Exploring AAV‐Mediated Gene Therapy for Inner Ear Diseases: from Preclinical Success to Clinical Potential

Abstract

Hearing loss imposes a significant global health burden and often results from genetic factors and various external influences, such as noise exposure and the use of ototoxic drugs. Recent advancements in adeno‐associated virus (AAV)‐mediated gene therapy offer promising new avenues for the treatment of inner ear diseases. Clinical trials of AAV‐mediated gene therapies show impressive preliminary results, although further research is needed to evaluate the safety and long‐term effects of these therapies. Preclinical AAV‐mediated gene therapy is notable for its high transduction efficiency and safety. In this article, the development of AAV‐mediated gene therapies is reviewed for inner ear diseases, categorizing these therapies by their strategies for treating hereditary hearing loss, including gene replacement and cluster regularly interspaced short palindromic repeat‐based methods. AAV‐mediated hair cell regeneration therapy is also reviewed for acquired hearing loss, as well as methods to prevent acquired hearing loss. In this article, it is hoped to provide a comprehensive overview of recent progress in AAV‐mediated gene therapy and its future potential, thereby highlighting the importance of this therapy for researchers and clinicians in the field.