2024-05-09 13:23:18
There was news that a baby born with hearing impairment in the UK had his hearing restored through gene therapy.
According to the British daily The Times and dpa News on the 9th (local time), Opal Sandy, an 18-month-old child living in Oxfordshire, England, had a congenital hearing impairment that prevented her from hearing anything.
However, Opal participated in a clinical trial to treat hereditary hearing loss with gene therapy, and is now said to have regained almost normal hearing. It is known that Opal is the first person to regain hearing through gene therapy.
Opal was suffering from hearing problems due to a disease called ‘auditory neuropathy‘, which interferes with the nerve impulses that transmit sound to the brain. The disease may be caused by a defect in the OTOF gene, which produces a protein that allows cells in the ear to communicate with the auditory nerve.
Opal participated in a clinical trial led by Cambridge University Hospitals NHS Foundation Trust, which provides public health services. The clinical trial focused on using the latest gene therapy to replace defective cells by sending a copy of the gene carrying the ear’s function to the cells of the ear.
Opal was the first patient in the clinical trial, which is scheduled to be conducted on up to 18 subjects in the UK, Spain, and the US.
Last September, Opal underwent surgery to inject a copy of a gene created by American biotechnology company Regeneron into her right ear.
Afterwards, Opal reacted to loud noises for the first time three weeks after surgery. Opal’s mother, Jo Sandy (33), recalled the situation at the time, saying, “I couldn’t believe it when the baby turned his head for the first time.”
“I was so surprised,” he said in a text message to his husband. He thought it was a coincidence. “I really can’t believe it.”
An examination in February, 24 weeks after the surgery, showed that Opal’s hearing had improved to the point where she could even hear whispers. Opal started speaking in the last six weeks.
Dr. Manohar Vance, the clinical trial’s principal investigator, called these results “dramatic” and said, “(The treatment) is a one-time treatment, and we expect (Opal) to return to her daily life after treatment.”
He added, “This clinical trial is just the beginning of gene therapy.”
It is estimated that approximately 20,000 people suffer from auditory neuropathy caused by OTOF gene mutation in the US, UK, Germany, France, Spain, and Italy.
Choi Jae-ho, Donga.com reporter [email protected]
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2024-05-09 13:23:18