Gene therapy helps restore hearing in kids
It is an effective treatment for patients with a specific form of autosomal recessive deafness caused by mutations of the OTOF (otoferlin) gene, called DFNB9
image for illustrative purpose
New York: Researchers have developed a gene therapy that can help restore hearing function for children suffering from hereditary deafness.
In the first human clinical trial that took place in December 2022, the team from Massachusetts Eye and Ear Infirmary in the US administered gene therapy to six children in China.
The results, now published in the journal The Lancet, found the gene therapy to be an effective treatment for patients with a specific form of autosomal recessive deafness caused by mutations of the OTOF (otoferlin) gene, called DFNB9.
"If children are unable to hear, their brains can develop abnormally without intervention," said Zheng-Yi Chen, Associate Scientist at Mass Eye and Ear.
"The results from this study are truly remarkable. We saw the hearing ability of children improve dramatically week by week, as well as the regaining of their speech," added Chen, Associate Professor of Otolaryngology - Head and Neck Surgery at Harvard Medical School.
Hearing loss affects more than 1.5 billion people worldwide, with congenital deafness making up about 26 million of those individuals. For hearing loss in children, more than 60 per cent have a genetic cause.
DFNB9 for example, is a hereditary disease caused by mutations of the OTOF gene and a failure to produce a functioning otoferlin protein, which is necessary for the transmission of the sound signals from the ear to the brain.
There are currently no FDA-approved drugs to help with hereditary deafness, which has opened the door for new solutions like gene therapies.
