Gene Therapy Restores Hearing in Children with Hereditary Deafness

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A recent study published in The Lancet highlights the success of gene therapy in treating congenital deafness.

A recent study published on Wednesday revealed that children with hereditary deafness regained their hearing through a form of gene therapy.

A clinical trial, co-led by researchers from Mass Eye and Ear, a specialty hospital in Boston, focused on six children with a genetic deafness condition known as DFNB9.

The deafness in question is attributed to mutations of the OTOF gene. These mutations result in the absence of a crucial protein called otoferlin, essential for transmitting sound signals from the ear to the brain, as outlined by the researchers.

Commencing in December 2022, the trial was conducted at the Eye & ENT Hospital of Fudan University in Shanghai. It utilized an inactive virus carrying a functional version of the OTOF gene, which was carefully introduced into the inner ear of the six children at varying doses. The participants were then observed for a period of 26 weeks.

Published in The Lancet, the results revealed that five out of the six children, previously classified as experiencing total deafness, regained their hearing. Additionally, they demonstrated the restored ability to engage in normal conversation, as stated in a release.

Researchers emphasize that this is the inaugural human clinical trial utilizing gene therapy for treating this condition, encompassing the highest number of patients treated and the lengthiest follow-up period to date.

Researchers emphasize that this is the inaugural human clinical trial utilizing gene therapy for treating this condition, encompassing the highest number of patients treated and the lengthiest follow-up period to date.

This marks the first human clinical trial employing gene therapy to treat this condition, featuring the highest number of patients treated and the most extended follow-up period, according to the researchers.

Dr. Zheng-Yi Chen, an associate scientist in the Eaton-Peabody Laboratories at Mass Eye and Ear and associate professor of otolaryngology–head and neck surgery at Harvard Medical School, expressed, "For us, it really is a milestone. We are absolutely thrilled... It will have a huge impact on the field," as reported by ABC News.

"Hearing loss affects so many people and, up to now, there's no single FDA-approved drug to treat any type of hearing loss, including genetic hearing loss," Dr. Zheng-Yi Chen continued. "So, this is the first time where a brand-new type of gene therapy approach can actually be used to treat the children who are completely deaf and regain the function of hearing."

Dr. Zheng-Yi Chen highlighted that hearing impairment in children can significantly impact their development and result in lasting consequences. He emphasized the importance of early hearing screenings for infants, underscoring the critical role hearing plays in overall development.