Catalog No.: PC-73087Not For Human Use, Lab Use Only.
BRD4780 is a small molecule that releases MUC1-fs from TMED9-enriched compartments and promotes its anterograde trafficking to the lysosome, directly binds to TMED9.
BRD4780 is a small molecule that releases MUC1-fs from TMED9-enriched compartments and promotes its anterograde trafficking to the lysosome, directly binds to TMED9.
BRD4780 selectively reduces mutant (EC50=143 nM) but not wild-type MUC1.
BRD4780 phenocopied the effect of TMED9 deletion.
BRD4780 directly binds to TMED9, BRD4780 dose-dependently (EC50 = 143 nM) removes of mutant MUC1-fs without decreasing wild-type MUC1.
BRD4780 showed no effect on MUC1 transcriptional regulation.
BRD4780 rescued P cells from THP-induced cell death, and significantly reduced UPR activation.
BRD4780 removes mutant MUC1-fs from kidneys of heterozygous knockin mice and patient iPSC-derived kidney organoids.
BRD4780 is effective in removing other misfolded proteins (mutant C126R UMOD protein, rhodopsin mutation P23H), but not the mutant version of huntingtin (97 polyQ).
