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Nucleic Acids Res. 2003 Nov 1;31(21):6064-78.

Therapeutic modulation of endogenous gene function by agents with designed DNA-sequence specificities.

Author information

  • 1Department of Molecular Cell Biology, Leiden University Medical Center, Leiden, The Netherlands.

Abstract

Designer molecules that can specifically target pre-determined DNA sequences provide a means to modulate endogenous gene function. Different classes of sequence-specific DNA-binding agents have been developed, including triplex-forming molecules, synthetic polyamides and designer zinc finger proteins. These different types of designer molecules with their different principles of engineered sequence specificity are reviewed in this paper. Furthermore, we explore and discuss the potential of these molecules as therapeutic modulators of endogenous gene function, focusing on modulation by stable gene modification and by regulation of gene transcription.

PMID:
14576293
[PubMed - indexed for MEDLINE]
PMCID:
PMC275457
Free PMC Article

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