Innovative Gene Therapy Protects Hearing and Balance in Preclinical Studies

Researchers from Tel Aviv University's Gray Faculty of Medical & Health Sciences have developed a novel gene therapy approach that effectively safeguards both hearing and balance functions in models of inner ear dysfunction. This cutting-edge strategy surpasses previous methods in efficiency and offers hope for treating various genetic mutations responsible for sensorineural hearing loss. The study, led by Professor Karen Avraham and Ph.D. candidate Roni Hahn, involved collaboration with experts from Boston Children's Hospital and Harvard Medical School. Published in the journal EMBO Molecular Medicine, the research focused on targeting mutations in the CLIC5 gene, essential for maintaining hair cell integrity in the inner ear. Deficiencies in this gene lead to progressive hearing loss and balance issues due to hair cell degeneration. The team employed an advanced form of adeno-associated viral vectors, called self-complementary AAV (scAAV), which demonstrated faster and more effective transduction of hair cells at lower doses compared to traditional vectors. In animal models, this gene therapy prevented hair cell deterioration, maintaining normal auditory and vestibular functions. Prof. Avraham highlighted that this innovative approach enhances therapeutic outcomes and could potentially pave the way for treatments addressing a wide spectrum of genetic ear disorders, offering new hope for patients affected by hereditary hearing and balance impairments.