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Nature Cell Biology 2, 428–434 (1 July 2000) | doi:10.1038/35017062

Transition-state analogue inhibitors of |[ggr]|-secretase bind directly to presenilin-1

William P. Esler , W. Taylor Kimberly , Beth L. Ostaszewski , Thekla S. Diehl , Chad L. Moore , Jui-Yi Tsai , Talat Rahmati , Weiming Xia , Dennis J. Selkoe & Michael S. Wolfe

The |[bgr]|-amyloid precursor protein (|[bgr]|-APP), which is involved in the pathogenesis of Alzheimer|[rsquo]|s disease, and the Notch receptor, which is responsible for critical signalling events during development, both undergo unusual proteolysis within their transmembrane domains by unknown |[ggr]|-secretases. Here we show that an affinity reagent designed to interact with the active site of |[ggr]|-secretase binds directly and specifically to heterodimeric forms of presenilins, polytopic proteins that are mutated in hereditary Alzheimer|[rsquo]|s and are known mediators of |[ggr]|-secretase cleavage of both |[bgr]|-APP and Notch. These results provide evidence that heterodimeric presenilins contain the active site of |[ggr]|-secretase, and validate presenilins as principal targets for the design of drugs to treat and prevent Alzheimer|[rsquo]|s disease.