A new study from Tel Aviv University (TAU) introduces an innovative treatment for deafness, based on the delivery of genetic material into the cells of the inner ear. The genetic material “replaces” the genetic defect and allows the cells to continue to function as normal.
The scientists were able to prevent a gradual decline in mice that had a genetic mutation for deafness. They argue that this novel treatment could lead to advances in the treatment of children born with various mutations that cause deafness.
The study was led by Professor Karen Avraham from the Department of Human Molecular Genetics and Biochemistry at the TAU Faculty of Medical Sciences and the Sagol School of Neuroscience. The paper was published in EMBO Molecular Medicine on 22 December 2020.
Deafness is the most common sensory disability in the world. According to the World Health Organization, there are approximately half a billion people with hearing loss worldwide today, and this figure is expected to double in the coming decades. One in 200 babies is born deaf, and one in 1,000 is born deaf. In about half of these cases, deafness causes genetic mutation. There are currently around 100 different species associated with hereditary deafness.
“In this study we focused on genetic deafness caused by a mutation in the SYNE4 gene – a rare deafness that our laboratory discovered several years ago in two Israeli families, and has since been identified in Turkey and in the UK too, “Dr Avraham reports.” Children who inherit the deficiency gene from both parents are born with normal hearing, but gradually lose their hearing during childhood . The mutation causes cell nucleus dysfunction in the hair cells inside the inner ear cochlea, which are acoustic receptors and are essential for hearing. This defect leads to the decay and eventual death of hair cells. “
“We applied innovative gene therapy technology: we created a harmless synthetic virus and used it to deliver genetic material – a standard version of the gene that is deficient in the mouse model and in the affected human families, “said Shahar Taiber, one of Professor Avraham’s students on the combined MD-PhD route.” We transmitted the virus to the inner ear of the mice, until it entered the hair cells and that he dropped his genetic paycheck. In doing so, we repaired the deficiency in the hair cells and allowed them to mature and function normally. “
The treatment was given shortly after birth and then mice were listened to using both psychological and behavioral tests. “The results are promising,” said Dr. Jeffrey Holt of Boston Children’s Hospital and Harvard Medical School, a colleague of the study. “Improve mice with normal hearing management, with almost the same sensitivity as healthy mice that do not have the mobility.”
The scientists are now developing similar treatments for other mutations that cause deafness.
This is an important study that demonstrates that inner ear gene therapy can be effectively applied in a mouse model of SYNE4 deafness to save hearing. The amount of audibility is amazing. This study is part of a growing body of literature demonstrating that gene therapy can be successfully applied in mouse models of hereditary hearing loss, and demonstrates the great potential of gene therapy as a treatment for deafness. . “
Professor Wade Chien, MD, NIDCD / NIH Eastern Gene Therapy Program and Johns Hopkins School of Medicine
Source:
Friends of America Tel Aviv University