Induction of endogenous gamma-globin gene expression with decoy oligonucleotide targeting Oct-1 transcription factor consensus sequence

J Hematol Oncol. 2009 Mar 27:2:15. doi: 10.1186/1756-8722-2-15.

Abstract

Human beta-globin disorders are relatively common genetic diseases cause by mutations in the beta-globin gene. Increasing the expression of the gamma-globin gene has great benefits in reducing complications associated with these diseases. The Oct-1 transcription factor is involved in the transcriptional regulation of the gamma-globin gene. The human gamma-globin genes (both Agamma and Ggamma-globin genes) carry three Oct-1 transcription factor consensus sequences within their promoter regions. We have studied the possibility of inducing gamma-globin gene expression using decoy oligonucleotides that target the Oct-1 transcription factor consensus sequence. A double-stranded 22 bp decoy oligonucleotide containing the Oct-1 consensus sequence was synthesized. The results obtained from our in vitro binding assay revealed a strong competitive binding of the decoy oligonucleotide for the Oct-1 transcription factor. When K562 human erythroleukemia cells were treated with the Oct-1 decoy oligonucleotide, significant increases in the level of the gamma-globin mRNA were observed. The results of our western blots further demonstrated significant increases of the fetal hemoglobin (HbF, alpha2gamma2) in the Oct-1 decoy oligonucleotide-treated K562 cells. The results of our immunoprecipitation (IP) studies revealed that the treatment of K562 cells with the Oct-1 decoy oligonucleotide significantly reduced the level of the endogenous gamma-globin gene promoter region DNA co-precipitated with the Oct-1 transcription factor. These results suggest that the decoy oligonucleotide designed for the Oct-1 transcription factor consensus sequence could induce expression of the endogenous gamma-globin gene through competitive binding of the Oct-1 transcription factor, resulting in activation of the gamma-globin genes. Therefore, disrupting the bindings of the Oct-1 transcriptional factors with the decoy oligonucleotide provides a novel approach for inducing expression of the gamma-globin genes. It also provides an innovative strategy for the treatment of many disease conditions, including sickle cell anemia and beta-thalassemia.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Binding Sites
  • Binding, Competitive
  • Cell Survival / drug effects
  • Consensus Sequence*
  • Gene Expression Regulation, Leukemic / drug effects*
  • Gene Targeting / methods
  • Humans
  • K562 Cells
  • Leukemia, Erythroblastic, Acute / genetics
  • Leukemia, Erythroblastic, Acute / pathology
  • Octamer Transcription Factor-1 / antagonists & inhibitors*
  • Octamer Transcription Factor-1 / metabolism
  • Oligonucleotides / pharmacology*
  • Protein Binding
  • Regulatory Sequences, Nucleic Acid / genetics
  • Up-Regulation
  • gamma-Globins / genetics*

Substances

  • Octamer Transcription Factor-1
  • Oligonucleotides
  • gamma-Globins