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Chemical genetics: Ligand-based discovery of gene function

Journal

NATURE REVIEWS GENETICS
Volume 1, Issue 2, Pages 116-125

Publisher

NATURE PUBLISHING GROUP
DOI: 10.1038/35038557

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Funding

  1. NCI NIH HHS [R01 CA097061, R01 CA097061-01] Funding Source: Medline

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Chemical genetics is the study of gene-product function in a cellular or organismal context using exogenous ligands. In this approach, small molecules that bind directly to proteins are used to alter protein function, enabling a kinetic analysis of the in vivo consequences of these changes. Recent advances have strongly enhanced the power of exogenous ligands such that they can resemble genetic mutations in terms of their general applicability and target specificity. The growing sophistication of this approach raises the possibility of its application to any biological process.

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