Efficient reduction of target RNAs by small interfering RNA and RNase H-dependent antisense agents. A comparative analysis

J Biol Chem. 2003 Feb 28;278(9):7108-18. doi: 10.1074/jbc.M210326200. Epub 2002 Dec 23.

Abstract

RNA interference can be considered as an antisense mechanism of action that utilizes a double-stranded RNase to promote hydrolysis of the target RNA. We have performed a comparative study of optimized antisense oligonucleotides designed to work by an RNA interference mechanism to oligonucleotides designed to work by an RNase H-dependent mechanism in human cells. The potency, maximal effectiveness, duration of action, and sequence specificity of optimized RNase H-dependent oligonucleotides and small interfering RNA (siRNA) oligonucleotide duplexes were evaluated and found to be comparable. Effects of base mismatches on activity were determined to be position-dependent for both siRNA oligonucleotides and RNase H-dependent oligonucleotides. In addition, we determined that the activity of both siRNA oligonucleotides and RNase H-dependent oligonucleotides is affected by the secondary structure of the target mRNA. To determine whether positions on target RNA identified as being susceptible for RNase H-mediated degradation would be coincident with siRNA target sites, we evaluated the effectiveness of siRNAs designed to bind the same position on the target mRNA as RNase H-dependent oligonucleotides. Examination of 80 siRNA oligonucleotide duplexes designed to bind to RNA from four distinct human genes revealed that, in general, activity correlated with the activity to RNase H-dependent oligonucleotides designed to the same site, although some exceptions were noted. The one major difference between the two strategies is that RNase H-dependent oligonucleotides were determined to be active when directed against targets in the pre-mRNA, whereas siRNAs were not. These results demonstrate that siRNA oligonucleotide- and RNase H-dependent antisense strategies are both valid strategies for evaluating function of genes in cell-based assays.

MeSH terms

  • Animals
  • Base Sequence
  • COS Cells
  • Dose-Response Relationship, Drug
  • Flow Cytometry
  • Humans
  • Intercellular Adhesion Molecule-1 / biosynthesis
  • Introns
  • Luciferases / metabolism
  • Molecular Sequence Data
  • Nucleic Acid Conformation
  • Oligonucleotides / pharmacology
  • Oligonucleotides, Antisense / pharmacology
  • PTEN Phosphohydrolase
  • Phosphoric Monoester Hydrolases / genetics
  • Phosphoric Monoester Hydrolases / metabolism
  • Protein Structure, Tertiary
  • RNA / metabolism
  • RNA, Messenger / metabolism
  • RNA, Small Interfering / metabolism*
  • Ribonuclease H / metabolism*
  • Time Factors
  • Tumor Cells, Cultured
  • Tumor Suppressor Proteins / genetics
  • Tumor Suppressor Proteins / metabolism

Substances

  • Oligonucleotides
  • Oligonucleotides, Antisense
  • RNA, Messenger
  • RNA, Small Interfering
  • Tumor Suppressor Proteins
  • Intercellular Adhesion Molecule-1
  • RNA
  • Luciferases
  • Ribonuclease H
  • Phosphoric Monoester Hydrolases
  • PTEN Phosphohydrolase
  • PTEN protein, human