Gene therapy delivered a functional OTOF gene to the inner ear of 10 patients aged 1–24, improving hearing in all participants.
Hearing improvements appeared as early as one month after treatment, reducing average perceptible sound threshold from 106 dB to 52 dB at six months.
Children aged 5–8 showed the strongest response, but significant gains were also observed in adults.
The treatment was safe and well tolerated, with the only common side effect being reduced neutrophil counts and no serious adverse events in 6–12 months follow-up.
Researchers plan to extend gene therapy approaches to other deafness genes such as GJB2 and TMC1 based on promising animal studies.
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