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Gene therapy currently being tested restores hearing in 5 out of 6 deaf children
Researchers said a gene therapy developed by a Chinese company restored hearing in children with congenital deafness, adding to growing evidence of the effectiveness of such treatments.
According to a report published in the magazine “The lancet“Five out of six young children suffering from severe deafness experienced improvement in their sense of hearing as well as improvement in speech recognition after six months of treatment with the treatment developed by RefreshingGen Therapeutics.”
All children suffered from severe deafness caused by mutations in the otoferlin gene. The protein otoferlin is essential for the transmission of sound signals from the ear to the brain.
Previous research suggests that mutations in this gene are responsible for two to three percent of cases of congenital deafness. One in every 1,000 children in the United States suffers from moderate to severe hearing loss.
Side effects
During surgical procedures at Fudan University Eye, Nose, Ear and Throat Hospital, researchers used a harmless virus to deliver a copy of the human gene into patients’ inner ears.
After 26 weeks, they announced that five of the six children showed signs of improved hearing, as well as significant improvements in speech perception and the ability to hold conversations.
They added that the side effects are mostly minor and none of them have a long-term effect.
Researchers are not sure why the sixth child did not respond to treatment.
One possible explanation is that some of the gene therapy solution leaked from the inner ear during or after surgery.
“For the first time in his life,” a Moroccan boy could be heard saying after undergoing a new gene therapy in the United States.
In the same context, an 11-year-old boy was able to hear “for the first time in his life” after a new genetic treatment, a hospital in the American city of Philadelphia said. For example, the child who grew up in Morocco learned to recognize the roar of cars, his father’s voice and the sound of hair clippers.
The Children’s Hospital of Philadelphia (CHOP), which performed the treatment, said in a statement that this success, the first of its kind in the United States, gives hope to patients around the world suffering from hearing loss caused by genetic mutations Suffer. Issam Dam suffers from “severe deafness” due to a very rare defect in a gene.
“Gene therapy for hearing loss is a goal that physicians and scientists specializing in hearing loss have pursued for more than 20 years,” said surgeon John Germiller, director of clinical research in the hospital’s Department of Otolaryngology. “We finally did it,” he added.
He explained in the statement that the gene therapy used on the patient “is aimed at correcting a very rare genetic defect, but these studies could pave the way for the future use of more than 150 other genes that are used in children lead to hearing loss.”
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