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.

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[–] jpreston2005@lemmy.world 20 points 5 days ago* (last edited 5 days ago)

Ok, so the study was specifically targeting 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.

researchers used a synthetic adeno-associated virus (AAV) to deliver a working version of the OTOF gene directly into the inner ear.

Super cool! And now they're targeting other genetic forms of deafness hoping to deliver similar results. Neat!

"OTOF is just the beginning," says Dr. Duan. "We and other researchers are expanding our work to other, more common genes that cause deafness, such as GJB2 and TMC1. These are more complicated to treat, but animal studies have so far returned promising results. We are confident that patients with different kinds of genetic deafness will one day be able to receive treatment."