Thanks to experimental gene therapy, five of the six Chinese infants who were born deaf due to a rare genetic abnormality are now able to hear of deafness gain
The therapy involved a hollowed-out virus loaded with a healthy version of the gene responsible for producing otoferlin, a protein necessary for the transmission of sound signals from the inner ear to the brain. Doctors injected the virus into the inner ears of the children through a special surgical procedure, and five of the kids experienced hearing recovery within six months.
“These studies’ findings are genuinely astounding. We observed children’s hearing abilities increase significantly each week, along with their speech returning, according to Zheng-Yi Chen, a researcher at Harvard-affiliated Mass Eye and Ear in Boston who works as an associate scientist in Eaton-Peabody Laboratories.
Deafness Gain
The six youngsters were treated by doctors in China in collaboration with Mass Eye and Ear researchers, and the trial was conducted at the Eye & ENT Hospital of Fudan University in Shanghai. These outcomes support those of a U.S.-based experiment that treated an 11-year-old Moroccan boy receiving treatment at the Children’s Hospital of Philadelphia for otoferlin deafness, and the trial’s findings were successful. The Chinese trial treated its first patient more than a year ago, in December 2022, and thus represents the first human clinical trial to administer gene therapy for otoferlin deafness, researchers said.
Deafness Gain
The Moroccan boy received his gene therapy in October in one ear. Within four months, his hearing improved enough that he now has only mild to moderate hearing loss in the treated ear, according to a CHOP news release. “There’s no sound I don’t like,” patient Aissam Dam told the New York Times through an interpreter. “They’re all good.” About one in 500 infants is born with or develops hearing loss during early childhood, according to the U.S. Centers for Disease Control and Prevention.
Deafness Gain
More than 60% of hearing loss cases in children stem from genetic reasons, researchers said in background notes. Mutation of the otoferlin gene is one of the reasons for congenital deafness. But because this condition is caused by a lack of the signaling protein, researchers had hoped it could be reversed with gene therapy. About 200,000 people around the world are affected by otoferlin deafness, the Times reported. All six children in the Chinese study had total deafness due to their otoferlin mutation, researchers said.
Deafness Gain
Within a half-year of treatment, five of the kids had demonstrated hearing recovery, dramatic improvements in their speech perception, and a restored ability to conduct normal conversation. The treatment also produced no major side effects in the children, researchers said. Researchers next plan to expand the trial to a larger sample size and continue tracking treated kids to see how well they do.
Deafness Gain
“Not since cochlear implants were invented 60 years ago has there been an effective treatment for deafness,” Chen said in a hospital news release. “This is a huge milestone that symbolizes a new era in the fight against all types of hearing loss.” The new study was published on January 24 in the Lancet journal. Researchers from both the United States and China plan to present their findings early next month at the annual meeting of the Association for Research in Otolaryngology.
Deafness Gain
images source: Google
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