Revolutionary gene therapy successfully restores hearing in a young girl from the UK.

A British girl who was born deaf is now able to hear without assistance thanks to a groundbreaking gene therapy treatment.

Opal Sandy, who received treatment shortly before her first birthday, is now able to hear even soft sounds like whispers and has begun to speak, uttering words such as "Mama," "Dada," and "uh-oh." The groundbreaking therapy, administered as an infusion into her ear, replaces faulty DNA responsible for her type of inherited deafness.

Opal is part of a trial that is enrolling patients in the UK, US, and Spain. Similar treatments for the Otof gene mutation Opal has are also being explored by doctors in other countries, including China.

While Opal's parents, Jo and James from Oxfordshire, acknowledge that the results have been astonishing, they admit that allowing Opal to be the first to undergo this treatment, developed by Regeneron, was incredibly daunting.

Nora, Opal's five-year-old sister, also has the same type of deafness but manages well with an electrical cochlear implant. Unlike hearing aids, which amplify sound, the cochlear implant provides a "sensation" of hearing by directly stimulating the auditory nerve that communicates with the brain. This stimulation bypasses the damaged sound-sensing hair cells in a part of the inner ear called the cochlea.

How do sounds appear with a cochlear implant?

In contrast, the treatment employs a modified, harmless virus to introduce a functional version of the Otof gene into these cells.

Opal underwent the therapy in her right ear, while under general anesthesia, and received a cochlear implant in her left ear.

Within just a few weeks, she began to perceive loud sounds, like clapping, in her right ear.

Six months later, Opal's doctors at Addenbrooke's Hospital in Cambridge confirmed that her ear had nearly normal hearing for soft sounds, including very faint whispers.

"It's a very joyful time."

Experts are optimistic that the therapy could potentially be effective for other forms of profound hearing loss as well.

More than half of all cases of hearing loss in children are linked to genetic factors.

Professor Bance hopes that the trial can pave the way for gene therapy to be utilized in treating more prevalent forms of hearing loss.

"I envision a future where gene therapy becomes a viable option for young children, enabling us to effectively restore their hearing without the need for cochlear implants or other replaceable technologies," he stated optimistically.