Future studies using CECs from affected individuals will be helpful in defining an effective ASO concentration, which can lead to a good clinical dose estimate when considering introduction to the anterior ocular chamber, which has a small fixed but rapidly exchanging volume. Detailed pharmacokinetic studies will be required to define the optimal dosing time interval, which is expected to be months, based on the rapid cellular uptake and the tissue/cellular longevity of similar 2′Ome-PS-ASOs in studies that have examined intraocular dosing of ASOs.48 It is important to note that in the final stages of FECD there is significant endothelial cell loss, and consequently ASO-specific treatment is intended to prevent further cell loss. The most likely group of affected individuals to benefit from such a therapeutic intervention will be those individuals who are in the early stages of disease and have not yet experienced significant endothelial cell loss. Importantly, these at-risk individuals can be effectively identified by a CTG18.1 genotyping test.