MANF clears mutant uromodulin in human kidney organoids of autosomal dominant tubulointerstitial kidney disease
In summary, we discovered that MANF restores compromised autophagy by blunting ER-calcium efflux in ADTKD organoids (Supplemental Figure 4I). By targeting IP3R1, MANF may emerge as a potent therapy to correct disrupted proteostasis in ADTKD and other protein misfolding diseases. Additionally, our ADTKD organoids provide a human-relevant platform for high-throughput therapeutic screening, including RNA-based therapies.
https://www.jci.org/articles/view/205631