Challenging corneal diseases and microRNA expression: focus on rare diseases and new therapeutic frontiers

MicroRNAs (miRNAs) function as posttranscriptional regulators of gene expression by targeting specific messenger RNA (mRNA). This interaction modulates mRNA stability or translational efficiency, ultimately impacting the level of protein production. Emerging evidence suggests that miRNAs act as critical regulators in corneal diseases. These molecules finetune key processes like cell proliferation, differentiation, inflammation, and wound healing. We reviewed the literature to understand the role that miRNAs may play in the development of challenging and poorly understood corneal diseases. We focused on vernal keratoconjunctivitis, neurotrophic keratitis, keratoconus, Fuchs endothelial corneal dystrophy, and limbal stem cell deficiency. Furthermore, we explored currently studied agonists or antagonists of miRNAs that share similar pathways with ocular diseases and could be employed in ophthalmology in the future. The distinct miRNA expression profiles observed in different ocular surface pathologies, combined with the remarkable stability and relatively easy access of miRNA sampling in biofluids, present possibilities for the development of noninvasive and highly accurate diagnostic tools. Furthermore, comprehending miRNA's pathophysiological role could open new frontiers to a more comprehensive understanding of the pathophysiology underlying ocular surface diseases, thereby paving the way for the creation of novel therapeutic strategies.