Pharmacological rescue of cilia trafficking defects in IFT140 retinal organoid and RPE models of retinal dystrophy
Julio C. Corral-Serrano, Yi Jiang, Nele Schwarz, Sara Eliza Nieuwenhuis, Kelly Ziaka, Siobhan E. Guilfoyle, Rosellina Guarascio, Adriana Bakoulina, Marian Seda, Jeshmi Jeyabalan, Diego Ottaviani, Esben Lorentzen, Isabelle Perrault, Alison J. Hardcastle, Tina Beyer, , Michael E. Cheetham
This study uses human stem cell models to investigate IFT140 variants causing retinal dystrophy and identifies eupatilin as a potential therapeutic for cilia defects.
bioRxiv · May 3, 2026 · PreprintNeuroscience