UK Girl Born Deaf Can Now Hear, Thanks To Pioneering Gene Therapy

A little girl from Oxfordshire in the United Kingdom is able to hear for the first time in her life after she underwent a pioneering gene therapy. Opal Sandy was born with a hearing impairment and underwent treatment shortly after her first birthday. Now, six months since, the toddler can hear unaided and is starting to talk. Her parents, Jo and James, proudly shared with BBC News that their little girl can hear sounds as soft as whispers and says words like 'mama', 'dada', or 'uh-oh!'  

Little Girl Born Deaf Can Now Hear

Opal Sandy's story of being born with a hearing and speech impairment to now responding to sounds and interacting is heartwarming. The little girl is one of the world's first patients to receive this one-of-a-kind gene therapy, BBC News reported. 

Developed by a biotechnology Research and Development firm called Regeneron, the therapy involves an infusion into the ear which reportedly replaces the faulty DNA causing the inherited deafness.

Doctors are also exploring similar treatments for the Otof gene mutation that Sandy has. Sandy's parents told the outlet about the nerve-wracking experience of allowing the little girl to be one of the first to test this treatment. 

Jo explained, "It was really scary, but I think we'd been given this unique opportunity." Opal's sister Nora also reportedly has the same type of deafness and manages well wearing an electrical cochlear implant.

Little girl Opal had the therapy in her right ear, under general anaesthetic, and a cochlear implant put into her left. For a few weeks, she could only hear loud sounds, but six months on, doctors reported that her hearing was now normal.

BBC reported that the implant that Nora uses does not make sounds louder, but rather gives the "sensation" of hearing by directly stimulating the auditory nerve that communicates with the brain.

The device bypasses the damaged sound-sensing hair cells in a part of the inner ear known as the cochlea. On the other hand, Opal's gene therapy treatment uses a modified, harmless virus to deliver a working copy of the Otof gene into these cells.

The gene therapy reportedly replaces the damaged sound-sensing DNA with a working copy. Experts have commended the scientists for the successful experiment. Scientists hope that the therapy can also work for profound hearing loss.

"It's wonderful seeing her respond to sound," chief investigator and ear surgeon Prof Manohar Bance at Addenbrooke's Hospital in Cambridge told BBC News. "What I am hoping is that we can start to use gene therapy in young children."

According to experts, hearing loss caused by a variation in the Otof gene is not commonly detected until children are two or three years old when a speech delay is likely. They are hopeful that gene therapy can replace implants or aids.

"The younger we can restore hearing, the better for all children because the brain starts to shut down its plasticity [adaptability] after the age of about three or so," Prof Bance told BBC. Scientific data from Opal's experience is being presented at the American Society of Gene and Cell Therapy in Baltimore, in the US.