Therapeutic modulation of endogenous gene function by agents with designed DNA-sequence specificities
Open Access
- 1 November 2003
- journal article
- review article
- Published by Oxford University Press (OUP) in Nucleic Acids Research
- Vol. 31 (21) , 6064-6078
- https://doi.org/10.1093/nar/gkg815
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.Keywords
This publication has 98 references indexed in Scilit:
- Pyrimidine Morpholino Oligonucleotides Form a Stable Triple Helix in the Absence of Magnesium IonsBiochemical and Biophysical Research Communications, 2000
- Analysis of zinc fingers optimized via phage display: evaluating the utility of a recognition codeJournal of Molecular Biology, 1999
- Artificial Nine Zinc-Finger Peptide with 30 Base Pair Binding SitesBiochemistry, 1998
- Efficient Inhibition of Transcription Elongationin vitroby Oligonucleotide Phosphoramidates Targeted to Proviral HIV DNAJournal of Molecular Biology, 1996
- Splase: A new class IIs zinc-finger restriction endonuclease with specificity for Sp1 binding sitesProtein Journal, 1996
- Inhibition of aromatase expression by a psoralen-linked triplex-forming oligonucleotide targeted to a coding sequenceFEBS Letters, 1995
- Inhibition of in Vitro Transcription by a Triplex-Forming Oligonucleotide Targeted to Human c-myc P2 PromoterBiochemistry, 1995
- Transport of oligonucleotides across natural and model membranesBiochimica et Biophysica Acta (BBA) - Reviews on Biomembranes, 1994
- Oligo(2′‐O‐methyl)ribonucleotides Effective probes for duplex DNAFEBS Letters, 1992
- GAL4-VP16 is an unusually potent transcriptional activatorNature, 1988