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Hearing loss is the most common sensory disability worldwide, and more than half of cases present at birth are caused by genetic mutations.
By Shula Rosen
Scientists at Tel Aviv University have developed an experimental gene therapy that preserved both hearing and balance in lab studies, raising hopes for new treatments for people born with genetic ear disorders.
The research, published in EMBO Molecular Medicine, was led by Prof. Karen Avraham, dean of the Gray Faculty of Medical and Health Sciences, together with doctoral student Roni Hahn.
The project was carried out with Boston Children’s Hospital and Harvard Medical School, with support from several international science foundations.
Hearing loss is the most common sensory disability worldwide, and more than half of cases present at birth are caused by genetic mutations.
The team focused on changes in a gene called CLIC5, which normally protects the delicate hair cells in the inner ear that detect sound and control balance.
When this gene malfunctions, those cells gradually die, leading to deafness and unsteadiness.
To counter this process, the scientists used harmless viruses to deliver a working copy of the CLIC5 gene directly into ear cells.
They relied on a next-generation viral carrier known as self-complementary AAV, designed to deliver genes faster and more effectively than earlier versions.
In tests on mice missing the CLIC5 gene, the treatment preserved the structure of the inner ear’s hair cells and kept them functioning.
Animals that received the therapy performed better in hearing and balance tests compared with untreated mice.
The researchers said the results suggest that gene therapy could be customized not only to restore hearing but also to protect balance, a critical function often overlooked in ear disorders.
While human trials are still a way off, the team noted that advances in viral delivery systems make gene therapy safer and more efficient, requiring lower doses to work.
They believe this discovery could lead to future treatments for a range of hereditary conditions that affect the inner ear.
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