Precise gene therapy for deafness: Targeted gene delivery and controlled expression

Guisheng Zhong, PhD, Associate Professor

Dr. Zhong‘s lab is dedicated to tackling the profound challenge of permanent hearing loss, a condition typically irreversible because mammals cannot naturally regenerate the sensory hair cells (HCs) essential for sound perception. The lab integrates deep fundamental research—discovering the molecular blueprints needed to reprogram non-sensory cells into functional HCs—with the development of advanced translational tools. This dual focus creates a clear path from discovery to cure. 

Dr. Zhong's lab has engineered critical technologies to realize this vision, including the innovative AAV vectors optimized for safe and efficient delivery into the inner ear, and the innovative ARBITER system. This system functions as a high-precision molecular platform, identifying and fine-tuning gene "switches" to ensure therapeutic gene expression is tightly restricted to the correct cell populations. The research progress has led to remarkable clinical success: their gene therapy approach, formalized as the EA0010 ear injection solution, has successfully restored natural hearing in  OTOF patients. Dr. Zhong's lab aims to restore natural hearing to deaf patients caused by both genetic and environmental factors.