Drug design from the cryptic inhibitor envelope.

Abstract

Conformational dynamics plays an important role in enzyme catalysis, allosteric regulation of protein functions and assembly of macromolecular complexes. Despite these well-established roles, such information has yet to be exploited for drug design. Here we show by nuclear magnetic resonance spectroscopy that inhibitors of LpxC--an essential enzyme of the lipid A biosynthetic pathway in Gram-negative bacteria and a validated novel antibiotic target--access alternative, minor population states in solution in addition to the ligand conformation observed in crystal structures. These conformations collectively delineate an inhibitor envelope that is invisible to crystallography, but is dynamically accessible by small molecules in solution. Drug design exploiting such a hidden inhibitor envelope has led to the development of potent antibiotics with inhibition constants in the single-digit picomolar range. The principle of the cryptic inhibitor envelope approach may be broadly applicable to other lead optimization campaigns to yield improved therapeutics.

Department

Description

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Citation

Published Version (Please cite this version)

10.1038/ncomms10638

Publication Info

Lee, Chul-Jin, Xiaofei Liang, Qinglin Wu, Javaria Najeeb, Jinshi Zhao, Ramesh Gopalaswamy, Marie Titecat, Florent Sebbane, et al. (2016). Drug design from the cryptic inhibitor envelope. Nat Commun, 7. p. 10638. 10.1038/ncomms10638 Retrieved from https://hdl.handle.net/10161/11782.

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Scholars@Duke

Toone

Eric John Toone

Professor Emeritus of Chemistry

Dr. Toone is a physical organic chemist who studies relationships between structure and activity in the context of biology. Currently active programs exist in biocatalysis/applied enzymology, ligand binding and the activity of water, and the synthesis of novel donors of nitric oxide. The study of these problems makes use of synthetic organic chemistry, traditional enzymology, isothermal titration microcalorimetry, and the techniques of directed evolution.

Zhou

Pei Zhou

Professor of Biochemistry

The Zhou lab focuses on the elucidation of the structure and dynamics of protein–protein and protein–ligand interactions and their functions in various cellular processes. Our current efforts are directed at enzymes and protein complexes involved in bacterial membrane biosynthesis, translesion DNA synthesis, co-transcriptional regulation, and host-pathogen interactions. Our investigations of these important cellular machineries have led to the development of novel antibiotics and cancer therapeutics, as well as the establishment of new biotechnology adventures.

 

The Zhou lab integrates a variety of biochemical and biophysical tools, including NMR, X-ray crystallography, cryo-EM, and enzymology. The lab has played a major role in the development and application of innovative NMR technologies, including high-resolution, high-dimensional spectral reconstruction techniques.


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