A lipid-modulating protein that exemplifies the value of human genetics for target validation has provided a fertile testing ground for new drug modalities including long-acting RNA interference drugs, vaccines against self-antigens, CRISPR therapeutics and small molecules that control ribosomal activity.
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Mullard, A. Nine paths to PCSK9 inhibition. Nat Rev Drug Discov 16, 299–301 (2017). https://doi.org/10.1038/nrd.2017.83
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DOI: https://doi.org/10.1038/nrd.2017.83
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