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Potent Inhibition of Necroptosis by Simultaneously Targeting Multiple Effectors of the Pathway.

Catia L PierottiMaria C TanzerAnnette V JacobsenJoanne M HildebrandJean-Marc GarnierPooja SharmaIsabelle S LucetAngus D CowanWilhelmus J A KerstenMeng-Xiao LuoLung-Yu LiangCheree FitzgibbonSarah E GarnishAnne HempelUeli NachburDavid C S HuangPeter E CzabotarJohn SilkeMark F van DelftJames G MurphyGuillaume L Lessene
Published in: ACS chemical biology (2020)
Necroptosis is an inflammatory form of programmed cell death that has been implicated in various human diseases. Compound 2 is a more potent analogue of the published compound 1 and inhibits necroptosis in human and murine cells at nanomolar concentrations. Several target engagement strategies were employed, including cellular thermal shift assays (CETSA) and diazirine-mediated photoaffinity labeling via a bifunctional photoaffinity probe derived from compound 2. These target engagement studies demonstrate that compound 2 binds to all three necroptotic effector proteins (mixed lineage kinase domain-like protein (MLKL), receptor-interacting serine/threonine protein kinase 1 (RIPK1) and receptor-interacting serine/threonine protein kinase 3 (RIPK3)) at different levels in vitro and in cells. Compound 2 also shows efficacy in vivo in a murine model of systemic inflammatory response syndrome (SIRS).
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