Co-Led by the team of Zheng-Yi Chen. Featured in HMS News and the Harvard Gazette.

A novel gene therapy approach has given five children who were born deaf the ability to hear. The method, which overcomes a roadblock presented by large genes, may be useful in other treatments, according to researchers.

The work, conducted in Fudan, China, by a team co-led by Harvard Medical School researchers at Massachusetts Eye and Ear and by collaborators at Fudan University’s Eye & ENT Hospital, treated six children aged 1 to 7 who had a mutation of the OTOF gene, which manufactures a protein important in transmitting signals from the ear to the brain. Five of the children showed improvement in hearing over the 26-week trial, with four outcomes described by researchers as “robust”.