Gene Therapy Restored Hearing in Deaf Child
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Gene Therapy Restored Hearing in Deaf Child: [Paywalled in some regions]
Regeneron's gene therapy is being tested in a clinical trial.
An experimental gene therapy gave a deaf child the ability to hear, her family and investigators for a clinical trial say.
Opal Sandy, the girl, received an injection of DB-OTO, Regeneron's gene therapy, in her ear when she was 11 months old. Her hearing was assessed as normal within six months.
"When Opal could first hear us clapping unaided it was mind-blowing," Jo Sandy, the girl's mother, said in a statement released by the UK's National Health Service (NHS).
[...] "Providing the full complexity and spectrum of sound in children born with profound genetic deafness is a phenomenon I did not expect to see in my lifetime," Dr. Lawrence Lustig, chair of Columbia University's Department of Otolaryngology and one of the trial investigators, said in a statement.
Opal was born with genetic deafness from mutations of the otoferlin gene. About one in every 500 children are born in the United States with little or no ability to hear, and genetic changes like variants of the otoferlin gene are one cause.
Variants in the gene inhibit the production of a protein necessary for communication between the inner ear and the auditory nerve. The problem can be partially rectified with hearing aids or cochlear implants.
"It was our ultimate goal for Opal to hear all the speech sounds. It's already making a difference to our day-to-day lives, like at bath-time or swimming, when Opal can't wear her cochlear implant," James Sandy, Opal's father, said.
The DB-OTO is aimed at restoring the full spectrum of sound to people with mutated otoferlin gene. It's given as an intracochlear injection into one ear, or administered directly to the inner ear. A phase 1/2 clinical trial testing the gene therapy started in 2023, enrolling children in the United States, the United Kingdom, and Spain.
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