Gene therapy cured deafness

“In a small trial in China, a single-injection gene therapy improved hearing in all ten congenitally deaf participants. One even gained near-normal hearing within just four months,” reports The Doomslayer.

ScienceDaily reports:

A new gene therapy is giving people born deaf the chance to hear, often within just weeks. In a small but groundbreaking study, researchers delivered a working copy of a key hearing gene directly into the inner ear using a single injection. All ten patients, ranging from young children to adults, experienced improved hearing, with some showing rapid gains in just one month….

Researchers at Karolinska Institutet, working with hospitals and universities in China, treated ten patients and saw hearing improve in every case….

The trial included ten patients between the ages of 1 and 24 who were treated at five hospitals in China. All had a genetic form of deafness linked to mutations in a gene called OTOF. These mutations prevent the body from producing enough of the protein otoferlin, which is essential for sending sound signals from the inner ear to the brain. To address this, researchers used a synthetic adeno-associated virus (AAV) to deliver a working version of the OTOF gene directly into the inner ear. The treatment was given as a single injection through a membrane at the base of the cochlea known as the round window.

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