Gene Therapy for Corneal Diseases

Corneal diseases are a major cause of blindness and visual impairment worldwide. Although conventional pharmacological therapies can alleviate mild to moderate corneal disorders, they often fail to restore vision in advanced disease, leaving corneal transplantation as the current standard treatment. However, donor tissue shortages, graft rejection, and surgical complications continue to limit its long-term success, highlighting the urgent need for effective nonsurgical therapeutic strategies. Gene therapy has emerged as a promising approach for treating inherited and acquired corneal diseases by targeting the underlying molecular mechanisms that drive disease progression. Recent advances in genome-editing technologies, gene-delivery systems, and vector engineering have substantially expanded the clinical potential of gene-based interventions. This review summarizes current progress in gene-editing strategies, gene-delivery platforms, and their therapeutic applications across a broad spectrum of corneal diseases. In addition, we discuss the major challenges and future perspectives associated with delivery efficiency, safety, vector optimization, and clinical translation. Collectively, these advances highlight the potential of gene therapy to transform the management of corneal diseases by providing safe, targeted, and long-lasting therapeutic strategies for restoring corneal function and vision.