July 30, 2025
esearchers at Boston Children’s Hospital used gene therapy to restore hearing in mice born deaf due to mutations in the OTOF gene.
The team delivered a healthy version of the gene directly into the inner ear of newborn mice. Weeks later, the mice showed normal responses to sound—a first for congenital hearing loss treatment in mammals.
“This is a major step toward curing genetic deafness,” said Dr. Gwenaelle Geleoc, co-author of the study published in Science Translational Medicine.
Mutations in OTOF prevent proper signaling between sound-sensing cells and the auditory nerve. Fixing this could help thousands of newborns with similar conditions.
Human trials may follow in coming years, though challenges remain in translating the therapy safely.
