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Published January 2001 | Published
Journal Article Open

Chemical approaches to control gene expression

Abstract

A current goal in molecular medicine is the development of new strategies to interfere with gene expression in living cells in the hope that novel therapies for human disease will result from these efforts. This review focuses on small-molecule or chemical approaches to manipulate gene expression by modulating either transcription of messenger RNA-coding genes or protein translation. The molecules under study include natural products, designed ligands, and compounds identified through functional screens of combinatorial libraries. The cellular targets for these molecules include DNA, messenger RNA, and the protein components of the transcription, RNA processing, and translational machinery. Studies with model systems have shown promise in the inhibition of both cellular and viral gene transcription and mRNA utilization. Moreover, strategies for both repression and activation of gene transcription have been described. These studies offer promise for treatment of diseases of pathogenic (viral, bacterial, etc.) and cellular origin (cancer, genetic diseases, etc.).

Additional Information

© 2000 Cognizant Communication Corporation. Publication date: January 1, 2001. Research in the J.M.G. and P.B.D. laboratories is supported by grants from the National Institute of General Medical Sciences. J.M.T. thanks the Howard Hughes Medical Institute for a predoctoral fellowship. We thank Dr. J. K. Barton for providing Figure 9.

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