Cysteamine-bicalutamide combination treatment restores alpha-ketoglutarate and corrects proximal tubule phenotype in cystinosis
A. Jamalpoor, C. A. van Gelder, F. A. Yousef Yengej, E. A. Zaal, S. P. Berlingerio, K. R. Veys, C. Pou Casellas, K. Voskuil, K. Essa, C. M. Ammerlaan, L. R. Rega, R. E. N. van der Welle, M. R. Lilien, M. B. Rookmaaker, H. Clevers, , M. J. Janssen
This study identifies alpha-ketoglutarate as a key metabolite in cystinosis and shows that combining cysteamine with bicalutamide corrects proximal tubule defects in cell models and zebrafish.
bioRxiv · Feb 10, 2020 · PreprintPharmacology & Toxicology