Deaf baby can hear after 'mind-blowing' gene therapy treatment [View all]

By Emily Cooke published 5 hours ago
Seven months after her treatment, the baby girl can now respond to her parents' voices without the aid of a cochlear implant.



Image of a toddler girl sat with her mother on her left and her father on her right. They are all smiling at the camera. The mother is wearing a black-and-white polka dot top, the toddler is wearing a bright yellow top and the father is wearing a grey shirt.
Opal Sandy from the U.K. was treated as part of an ongoing global trial investigating a new gene therapy for a rare type of congenital hearing loss. She's pictured in the image above with her parents. (Image credit: Cambridge University Hospitals NHS Trust)

A toddler who was born completely deaf due to a rare genetic condition can now hear unaided thanks to a pioneering gene therapy.

Opal Sandy, who is now an 18-month-old girl from the U.K., is the youngest child in the world to receive this type of gene therapy, which uses a harmless, modified virus to correct genetic mutations in the body's cells. In this case, the therapy replaced a mutant gene associated with deafness with a working copy of that gene, according to a statement released May 9 by Cambridge University Hospitals.

Very similar gene therapies are being tested in other trials and have shown early success in treating slightly older children with the same type of hearing loss as Opal.

Known as auditory neuropathy, this type of hearing loss arises when the ears can detect sound but can't relay that information to the brain. The condition is caused by mutations in a gene called OTOF that normally makes otoferlin, a protein needed for the inner ear to talk to neurons that are linked to the brain. The condition accounts for between 1% and 8% of cases of congenital hearing loss that occurs in the absence of other symptoms.

More:
https://www.livescience.com/health/genetics/deaf-baby-can-hear-after-mind-blowing-gene-therapy-treatment