A multicenter study found a one-time treatment targeting the OTOF gene restored hearing in most participants, with effects lasting up to 2.5 years. An international study co-led by investigators from Mass General Brigham and the Eye & ENT Hospital of Fudan University shows that a gene therapy for a rare form of genetic deafness restored hearing in approximately 90% of participants, with results lasting up to 2.5...
Audiologists treating patients with OTOF-related deafness (a genetic condition causing the inner ear's sound-signalling cells to malfunction) should be aware this therapy may represent a future treatment pathway, but it remains investigational — no change to routine clinical practice is warranted yet.
Durable hearing restoration from a single gene therapy dose in a genetic deafness population marks a significant milestone that could eventually reshape how congenital hearing loss is managed.
- 01A multicenter study co-led by Mass General Brigham and Fudan University tested OTOF-targeting gene therapy.
- 02A single treatment restored hearing in most participants with OTOF-related deafness.
- 03Hearing benefits persisted for up to 2.5 years post-treatment.
- 04OTOF mutations disrupt the protein (otoferlin) needed for inner ear hair cells to send sound signals to the brain.
- 05This is among the longest follow-up data yet reported for inner-ear gene therapy in humans.
A one-time OTOF-targeting gene therapy restored hearing in most participants with OTOF-related deafness.
studysupportedHearing restoration effects lasted up to 2.5 years after a single gene therapy dose.
studysupported- Publication type
- research_article
- Evidence level
- 2b
- Population
- Children with OTOF gene mutation-related congenital deafness
- Intervention
- Single-dose OTOF-targeting gene therapy delivered to the inner ear
Primary outcomes
Hearing restoration (audiometric thresholds); Durability of hearing improvement at up to 2.5 years follow-up