Cardiac glycosides restore autophagy flux in an iPSC-derived neuronal model of WDR45 deficiency
A. Papandreou, N. Singh, L. Gianfrancesco, D. Budinger, K. Barwick, A. Agrotis, C. Luft, Y. Shao, A.-S. Lenaerts, A. Gregory, S. Y. Jeong, P. Hogarth, S. Hayflick, S. Barral, J. Kriston-Vizi, , R. Ketteler
This study identifies cardiac glycosides that restore autophagy and gene expression in an iPSC model of WDR45 deficiency, suggesting potential therapeutic applications for BPAN.
bioRxiv · Sep 13, 2023 · PreprintCell Biology