Novel approaches for gene-specific interference via manipulating actions of microRNAs: examination on the pacemaker channel genes HCN2 and HCN4

J Cell Physiol. 2007 Aug;212(2):285-92. doi: 10.1002/jcp.21062.

Abstract

Recent evidence has suggested microRNAs as viable therapeutic targets for a wide range of human disease. However, lack of gene-specificity of microRNA actions may hinder this application. Here we developed two new approaches, the gene-specific microRNA mimic and microRNA-masking antisense approaches, to explore the possibility of using microRNA's principle of actions in a gene-specific manner. We examined the value of these strategies as rational approaches to develop heart rate-reducing agents and "biological pacemakers" by manipulating the expression of the cardiac pacemaker channel genes HCN2 and HCN4. We showed that the gene-specific microRNA mimics, 22-nt RNAs designed to target the 3'untranslated regions (3'UTRs) of HCN2 and HCN4, respectively, were efficient in abrogating expression and function of HCN2 and HCN4. The gene-specific microRNA mimics repressed protein levels, accompanied by depressed f-channel conductance and the associated rhythmic activity, without affecting mRNA levels of HCN2 and HCN4. Meanwhile, we also designed the microRNA-masking antisense based on the miR-1 and miR-133 target sites in the 3'UTRs of HCN2 and HCN4 and found that these antisense oligodeoxynucleotides markedly enhanced HCN2/HCN4 expression and function, as reflected by increased protein levels of HCN2/HCN4 and If conductance, by removing the repression of HCN2/HCN4 expression induced by endogenous miR-1/miR-133. The experimental examination of these techniques and the resultant findings not only indicate feasibility of interfering miRNA action in a gene-specific fashion but also may provide a new research tool for studying function of miRNAs. The new approaches also have the potential of becoming alternative gene therapy strategies.

Publication types

  • Evaluation Study
  • Research Support, Non-U.S. Gov't
  • Retracted Publication

MeSH terms

  • 3' Untranslated Regions
  • Animals
  • Animals, Newborn
  • Arrhythmias, Cardiac / genetics
  • Arrhythmias, Cardiac / metabolism
  • Arrhythmias, Cardiac / therapy
  • Cell Line
  • Down-Regulation
  • Feasibility Studies
  • Genetic Therapy / methods*
  • Heart Rate* / genetics
  • Heart Ventricles / cytology
  • Heart Ventricles / metabolism
  • Humans
  • Hyperpolarization-Activated Cyclic Nucleotide-Gated Channels
  • Ion Channels / genetics
  • Ion Channels / metabolism*
  • Membrane Potentials
  • MicroRNAs / genetics
  • MicroRNAs / metabolism*
  • Mutation
  • Myocytes, Cardiac / metabolism*
  • Oligonucleotides, Antisense / genetics
  • Oligonucleotides, Antisense / metabolism
  • Potassium / metabolism*
  • Potassium Channels / genetics
  • Potassium Channels / metabolism*
  • RNA Interference*
  • RNA, Messenger / metabolism
  • Rats
  • Transfection

Substances

  • 3' Untranslated Regions
  • HCN2 protein, human
  • HCN4 protein, rat
  • Hcn2 protein, rat
  • Hyperpolarization-Activated Cyclic Nucleotide-Gated Channels
  • Ion Channels
  • MicroRNAs
  • Oligonucleotides, Antisense
  • Potassium Channels
  • RNA, Messenger
  • Potassium