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

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.

journal_name

Trends Mol Med

authors

Thomas G,Horwich A

doi

10.1016/j.molmed.2019.08.011

subject

Has Abstract

pub_date

2019-10-01 00:00:00

pages

821-823

issue

10

eissn

1471-4914

issn

1471-499X

pii

S1471-4914(19)30234-5

journal_volume

25

pub_type

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