Abstract
With collaboration between chemistry, X-ray crystallography, and molecular modeling, we designed and synthesized a series of novel piperazine sulfonamide BACE1 inhibitors. Iterative exploration of the non-prime side and S2' sub-pocket of the enzyme culminated in identification of an analog that potently lowers peripheral Abeta(40) in transgenic mice with a single subcutaneous dose.
2010 Elsevier Ltd. All rights reserved.
Publication types
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Research Support, U.S. Gov't, Non-P.H.S.
MeSH terms
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Alzheimer Disease / drug therapy
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Amyloid Precursor Protein Secretases / antagonists & inhibitors*
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Amyloid Precursor Protein Secretases / metabolism
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Amyloid beta-Peptides / antagonists & inhibitors
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Amyloid beta-Peptides / metabolism
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Animals
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Aspartic Acid Endopeptidases / antagonists & inhibitors*
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Aspartic Acid Endopeptidases / metabolism
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Binding Sites
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Crystallography, X-Ray
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Drug Design
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Mice
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Mice, Transgenic
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Models, Molecular
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Peptide Fragments / antagonists & inhibitors
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Peptide Fragments / metabolism
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Piperazine
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Piperazines / chemistry*
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Protease Inhibitors / chemical synthesis*
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Protease Inhibitors / chemistry
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Protease Inhibitors / therapeutic use
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Structure-Activity Relationship
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Sulfonamides / chemical synthesis
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Sulfonamides / chemistry*
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Sulfonamides / therapeutic use
Substances
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Amyloid beta-Peptides
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Peptide Fragments
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Piperazines
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Protease Inhibitors
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Sulfonamides
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amyloid beta-protein (1-40)
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Piperazine
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Amyloid Precursor Protein Secretases
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Aspartic Acid Endopeptidases
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Bace1 protein, mouse
Associated data
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PDB/3LNK
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PDB/3LPI
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PDB/3LPJ
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PDB/3LPK