Overview
- Researchers report in Nature on Wednesday that an inner‑ear gene therapy improved hearing in about 90% of 42 people with OTOF‑related deafness, with many detecting sound within weeks and about half reaching normal levels by 2.5 years.
- The treatment uses an adeno‑associated virus to deliver a working OTOF gene into cochlear hair cells so they can pass sound signals to the brain, restoring the link that is missing in DFNB9.
- The study spanned eight hospitals in China and treated participants aged about 10 months to 32 years with a single injection in one ear for most and in both ears for six, using one of three doses.
- Investigators reported no serious treatment‑related side effects or dose‑limiting toxicity across dose groups, and hearing gains tended to plateau around 26 weeks and then hold steady.
- Responses were age‑dependent with 100% restoration in children under three and 92% in those aged 3 to 8, smaller gains in adults, about 10% non‑responders, and plans for five‑year follow‑up plus regulatory steps in China and platform work for other genes such as GJB2.