Between October 19, 2022 and June 9, 2023, 425 participants were screened and six children were eventually enrolled in the trial. They had all been diagnosed with autosomal recessive deafness 9, which is caused by mutations in a gene called otoferlin, or OTOF. This had led to the patients’ severe to complete bilateral hearing loss.
Six months after the gene therapy, five of the children showed significantly improved hearing.
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Shu Yilai, lead scientist of the study, said in an interview with the Chinese new media platform Bioworld that the children’s hearing had “reached a level similar to that of 60-65 per cent” of the general population.
“On average, they went from being unable to hear below 95 decibels – similar to the sound of a motorbike – to being able to hear around 45 decibels, which is close to a normal speaking voice,” said Shu, who is a professor at Fudan University’s Eye and ENT Hospital.
Worldwide, 26 million people are living with congenital deafness. In China, around 30,000 babies are born every year with hearing impairment or loss, 60 per cent of which is due to genetic disorders. This can severely affect their language, speech and cognitive development.
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