Gene therapy improves hearing in people with congenital hearing loss or severe hearing impairments within one to several months, with tolerable side effects
Research conducted by the Karolinska Institute in Sweden and elsewhere has shown that gene therapy can be effective in improving the hearing of people with congenital hearing loss or severe hearing impairment. Positive effects have already been reported in treatments for pre-teen children, but this study is the first to target young people in their teens and twenties.
AAV gene therapy for autosomal recessive deafness 9: a single-arm trial | Nature Medicine
Gene therapy restored hearing in deaf patients | Karolinska Institutet
The research was conducted by the Karolinska Institutet , a medical university in Sweden, in collaboration with the Zhongda Hospital affiliated to Southeast University in China and funded by Ovid Therapeutics , a company specializing in genetic medicine. The paper was published in the academic journal Nature Medicine.
The study subjects were 10 patients aged between 1 and 24 years old who were hospitalized at the China University Hospital attached to Southeast University and other institutions. All 10 patients had hereditary hearing loss or severe hearing impairment caused by a mutation in the gene 'OTOF' that causes hearing loss, resulting in a deficiency of the protein otoferin, which plays an important role in transmitting auditory signals from the ear to the brain.
Dr. Maori Duan of the Karolinska Institutet and his colleagues used a synthetic adeno-associated virus (AAV) to deliver a single injection of a functional OTOF gene into the inner ear through a membrane at the base of the cochlea called the round window.
The majority of patients recovered some degree of hearing within one month of the injection, with the effects being particularly pronounced in younger patients aged 5 to 8. One 7-year-old girl's hearing was almost completely restored, and she was able to converse normally with her mother four months later.
Injecting the OTOF gene into the inner ear has already been used in children in China with positive results, but this is the first time it has been used to treat teenagers and adults.
Gene therapy successfully restores hearing in children born deaf - GIGAZINE
Dr Duan said further follow-up studies were needed to see how long the effects would last, but a six-month follow-up showed that all participants' hearing had improved significantly, with the average level of perceptible sound improving from 106 decibels to 52 decibels.
Although a side effect of the drug was a decrease in neutrophils, a type of white blood cell, this was within the acceptable range, and no serious side effects were found in a 6- to 12-month follow-up study.
'Treatment for OTOF is still in its infancy, and we are expanding our research to other genes that cause hearing loss, such as GJB2 and TMC1,' said Dr. Duan. 'The treatment will be more complicated, but we have already seen promising results in animal studies. I am confident that one day patients with various types of hereditary hearing loss will be able to receive treatment.'
in Science, Posted by logc_nt