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- W4367017626 abstract "A single shot of gene therapy lowers “bad” cholesterol by about 60% for at least 8 months in monkeys, a new study reports ( Nature 2021, DOI: 10.1038/s41586-021-03534-y ). The treatment inactivates a gene called PCSK9 , which stands for proprotein convertase subtilisin/kexin type 9. PCSK9 proteins typically bind receptors that break down artery-clogging low-density lipoprotein (LDL) cholesterol. Without functioning PCSK9, cells have more LDL receptors available to grab and remove LDL from the blood. Doctors currently treat high cholesterol with statins, as well as antibodies to the PCSK9 protein, which people must take daily or weekly. A long-term treatment would improve patient adherence to therapies, the researchers write. The gene therapy would be particularly relevant for people with a rare condition, familial hypercholesterolemia, which is caused by mutations in PCSK9 and leads to consistently high levels of cholesterol. “This is a landmark study, with important implications for a one-time" @default.
- W4367017626 created "2023-04-27" @default.
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- W4367017626 date "2021-05-24" @default.
- W4367017626 modified "2023-09-23" @default.
- W4367017626 title "Single CRISPR dose lowers cholesterol in monkeys" @default.
- W4367017626 doi "https://doi.org/10.1021/cen-09919-leadcon" @default.
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