Studies using DM1 cell and animal models have shown that targeting the CTG expansion within DMPK using an antisense oligonucleotide (ASO) approach leads to a reduction in RNA foci and downstream markers of toxicity.23, 24 To determine whether similar strategies could be effective for CTG18.1-associated FECD and to translate FECD therapies into the clinic, appropriate FECD disease models are essential. Given the lack of animal models for FECD, coupled with the general poor association of animal model phenotypes with human complex disease, corneal endothelial cell (CEC) cultures derived from affected individuals offer an ideal opportunity to probe disease mechanism and investigate therapeutic approaches.