Chemical Strike against a Dominant-Inherited MUC1-Frameshifted Protein Associated with Progressive Kidney Disease.

Chemical Strike against a Dominant-Inherited MUC1-Frameshifted Protein Associated with Progressive Kidney Disease.
Trends Mol Med. 2019 Sep 11;:
Authors: Thomas G, Horwich A
Abstract
In a recent paper by Dvela-Levitt et al., chemical screening using an immunofluorescent assay identified a compound that caused removal of a dominant-inherited misfolded secretory protein, mucin1-frameshifted, from an intracellular location in immortalized renal epithelial cells of a patient affected with progressive medullary cystic kidney disease. This illustrates the power of chemical screening at the cellular level to address specific proteinopathies and the utility of such compounds to illuminate novel cellular pathways that can clear toxic proteins.
PMID: 31521560 [PubMed - as supplied by publisher]

Source: Trends in Molecular Medicine - September 10, 2019 Category: Molecular Biology Authors: Thomas G, Horwich A Tags: Trends Mol Med Source Type: research