Gene therapy: New and future treatments for uveitis

Abstract Uveitis, characterized by intraocular inflammation, has significant management challenges due to its diverse etiologies and complicated pathophysiology. The current first‐line treatments primarily aim to calm inflammation with the underlying causes unaffected, often associated with systemic side effects, limited long‐term efficacy, and disease recurrence. Gene therapies, as powerful therapeutic approaches, have been applied to treat various genetic and non‐genetic diseases. However, the development of gene therapy for uveitis has been investigated less. This review discusses the possible targets and therapeutic approaches for uveitis gene therapy by analyzing some research examples in exogenous gene expression, RNAi, antisense therapy, and the CRISPR gene editing system. Furthermore, we discuss the limitations of listed gene therapies for uveitis and propose future research directions and potential strategies to overcome current challenges.