Gene therapy: scientists have found a way to restore hearing in children with congenital deafness
[ad_1]
Scientists say that hereditary otoferlin deafness can be cured by gene replacement.
Otoferlin deafness is an extremely rare form of the disease caused by a mutation in the otoferlin gene (a protein that helps nerve cells convert sound vibrations into signals that the brain can interpret) in one gene, Live Science writes.
The new technique is to replace the diseased gene with a healthy one. Genetic mutations cause from 1% to 8% of cases of congenital deafness and are quite rare – only 200 thousand people in the world suffer from otoferlin deafness.
In gene therapy, scientists use modified viruses to “deliver” working genes to the inner ear. The results of the study, which was published in the journal The Lancet, indicate that this method of treatment is effective for most patients, but a number of clinical trials are needed to fully approve the therapy.
| Scientists claim that hereditary deafness can be cured by replacing genes. Photo: InsideCreativeHouse/Depositphotos |
“The results of this study are really amazing. We saw that the children’s hearing improved week after week.” said Zheng-Yi Chen, a scientist in the Eaton-Peabody Laboratory at Mass Eye and Ear and an associate professor at Harvard Medical School.
Gene therapy has already been tested. Six children aged one to six took part in the test. Doctors diagnosed each of them with complete hearing loss.
During the trial, each child was injected with modified genes into one ear through the membrane that separates the middle ear from the inner ear. Doctors expected that five children’s hearing would be restored within 26 weeks. Their expectations were met: hearing improved about four to six weeks after treatment, along with speech.
Three out of five children had implants in untreated ears. They help improve hearing by actually bypassing the inner ear, giving sound another path to the brain. Thanks to it, the child can hear, but not so well.
The researchers note that after the use of gene therapy, these children began to understand language better, as well as communicate without implants.
This method of treatment did not cause serious side effects, but the children temporarily experienced fever and changes in the number of leukocytes in the blood. However, in order to patent such a treatment method, a number of additional studies should be conducted to find out what dose of the modified virus should be administered to be safe.
At the same time, the first successes are already there – 11-year-old boy Issam Dam became the first child to undergo treatment in the United States, reports The New York Times. Four months have passed since the beginning of therapy. Now he has only a mild form of deafness.
 |
| The Children’s Hospital of Philadelphia recently performed America’s first gene therapy procedure to treat hereditary hearing loss. Photo: Children’s Hospital of Philadelphia |
Previously, we wrote about how often hearing should be checked.
Read also: Can headphones make your hearing worse and how not to listen to music
Vera Shurmakevich, “UP. Life”
[ad_2]
Original Source Link
