Children with genetic deafness have hearing restored with gene therapy, study finds

Children with hereditary deafness regained their hearing thanks to a type of gene therapy, a new study published on Wednesday found.

In a clinical trial, co-led by investigators from Mass Eye and Ear, a specialty hospital in Boston, six children who had a form of genetic deafness called DFNB9 were examined.

This deafness is caused by mutations of the OTOF gene. This mutation fails to produce a protein known as otoferlin, which is necessary for the transmission of sound signals from the ear to the brain, according to the researchers.

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The trial — which began in December 2022 — took place at Eye & ENT Hospital of Fudan University in Shanghai and involved the use of an inactive virus carrying a functioning version of the OTOF gene.

It was carefully introduced into the inner ear of the six children at differing doses, and they were observed for 26 weeks.

Results, published in The Lancet, showed five of the six children, who were classified as having total deafness, recovered their hearing “and the restored ability to conduct normal conversation,” according to a release.

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