GeneticsJCI InsightPeer-reviewed
Therapeutic splice modulation of COL4A5 reinstates collagen IV assembly in an organoid model of X-linked Alport syndrome
Hassan Saei, Bruno Estebe, Nicolas Goudin…·Guillaume Dorval
The paper demonstrates that ASO treatment restores 5(IV) collagen in XLAS patient-derived kidney organoids, supporting therapeutic potential for splicing modulation.
5 citations