A new study from Tel Aviv University presents an innovative treatment for deafness, which is based on the introduction of genetic cargo into the cells of the inner ear. As part of the treatment, the genetic load is inserted into the damaged cell in such a way that it actually "repairs" the genetic defect and allows the cell to continue to function normally
Deafness is the most common sensory impairment. According to the World Health Organization, there are about half a billion hearing impaired people today, and this number is expected to double in the coming decades. One in 200 children is born with a hearing impairment and one in a thousand children is born deaf. In half of the cases the cause of the deafness is a genetic mutation. Currently, the medical literature recognizes about 100 genes in which mutations can cause hearing loss.
A new study from Tel Aviv University presents an innovative treatment for deafness, which is based on the introduction of genetic cargo into the cells of the inner ear. As part of the treatment, the genetic load is inserted into the damaged cell in such a way that it actually "repairs" the genetic defect and allows the cell to continue functioning normally. As part of the study, the researchers were able to prevent the deterioration of the hearing of mice with gradual deafness. According to them, the treatment may lead to a breakthrough in the treatment of children born with a variety of mutations that cause hearing loss.
The leaders of the research are Prof. Keren Avraham and Shahar Tiber, a doctoral student in the combined medicine and research track (MD-PhD), from the Department of Human Molecular Genetics and Biochemistry, at the Sackler School of Medicine and the Segol School of Neuroscience at Tel Aviv University. Also participating in the research were Prof. David Sheprincek from the School of Neurobiology, Biochemistry and Biophysics in the George S. Wise Faculty of Life Sciences of Tel Aviv University, and Prof. Jeffrey Holt from Boston Children's Hospital and Harvard Medical School. The study was published in the journal EMBO Molecular Medicine.
A thought breakthrough
"In this study, we focused on genetic deafness resulting from a mutation in the SYNE4 gene - a rare deafness that was discovered a few years ago in our laboratory among two families in Israel, and has since been diagnosed in Turkey and England." Prof. Avraham explains. "Children who inherit the defective gene from both their parents are born with normal hearing, but their hearing deteriorates during childhood. The reason for this is that the mutation causes a change in the location of the nucleus and the death of the hair cells in the cochlea in the inner ear, which are responsible for receiving sound waves."
"We worked with innovative technology from the field of genetic therapies: we produced an artificial virus, which does not cause disease, and inserted genetic material into it - a normal version of the gene that is damaged in families and mice," says Shahar Tiber. "We injected the virus into the inner ear of the mice, so that the hair cells were infected with the virus and absorbed the genetic material we inserted into it. This "adhesion" actually repairs the damaged cell and allows the hair cells to continue to function properly."
The treatment was given near birth, after which the researchers tested the mice's hearing through physiological and behavioral tests. The treated mice developed normal hearing, almost identical to that of normal mice without the mutation. In light of the success of the research, the researchers are now developing similar treatments for additional mutations that cause deafness.
Prof. Benny Negris, Director of the Department of Otorhinolaryngology at the Meir Medical Center and head of the Department of Otorhinolaryngology at the Faculty of Medicine at Tel Aviv University, who was not involved in the project, responded to the study: "Unlike the conventional treatments for improving hearing today, such as hearing aids And the cochlear implant, which is given after the damage has already occurred and achieves only a partial improvement, Prof. Avraham presents an innovative and effective method for treating hearing impairments even before the damage is caused. The therapeutic approach presented in the study is a breakthrough in thought, a 'equal breaker' in the treatment of deafness."
The research was mainly supported by the Israel-US Binational Science Foundation (BSF), the National Institutes of Health (NIH), the European Research Council (ERC), and the National Science Foundation's Personalized Medicine Research Program.
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