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Updated: Aug 30, 2026

In Vitro Selection of Engineered Transcriptional Repressors for Targeted Epigenetic Silencing
Published on: May 5, 2023
Therapeutic modulation of endogenous gene function by agents with designed DNA-sequence specificities
Taco G Uil1, Hidde J Haisma, Marianne G Rots
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.
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