Clitocine is a Potent Readthrough Drug in Laminin-332 Deficient Junctional Epidermolysis Bullosa (ESDR 2026) - Sep 9, 2026 - Abstract #419; Pres time: Sep 10, 2026; 05:00 PM - 06:00 PM; Location: Forum 4; "The readthrough activity of artesunat, atazanavir, erythromycin, clitocine, gentamicin and TRID-C5 (gentamicin, paromomycin, CC-90009, apocynin and melatonin) was evaluated, and DMSO as a vehicle control. Gene expression studies showed clitocine restores the expression of a network of genes coding for extracellular matrix proteins associated with laminin-332 in JEB-LAM3 keratinocytes, but it has a broad off-target effect in particular in NHK. Overall, clitocine showed the most consistent readthrough activity across different mutation LAMB3 types, supporting its further evaluation as a candidate for topical translational readthrough therapy in JEB-LAMB3."
Dermatology
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Rui Zhang; Haifa El Mabrouk; Cristina Has; Saliha Beysa Sayar
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