Creating a new gene therapy for hereditary deafness

New studies offer new treatment for deafness in which genetic material is delivered to inner ear cells. Healthy genetic material replaces the genetic defect, and the cells return to their normal function. In this way, the scientists were able to prevent the gradual hearing loss in mice that had a genetic mutation in deafness. They claim that the new treatment could lead to advances in the treatment of children with mutations that eventually lead to deafness.

Deafness is the most common sensory disability worldwide. According to the World Health Organization, there are currently about half a billion deaf people worldwide, and that number is projected to double in the coming decades. One in every two hundred children is born with a hearing impairment, and one in every 1,000 children is born deaf. About half of these cases of deafness are caused by genetic mutations. So far, about a hundred different genes have been identified that are linked to inherited deafness. Professor Karen Orham, Says the authors of the study:

In this study, we focused on genetic deafness caused by a mutation in the SYNE4 gene, which is a rare deafness that was identified several years ago in two families in the country and has since been identified in Turkey and the United Kingdom. Children who inherit a defective gene from both parents are born with normal hearing; But they gradually lose their hearing as children. The mutation causes the nucleus accumbens of the inner cochlear hair cells to not be in position. These hair cells act as receptors for sound waves and are essential for hearing. The defect caused by the mutation causes the destruction and eventual death of hair cells.

City of Tiberias, One of the study researchers, believes:

We used a new gene therapy technology to create a harmless synthetic virus and use it to deliver genetic material. The virus was a natural version of a gene that is defective in mice and humans. We injected the virus into the inner ear of mice; It entered the hair cells and released its genetic load. By doing this, we eliminated the defect in the hair cells and gave them the ability to mature and function normally.

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Treatment was performed immediately after birth and the mice’s hearing was monitored using behavioral and physiological tests. Professor Jeffrey Holt“These findings are very promising,” said Boston Children’s Hospital, Harvard Medical School and a study fellow. “The mice benefited from normal hearing, and their hearing sensitivity was almost similar to that of healthy non-mutant mice.”

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New study scientists are working on similar treatments for other mutations that cause deafness. Professor Wade China, From Johns Hopkins School of Medicine, adds:

This is an important study that shows that inner ear gene therapy can be used effectively in a model of SYNE4 deaf mice and save their hearing. The degree of hearing improvement is important. The present study is one of many studies showing that gene therapy can be used successfully in mice with inherited deafness and demonstrates the enormous potential of gene therapy as a treatment for deafness.

The results of this research in the journal EMBO Molecular Medicine It’s been published.

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