Experimental treatment in the field of gene therapy allowed to restore hearing in children with congenital deafness
Kyiv • UNN
Advances in gene therapy have helped restore hearing in five out of six children born deaf due to a mutation in the OTOF gene. The successful treatment involved injecting a functioning version of otoferlin, a key protein for hearing recovery, into the inner ear.
A new development in the field of gene therapy has helped restore hearing in five children who were born deaf. The Harvard Gazette writes about this with reference to a scientific article in the Lancet magazine, UNN reports.
Details
The study, conducted in China and led by a team from the Massachusetts Eye and Ear Infirmary and Harvard Medical School, involved six children aged 1 to 7 years who suffered from deafness. All of them had an inherited mutation in the OTOF gene, which produces a protein important for transmitting signals from the ear to the brain.
The article explains that people can hear when sound waves in the air cause thousands of sensitive hair cells in the inner ear to vibrate and release a chemical that transmits this information to the brain. The protein Otoferlin is essential for the release of this chemical messenger because without it, the ear cannot communicate with the brain.
The children were injected with a working version of otoferlin into their inner ears. The inner ear cells read it and began to produce the substances necessary to transmit signals to the brain.
Five of the six children showed improvement in their hearing over the course of the 26-week study, with four of the results described by the researchers as "sustained." Because hearing is a critical factor in language learning, the researchers also measured speech perception-the ability to recognize sound as speech-and all five of those who responded to the treatment showed improvement in this area.
This opens the door to the development of other treatments for different types of genetic deafness
According to Chen, hearing loss affects more than 1.5 billion people worldwide, including about 30 million cases of genetic disorders in children. The participants in the current study suffered from a disease called DFNB9, which leads to complete deafness due to a mutation in the OTOF gene.
