AAV gene therapy for autosomal recessive deafness 9: a single-arm trial
Nature Medicine, Published online: 02 July 2025; doi:10.1038/s41591-025-03773-w
Preliminary results from an investigator-initiated clinical trial showed that an AAV-OTOF gene therapy was safe and led to hearing improvements in ten patients with congenital deafness with 6–12 months of follow-up, including in a teenager and a young adult, expanding the age range from previous trials.
AAV gene therapy for autosomal recessive deafness 9: a single-arm trial
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