Gene therapy trials restore hearing to children born deaf

A gene therapy trial has successfully provided children who were born deaf the ability to hear.

The trial, conducted in Fudan, China, at the Fudan University’s Eye & ENT Hospital and by a team at the Massachusetts Eye and Ear Infirmary and Harvard Medical School, six children who suffer from a gene mutation that affects signals transmitted from the ear to the brain were treated.

The children, ages one through seven, participated in a 26-week trial in 2022 that examined the otoferlin gene. Five out of six of them showed improvement or recovery in hearing, the study found. Speech perception also improved in children who had hearing recovery.

By the end of the 26-week study, the three older children who had cochlear implants turned off could understand and respond to speech. Two were able to recognize speech in a noisy room and have a telephone conversation, the Harvard Gazette noted.

The Children’s Hospital of Philadelphia announced Tuesday that initial results of the trial in a patient indicate the treatment was successful.

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