Researchers at Shanghai’s Fudan University have restored partial hearing in four out of five deaf children using a novel gene therapy — injecting a modified virus carrying a corrected copy of a single faulty gene directly into the inner ear. The results mark a meaningful step forward in treating a rare but well-defined form of inherited deafness.
At a glance
- Gene therapy: Scientists modified a harmless virus to carry a working copy of the otoferlin gene, then injected it into each child’s cochlea — the fluid-filled, spiral-shaped cavity of the inner ear.
- Otoferlin gene defect: The target mutation disrupts a protein essential for transmitting sound signals from the inner ear to the brain, and accounts for 1 to 3 percent of all congenital deafness worldwide.
- Hearing restoration: Four of the five children who received the treatment gained roughly 60 to 65 percent of typical hearing ability, according to Fudan University head and neck surgeon Yilai Shu.
How the treatment works
The otoferlin gene encodes a protein that acts as a messenger at the synapse between the inner ear’s hair cells and the auditory nerve. Without a functioning copy, sound vibrations never become electrical signals the brain can read — and the child hears nothing from birth.
The Fudan team’s approach uses an adeno-associated virus, a well-studied viral delivery vehicle that is harmless on its own. The researchers loaded it with a corrected otoferlin sequence and injected it directly into the cochlea. Once inside, the virus enters the hair cells and begins producing the working protein.
The cochlear injection route matters. Delivering the therapy locally means the corrected gene stays concentrated where it is needed, limiting exposure elsewhere in the body. It also reflects years of work refining surgical technique to reach the inner ear safely in pediatric patients.
Why these results stand out
Four out of five children recovering measurable hearing from a single intervention is a striking early result. Most of the children treated had heard very little or nothing since birth, so even partial restoration opens the door to spoken language development during the critical window of early childhood.
Shu and his colleagues are not alone in this race. Researchers at the University of Cambridge have a parallel otoferlin program underway, and pharmaceutical company Regeneron reported in late 2023 C.E. that a child in its own clinical trial was showing encouraging early signs — evidence that the field is converging on a similar biological target from multiple directions.
The Fudan results were reported through MIT Technology Review, which spoke directly with Shu about the trial’s scope and outcomes.
What comes next
The therapy currently targets only the otoferlin mutation, which means it applies to a small fraction of the roughly 26 million people worldwide living with genetic hearing loss. Otoferlin defects account for just 1 to 3 percent of congenital deafness cases. That is an important ceiling on the immediate reach of this treatment.
Still, the scientific community sees the Fudan work as a proof of concept for a broader gene therapy approach to hearing. If a single corrected gene can unlock hearing in children with otoferlin mutations, the same platform logic — modified virus, targeted cochlear delivery, restored protein — could in principle be adapted for other single-gene hearing disorders as more of them are characterized.
There are also open questions about durability. It is not yet clear how long the restored hearing holds, whether booster treatments will be needed, and how outcomes compare in children treated at different ages. Longer follow-up data and larger trials will be essential before the therapy moves toward standard clinical use.
For the four children already hearing, those questions are for the future. For now, a door that was closed from birth has opened.
Read more
For more on this story, see: Futurism / Neoscope
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