PARIS: A gene therapy, described as a “game changer,” is creating optimism as several children born deaf regain the ability to hear in what could be the first major breakthrough in treating hereditary deafness in decades.
Researchers worldwide are exploring this transformative procedure, focusing on a rare genetic mutation that affects a small subset of the 26 million people globally suffering from inherited deafness.
A recent success story emerged from the Children’s Hospital of Philadelphia, announcing that 11-year-old Aissam Dam, born deaf, is now experiencing sound for the first time. This achievement follows a trial in China, reported in The Lancet journal, where six children received a similar gene therapy, with five gaining the ability to hear, marking the first human trial of its kind.
Ray of Hope for Children Born Deaf
The therapy targets a genetic mutation in the OTOF gene, affecting people who cannot produce the vital protein otoferlin. This protein is essential for hair cells in the inner ear to convert sound vibrations into electrical signals transmitted to the brain. The treatment involves injecting a harmless virus carrying a functional OTOF gene into the inner ear, effectively restoring hearing.
Researchers anticipate a groundbreaking shift in therapeutic care, potentially revolutionizing treatments for various forms of hereditary deafness. Trials in France and the United States, utilizing a comparable technique, further underscore the potential of this gene therapy.
While this advancement addresses a specific mutation affecting around three percent of congenital deafness cases, it offers hope for expanding the treatment to cover a broader range of genetic hearing loss causes. The success of the otoferlin treatment in both mice and human trials opens doors for future therapies.
