An overview of human pluripotent stem cell applications for the understanding and treatment of blindness

Human pluripotent stem cells (hPSCs) hold great promise for human therapies including for treatment of eye-related disease. Given that hPSCs can differentiate into any cell type of the body, they could potentially be used for cell therapies or as platforms to better understand pathological processes in vitro, subsequently enabling the screening of novel treatments. In this chapter, we review the current progress in the differentiation of hPSCs towards specific lineages of the eye, describe their value as tools for disease modelling and drug screening and examine current trials for cell replacement therapies with a focus on retinal dystrophies and optic neuropathies.