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Intracellular expression of engineered RNase P ribozymes effectively blocks gene expression and replication of human

Kihoon Kim1, Sean Umamoto, Phong Trang

  • 1Program in Infectious Diseases and Immunity, School of Public Health, University of California, Berkeley, California 94720, USA.

RNA (New York, N.Y.)
|February 19, 2004
PubMed

Insights

Ribozymes targeting human cytomegalovirus (HCMV) mRNAs significantly reduced viral protease and capsid assembly protein expression. This ribozyme therapy blocked viral growth and DNA packaging, showing promise for antiviral gene therapy.

Area of Science:

  • Molecular Biology
  • Virology
  • Biochemistry

Background:

  • Human cytomegalovirus (HCMV) is a significant pathogen, necessitating novel antiviral strategies.
  • Ribozymes, catalytic RNA molecules, offer precise gene targeting capabilities.
  • RNase P ribozymes have shown potential for therapeutic applications.

Purpose of the Study:

  • To investigate the efficacy of an RNase P ribozyme (M1GS RNA) against human cytomegalovirus (HCMV).
  • To assess the ribozyme's ability to inhibit the expression of essential viral genes.
  • To evaluate the therapeutic potential of ribozymes in controlling HCMV replication.

Main Methods:

  • Constructed a ribozyme (M1GS RNA) from the RNase P of Escherichia coli.
  • Targeted the overlapping region of HCMV protease (PR) and capsid assembly protein (AP) mRNAs.
  • Expressed the ribozyme in cells and measured viral gene expression and replication.

Main Results:

  • Achieved >80% reduction in PR and AP expression levels.
  • Demonstrated a ~2000-fold inhibition of HCMV viral growth in ribozyme-expressing cells.
  • Observed blocked viral genomic DNA packaging into capsids, indicating impaired viral assembly.

Conclusions:

  • RNase P ribozymes are highly effective in inhibiting HCMV replication by targeting essential viral mRNAs.
  • The ribozyme specifically downregulates PR and AP expression, disrupting viral capsid formation.
  • This study validates the feasibility of using ribozymes for gene therapy against HCMV infections.

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