Therapeutic splice modulation of COL4A5 reinstates collagen IV assembly in an organoid model of X-linked Alport syndrome
Hassan Saei, Bruno Estebe, Nicolas Goudin, Mahsa Esmailpour, Julie Haure, Olivier Gribouval, Christelle Arrondel, Vincent Morinière, Pinyuan Tian, Rachel Lennon, Corinne Antignac, Géraldine Mollet, Guillaume Dorval
The paper demonstrates that ASO treatment restores 5(IV) collagen in XLAS patient-derived kidney organoids, supporting therapeutic potential for splicing modulation.
JCI Insight · Dec 18, 2025Peer-reviewedGenetics