We are proud to share that Dr. Fan-Gang Zeng of the UC Irvine Department of Otolaryngology has co-authored a landmark article published in this month’s edition of Nature Medicine, one of the world’s most prestigious scientific journals.
The paper presents results from the first multicenter clinical trial of gene therapy for hereditary deafness, specifically targeting mutations in OTOF, a gene crucial to the transmission of information from the ear to the brain. Involving ten patients ranging from 1.5 to 23.9 years old, the study demonstrates both the safety and efficacy of an AAV-OTOF vector injection.
The findings are remarkable:
- All participants tolerated the gene therapy procedure well, with no serious adverse events reported.
- Most notably, the intervention restored hearing in individuals previously diagnosed with complete deafness, improving thresholds to the level of moderate hearing loss (approximately 50 dB HL).
- Hearing gains occurred rapidly—largely within the first month of treatment.
This work also breaks new ground in demonstrating successful bilateral and repeat injections, with encouraging results in adolescents and adults, expanding the potential scope of future treatment.
Dr. Zeng’s contribution marks a significant milestone in auditory neuroscience and therapeutic innovation. These promising findings not only deepen our understanding of gene therapy’s biological mechanisms but also offer real clinical hope to individuals with hereditary hearing loss.
Congratulations to Dr. Zeng on this exceptional achievement and the recognition it brings to UC Irvine Department of Otolaryngology’s leadership in hearing and communication sciences.
