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  • RESEARCH HIGHLIGHT

A novel approach to reverse proteinopathies

  1. Grant Otto
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Autosomal dominant tubulointerstitial kidney disease-MUC1 (ADTKD-MUC1) is a slowly progressive disease, for which there are no therapies and the pathogenetic mechanism is unknown. Writing in Cell, Dvela-Levitt et al. report that ADTKD-MUC1 is a toxic proteinopathy and identify a small molecule that clears the accumulated toxic protein by modifying its intracellular trafficking.

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Nature Reviews Drug Discovery 18, 665 (2019)

doi: https://doi.org/10.1038/d41573-019-00133-5

References

  1. Dvela-Levitt, M. et al. Small molecule targets TMED9 and promotes lysosomal degradation to reverse proteinopathy. Cell 178, 1–15 (2019)

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