Matches in SemOpenAlex for { <https://semopenalex.org/work/W4366816178> ?p ?o ?g. }
- W4366816178 abstract "Familial hypercholesterolemia (FH) patients suffer from excessively high levels of Low Density Lipoprotein Cholesterol (LDL-C), which can cause severe cardiovascular disease. Statins, bile acid sequestrants, PCSK9 inhibitors, and cholesterol absorption inhibitors are all inefficient at treating FH patients with homozygous LDLR gene mutations (hoFH). Drugs approved for hoFH treatment control lipoprotein production by regulating steady-state Apolipoprotein B (apoB) levels. Unfortunately, these drugs have side effects including accumulation of liver triglycerides, hepatic steatosis, and elevated liver enzyme levels. To identify safer compounds, we used an iPSC-derived hepatocyte platform to screen a structurally representative set of 10,000 small molecules from a proprietary library of 130,000 compounds. The screen revealed molecules that could reduce the secretion of apoB from cultured hepatocytes and from humanized livers in mice. These small molecules are highly effective, do not cause abnormal lipid accumulation, and share a chemical structure that is distinct from any known cholesterol lowering drug." @default.
- W4366816178 created "2023-04-25" @default.
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- W4366816178 date "2023-04-24" @default.
- W4366816178 modified "2023-10-15" @default.
- W4366816178 title "A human iPSC-derived hepatocyte screen identifies compounds that inhibit production of Apolipoprotein B" @default.
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- W4366816178 doi "https://doi.org/10.1038/s42003-023-04739-9" @default.
- W4366816178 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/37095219" @default.
- W4366816178 hasPublicationYear "2023" @default.
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