Mice were protected from hearing loss

A new gene therapy for deafness has been developed

Olga Ivanova, Naked Science

Scientists from Israel have developed an innovative method for the treatment of deafness based on the delivery of genetic material to the cells of the inner ear. So far, it has been tested on mice and concerns only one type of deafness, but in the future, the researchers hope to apply their technique to other types of the disease by testing the method on humans.

The work was published in the journal EMBO Molecular Medicine (Taiber et al., Neonatal AAV gene therapy rescues hearing in a mouse model of SYNE4 deafness).

Deafness is considered the most common sensory disability in the world. According to WHO, today it affects about 466 million people, and in the next decade, as scientists predict, this figure will double. According to statistics, every two hundredth child is born with hearing impairment, and every thousandth is deaf. Sixty percent of cases are caused by genetic factors.

Scientists know more than 120 genes that are associated with hereditary deafness. It is the genetic form that the work of researchers from Tel Aviv University (Israel) is aimed at. And although today experts are making serious efforts to develop genetic and other forms of treatment for the disease, almost all of them, according to the authors of the new study, are limited to sound amplification, and not its complete restoration.

Israeli experts have focused their attention on a mutation in only one gene – SYNE 4, which causes a rare form of deafness. Children who inherit such a defect are born with normal hearing, but lose it as they grow up. The mutation causes the incorrect localization of nuclei in the so-called hair cells located inside the cochlea of the inner ear. These cells serve as sound wave receptors and are necessary for the development of normal hearing. As a result, the defect leads to the death of hair cells and deafness.

To deliver normal genetic material to the tissues, the scientists used AAV9–PHP.B, a newly developed synthetic adenoassociated virus. It was injected into the inner ear of newborn mice with the defective SYNE 4 gene. After that, the researchers monitored the rodents' hearing using physiological and behavioral tests.

Figure from the article by Taiber et al. – VM.

As a result, experts stated that all mice injected with the virus with the corrected gene "recovered": they developed normal hearing with almost the same sensitivity as their healthy counterparts. Already today, scientists have begun to develop similar therapies for other mutations that cause deafness.

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