Department of Microbiology and Molecular Genetics, University of Pittsburgh School of Medicine, Pittsburgh, PA 15219, USA.
Department of Genetics and HHMI, Yale School of Medicine, New Haven, CT 06510, USA.
Trends Mol Med. 2019 Oct;25(10):821-823. doi: 10.1016/j.molmed.2019.08.011. Epub 2019 Sep 11.
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.
在 Dvela-Levitt 等人最近的一篇论文中，使用免疫荧光检测的化学筛选发现了一种化合物，该化合物能将突变的黏蛋白 1 从患有进行性髓质囊性肾病患者的永生化肾上皮细胞的细胞内位置移除。这说明了在细胞水平进行化学筛选以解决特定蛋白病变的强大性，以及此类化合物阐明能清除毒性蛋白的新细胞途径的实用性。
