Harnessing extracellular vesicles in optic neuropathies and retinal degeneration

Optic nerve and retinal degenerative diseases are leading causes of permanent blindness worldwide. These diseases arise from pathological processes that impair communication among neurons, glial cells, and blood vessels, ultimately leading to progressive neurodegeneration. Most current clinical treatments are primarily symptomatic and do not address the underlying causes of neurodegeneration; therefore, new and more comprehensive therapeutic strategies are urgently needed. This review systematically summarizes the therapeutic and diagnostic roles of extracellular vesicles in optic nerve and retinal degenerative diseases. This review also discusses the challenges and unresolved issues in their clinical applications. Extracellular vesicles are known to participate in intercellular communication and carry bioactive cargos that can regulate various cellular biological processes. They modulate the degenerative microenvironment and exert beneficial effects in optic nerve and retinal diseases. Molecular markers derived from extracellular vesicles present in biological fluids are also associated with disease progression. These markers reflect distinct cellular states and are being explored for non-invasive diagnosis, as well as for predicting disease progression and therapeutic effects. Furthermore, with advances in extracellular vesicle technology, extracellular vesicles can now be modified through multiple approaches for different therapeutic purposes. As our understanding of extracellular vesicle biology and engineering methods continues to grow, extracellular vesicles are expected to play an increasingly important role in the treatment of degenerative diseases.