New studies aim to restore hearing to deaf children – using gene therapy

While the Novartis trial was ongoing, Lustig and others worked on ways to restore hearing in cases of genetic hearing loss. Lustig and his employees proved this in 2019 OTOF Gene therapy could be used for this purpose Restoring hearing in mice lacking the otoferlin protein. A separate group in Germany published similar results in 2021This shows that the results could be reproduced.

“For any gene therapy to work, your target cell has to be alive, not dead,” says Jeffrey Holt, a professor of otolaryngology and neurology at Harvard Medical School, who researches gene therapy for deafness but is not involved in the current studies. However, many genetic mutations associated with deafness cause the hair cells in the inner ear to die OTOF Mutations leave these cells intact. “That bodes well for this strategy,” he says.

While the trials are open to participants up to age 18, Holt says it may be best to give gene therapy earlier in life. “The hearing system goes through a maturation process, and if you deliver this at a time after the system has already matured and there is no sound, we don’t really know how it will handle this new information input,” he says. For this reason, adults find it more difficult to learn a new language than children. Children’s brains are very plastic – this means they can easily make new connections and learn new things.

Doctors recommend cochlear implant surgery before age three so children can learn sounds while their language skills develop. Older people can also benefit from the devices.

While the current studies focus on a rare type of deafness, company researchers believe other genetic mutations that cause deafness could be treated with gene therapy.

But not everyone believes that deafness requires medical intervention. Jaipreet Virdi, a deaf historian of medicine, technology and disability at the University of Delaware, says that gene therapy is a continuation of the debate that began in the 1990s over cochlear implants, which some deaf people see as a threat to the cochlear implants consider the deaf community. They argue that implanting deaf children before they have a chance to acquire language denies them autonomy and access to deaf culture. “Expungement before the election — of an individual, not their parents — is problematic,” Virdi says.

Wyatte Hall, a psychologist and health researcher at the University of Rochester who studies the role of language acquisition in the health of deaf people and is deaf himself, says hearing parents with a deaf child may see medical interventions or technology as a way to help their child fit into the world as they know it. But Hall says deaf people contribute to society’s wealth. “For as long as there have been deaf people on earth, people have always tried to fix us,” he says. “The fact that we are still here suggests that we still have inherent evolutionary value and that our differences contribute to the world in which we all live.”

He’s not necessarily against cochlear implants and gene therapy, but he doesn’t think parents should restrict access to sign language in favor of a purely medical approach. When working with families of deaf children, he emphasizes a “both” approach – the use of technology And Sign language. “When gene therapy or technology doesn’t work,” says Hall, “sign language serves as a protection for development.”