Chinese trial restores hearing in congenital deafness with gene therapy
A Chinese clinical trial has achieved the world's first successful restoration of hearing in congenitally deaf children using gene therapy. Researchers injected a missing gene, OTOF, into the inner ear cells of six children aged 1 to 7 years. All participants showed significant hearing improvement, with some able to hear normal conversation. The trial was conducted at Fudan University's Eye & ENT Hospital in Shanghai and published in The Lancet. This breakthrough offers a potential cure for a form of deafness caused by mutations in the OTOF gene, which affects about 2-8% of congenital deafness cases. The therapy uses a harmless virus to deliver the correct gene into cochlear hair cells. No serious adverse effects were reported. The team plans to expand the trial to more patients and younger ages. This marks a major milestone in gene therapy for sensory disorders.
Key facts
- World's first clinical trial for congenital deafness gene therapy
- Targeted OTOF gene mutations
- Six children aged 1-7 treated
- Conducted at Fudan University's Eye & ENT Hospital, Shanghai
- Results published in The Lancet
- All participants showed hearing improvement
- Some can now hear normal conversation
- No serious adverse effects reported
- Uses adeno-associated virus (AAV) vector
- Plans to expand trial to more patients and younger ages
Entities
Institutions
- Fudan University
- Eye & ENT Hospital
- The Lancet
Locations
- Shanghai
- China