PDGFR-α shRNA encoded nanoparticle with epithelial mesenchymal transformation interfering for corneal scarring treatment.

Maintaining the clarity of the cornea is crucial for optimal vision. Corneal scarring (CS), resulting from corneal inflammation, trauma, or surgery, can lead to a reduction in corneal transparency and visual impairment. While corneal transplantation is the primary method for restoring vision, the limited availability of corneal donor presents a significant challenge on a global scale. This study aimed to develop a non-viral gene complex utilizing gene silencing technology to deliver interfering fragments of the platelet-derived growth factor alpha receptor (PDGFR-α) to prevent CS. The hydrophilic segment of polyethylene glycol on the surface of the complex significantly improved its cellular safety as a delivery vehicle. The proton sponge effect of cationic carriers facilitates the escape of the target fragment from lysosomes and enables its entry into the cytoplasm for gene interference. In the TGF-β-induced epithelial-mesenchymal transition (EMT) cell model, it demonstrates remarkable capabilities in inhibiting cell fibrosis and migration. A mouse model was utilized to assess the gene complex's capacity to penetrate into the cornea and inhibit the production of corneal scar. This study highlights the significance of inhibiting the EMT process as a potential strategy for treating fibrosis, and has achieved technical success in intervening in corneal scarring.