Gene therapy yields lasting gains for patients with inherited deafness

BEIJING: An experimental gene therapy for people with an inherited form of deafness led to durable hearing improvements, a new study shows, with associated gains in patients’ ability to recognise speech.

The research corrected mutations in the OTOF gene, one of about 200 genes whose mutations are known to cause deafness from birth.

Patients 18 and younger saw the strongest gains in hearing and ability to recognise speech.

42 patients. One injection each. 90% could hear again.

The largest gene therapy trial for inherited deafness just published in Nature. Researchers delivered a working OTOF gene directly into the inner ear using an AAV vector.

Average hearing went from 106 dB to 52 dB. Half the… pic.twitter.com/WEyNn8cSrX

— Avi Roy (@agingroy) April 23, 2026

Improvements

Adults receiving the therapy also saw improvements, though the effect was smaller.

Overall, 90% of recipients saw their hearing improve, with half reaching normal levels by the study’s end at 2½ years.

The study, published April 22 in Nature, was conducted by researchers at Mass Eye and Ear, Harvard Medical School, and Fudan University, with additional trial sites in China, and involved 42 participants carrying the OTOF mutation and ranging in age from nine months to 32 years.

They were treated at eight trial centers across China.

Nature research paper: Multicentre gene therapy for OTOF-related deafness followed up to 2.5 yearshttps://t.co/IhXmtnuqj9

— nature (@Nature) April 23, 2026

Rehabilitation

Worldwide, about 430 million people are affected by hearing loss serious enough to require rehabilitation, including 34 million children, according to the World Health Organisation (WHO).

Sixty percent of deafness in newborns has genetic causes, with mutation in the OTOF gene responsible for between 2% and 8% of cases.

Babies with the OTOF mutation are completely deaf at birth, which affects speech acquisition and can hinder cognitive development.