Functional gamma-secretase inhibitors reduce beta-amyloid peptide levels in brain - PubMed (original) (raw)

doi: 10.1046/j.1471-4159.2001.00012.x.

V John, J P Anderson, L Z Chen, P de Saint Andrieu, L Y Fang, S B Freedman, B Folmer, E Goldbach, E J Holsztynska, K L Hu, K L Johnson-Wood, S L Kennedy, D Kholodenko, J E Knops, L H Latimer, M Lee, Z Liao, I M Lieberburg, R N Motter, L C Mutter, J Nietz, K P Quinn, K L Sacchi, P A Seubert, G M Shopp, E D Thorsett, J S Tung, J Wu, S Yang, C T Yin, D B Schenk, P C May, L D Altstiel, M H Bender, L N Boggs, T C Britton, J C Clemens, D L Czilli, D K Dieckman-McGinty, J J Droste, K S Fuson, B D Gitter, P A Hyslop, E M Johnstone, W Y Li, S P Little, T E Mabry, F D Miller, J E Audia

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Functional gamma-secretase inhibitors reduce beta-amyloid peptide levels in brain

H F Dovey et al. J Neurochem. 2001 Jan.

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Abstract

Converging lines of evidence implicate the beta-amyloid peptide (Ass) as causative in Alzheimer's disease. We describe a novel class of compounds that reduce A beta production by functionally inhibiting gamma-secretase, the activity responsible for the carboxy-terminal cleavage required for A beta production. These molecules are active in both 293 HEK cells and neuronal cultures, and exert their effect upon A beta production without affecting protein secretion, most notably in the secreted forms of the amyloid precursor protein (APP). Oral administration of one of these compounds, N-[N-(3,5-difluorophenacetyl)-L-alanyl]-S-phenylglycine t-butyl ester, to mice transgenic for human APP(V717F) reduces brain levels of Ass in a dose-dependent manner within 3 h. These studies represent the first demonstration of a reduction of brain A beta in vivo. Development of such novel functional gamma-secretase inhibitors will enable a clinical examination of the A beta hypothesis that Ass peptide drives the neuropathology observed in Alzheimer's disease.

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