On Sept. 14, 2025, scientists from the Gray Faculty of Medical & Health Sciences at Tel Aviv University introduced an innovative gene therapy method to treat impairments in hearing and balance caused by inner ear dysfunction.

According to the researchers, “This treatment constitutes an improvement over existing strategies, demonstrating enhanced efficiency and holds promise for treating a wide range of mutations that cause hearing loss.”

In this study, the researchers investigated a mutation in the CLIC5 gene, which is essential for maintaining the stability and function of hair cells in the auditory and vestibular systems. Deficiency of this gene causes progressive degeneration of hair cells, initially leading to hearing loss and later resulting in balance problems.

The researchers utilized an advanced, structurally optimized version of the AAV vector, the self-complementary AAV (scAAV). They found that this vector achieved faster and more efficient transduction of hair cells compared to traditional AAV methods, requiring a lower dose to achieve a similar therapeutic effect. In treated animal models, this approach prevented hair cell degeneration and preserved normal hearing and balance.

The study was published in the journal EMBO Molecular Medicine. The findings mark a significant step toward developing gene therapies for inherited hearing disorders, and may also inform treatments for other sensory and neurological conditions.