Differential Responses of COL17A1 Nonsense Mutations to Readthrough Drugs and NOG as a Novel Enhancer in Junctional Epidermolysis Bullosa
3 September 2026 at 08:00
COL17A1 nonsense mutations generate premature termination codons, reducing collagen XVII through truncated protein production and/or nonsense-mediated decay (NMD), and causing junctional epidermolysis bullosa. Translational readthrough drug offers a potential approach to nonsense mutations suppression. Different COL17A1 nonsense mutations showed distinct responses to readthrough drugs and NMD inhibitors, supporting personalized therapeutic strategies.