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Gene therapy trials restore hearing to children born deaf

Published in Tech News, Tools.

In this image from video provided by the Children’s Hospital of Philadelphia, an 11-year-old boy who was born with hereditary deafness prepares for a gene therapy procedure in Philadelphia in October 2023. On Tuesday, Jan. 23, 2024, the hospital announced his hearing has improved enough that he now has only mild to moderate hearing loss in the ear that was treated, and is hearing sound for the first time in his life. (Children’s Hospital of Philadelphia via AP)

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.