The discovery of a compound that interrupts the production of a specific protein could open up a fresh path to drug discovery.
Cells rely on complex molecular machines called ribosomes to translate the genetic code and make proteins. Robert Dullea at Pfizer Worldwide Research and Development in Cambridge, Massachusetts, Jamie Cate at the University of California, Berkeley, and their colleagues studied a compound that causes ribosomes to stall while producing the protein PCSK9, which slows the removal of cholesterol from the blood. They found that the compound temporarily stops ribosomes from synthesizing PCSK9 when they encounter a specific protein sequence, and affects the production of only a few other proteins.
Feeding the compound to rats reduced their cholesterol levels. Such ribosome-stalling compounds could be used to inhibit proteins that are difficult to target with other kinds of drugs, the authors say.
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Drug stalls protein translation. Nature 543, 592 (2017). https://doi.org/10.1038/543592c
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DOI: https://doi.org/10.1038/543592c
