Scientists from Tel Aviv University have developed a groundbreaking gene therapy that targets inner ear dysfunction, showing promise for treating hearing and balance impairments caused by genetic mutations.

This innovative approach focuses on the CLIC5 gene, which is essential for hair cell stability in the auditory and vestibular systems, with deficiencies leading to progressive hearing and balance issues.

In animal models, the therapy successfully prevented hair cell degeneration and preserved both hearing and balance, indicating its potential for human application.

The research received support from prominent institutions including the US-Israel Binational Science Foundation, NIH/NIDCD, and the Israel Science Foundation, and was published in EMBO Molecular Medicine.

The therapy employs engineered viral vectors, specifically self-complementary AAV (scAAV), which are already in clinical trials for other genetic conditions, highlighting its translational potential.

Researchers used an advanced form of AAV vectors called self-complementary AAV (scAAV), which enables faster and more efficient gene delivery to hair cells at lower doses.

The optimized scAAV vector delivers the therapeutic gene more effectively than traditional AAVs, reducing the required dosage and enhancing transduction efficiency.

This innovative use of scAAV vectors allowed for rapid and effective gene delivery in animal models, demonstrating significant progress in gene therapy techniques for inner ear conditions.

The study was led by Prof. Karen Avraham and Ph.D. student Roni Hahn, in collaboration with researchers from Harvard Medical School and Boston Children’s Hospital.

The research was featured on the cover of EMBO Molecular Medicine and was supported by multiple scientific organizations, emphasizing its significance in the field.