old British girl, making her the first in the world to benefit from this innovative treatment. Opal Sandy, who was born completely deaf due to auditory neuropathy, underwent the “one and done” gene therapy at Addenbrooke’s Hospital in the UK.

Professor Manohar Bance, the chief investigator for the trial, expressed his excitement over the results, stating that Opal’s hearing has improved significantly and could potentially lead to a cure for patients with this type of deafness. The therapy involves delivering a working copy of the OTOF gene, responsible for making the otoferlin protein, to the ear to enable communication between cells in the ear and the hearing nerve.

Opal’s parents noticed improvements in her hearing within weeks of the surgery, with Opal responding to sounds like loud clapping and whispers. The results have been described as “spectacular” by the medical team, with Opal’s hearing now close to normal.

The success of this trial marks a new era in the treatment of deafness, with potential implications for other genetic-related hearing conditions. While the treatment is not yet available on the NHS, the study paves the way for future advancements in gene therapies for deafness.

Opal’s story serves as a beacon of hope for families dealing with similar conditions, showcasing the transformative power of gene therapy in restoring hearing and improving quality of life.