Browsing by Author "Wagner, Florence"

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      Small Molecule Targets TMED9 and Promotes Lysosomal Degradation to Reverse Proteinopathy 

      Dvela-Levitt, Moran; Kost-Alimova, Maria; Emani, Maheswarareddy; Kohnert, Eva; Thompson, Rebecca; Sidhom, Eriene-Heidi; Rivadeneira, Ana; Sahakian, Nareh; Roignot, Julie; Papagregoriou, Gregory; Montesinos, Monica S.; Clark, Abbe R.; McKinney, David; Gutierrez, Juan; Roth, Mark; Ronco, Lucienne; Elonga, Esther; Carter, Todd A.; Gnirke, Andreas; Melanson, Michelle; Hartland, Kate; Wieder, Nicolas; Hsu, Jane C.-H.; Deltas, Constantinos; Hughey, Rebecca; Bleyer, Anthony J.; Kmoch, Stanislav; Živná, Martina; Barešova, Veronika; Kota, Savithri; Schlondorff, Johannes; Heiman, Myriam; Alper, Seth L.; Wagner, Florence; Weins, Astrid; Golub, Todd R.; Lander, Eric S.; Greka, Anna (2019)
      Intracellular accumulation of misfolded proteins causes toxic proteinopathies, diseases without targeted therapies. Mucin 1 kidney disease (MKD) results from a frameshift mutation in the MUC1 gene (MUC1-fs). Here, we show ...