Gene therapy for visual function recovery

Abstract

Glaucoma is an age-related neurodegenerative disease and the leading cause of blindness, but currently no fundamental treatment has been present. The main treatment is to reduce intraocular pressure, which is expected to delay the progression of the disease. However, there are many glaucoma patients for whom progression cannot be controlled by lowering intraocular pressure alone, and the development of a fundamental treatment is required. Meanwhile, the clinical application of gene therapy is increasing worldwide. Various gene therapy vectors are still being developed, and technological change is much faster in this field. Gene therapy has already been clinically applied to several neurodegenerative diseases, but gene therapy for glaucoma has not yet been established. Our group is investigating the development of a new treatment for glaucoma by gene therapy using neurotrophic factor signaling. And we aim not only to suppress disease progression by neuroprotection, but also to recover the visual function by axonal regeneration.