Gene Therapy Shows Promise for Congenital Deafness

Approximately 1–2 in 1000 babies worldwide are born with hearing loss due to genetic mutations. One such mutation in the OTOF gene disrupts neurotransmission from cochlear inner ear cells. Earlier small trials suggested that using a viral vector to deliver a normal copy of the OTOF gene into these cells could restore hearing, but follow-up was short.

In this multicenter, single-arm trial in China, 42 patients (3 adults and 39 children; mean age, 6 years) with complete hearing loss due to OTOF mutations received gene therapy at one of three doses and were followed up to 2.5 years.