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Am J Pharmacogenomics. 2004;4(5):313-20.

Chemical genomics: probing protein function using small molecules.

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  • 1The Eli and Edythe L. Broad Institute, Massachusetts Institute of Technology & Harvard University, Institute of Chemistry & Cell Biology (ICCB), Initiative for Chemical Genetics, Cambridge, Massachusetts, USA.


Chemical genomics is concerned with the effects of both genetic variation and chemical perturbation on the cellular effects of small molecules. Chemical genomics relies on selecting biological networks for study, such as those represented by different cell types or disease models, in order to build the desired specificity into the experimental design. The most relevant network property for such experiments is the global connectivity of all cellular proteins comprising the functional ensemble, as illustrated by case studies of the evolution of cyclooxygenase inhibitors and heat-shock protein modulators. Recent examples of chemical genomic profiling, particularly of different cell types, highlight the power of carefully planned experimental approaches in chemical genomics. These new approaches demonstrate the use of the genome to find new targets or new modes of biological interaction.

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