Retinal degenerations are a group of neurodegenerative diseases that afflict millions and cause irreversible blindness. By 2040, Age-Related Macular Degeneration (AMD; the number one cause of blindness in the western world) is projected to affect a staggering 280 million people worldwide. The major challenge of delivering safe and effective treatments to the retina is complex due to its immune-privileged nature and the risk of further damage from immune activation.
Our group is pioneering the use of extracellular vesicles (EV) for the treatment of retinal degenerations, and harnessing their molecular cargo, including microRNA (miRNA). Our findings show that EV isolated from various sources, including mesenchymal stem cells and red blood cells, have different abilities to deliver therapeutic payloads. Specifically, we have demonstrated that red blood cell EV delivered via intraocular injection offer significant protection against retinal degeneration and have the potential to enhance RNA therapy delivery. Further we propose that loading of specific miRNA cargo could improve the basal therapeutic potential of EV.
Overall, EV-based treatments offer a promising avenue for safe and effective therapies for retinal degeneration. While further research is needed to fully understand the mechanisms, optimize therapeutic potential and cell targeting, our findings provide compelling evidence that EV are a viable option for the development of new therapies to the retina especially for the currently incurable AMD.