Pseudoknot Interaction-Mediated Activation of Type i Hammerhead Ribozyme: A New Class of Gene-Therapeutic Agents

Mituhiro Kuriyama, Yoshinori Kondo, Yoshiyuki Tanaka

Research output: Contribution to journalArticlepeer-review

3 Citations (Scopus)

Abstract

Recently discovered hammerhead ribozymes that are activated through pseudoknot interactions (Watson-Crick base pairs between loops) are attractive candidates as gene-therapeutic agents because sequences of gene-therapeutic ribozymes can be designed simply based on the sequence complementarity against target RNAs. Herein, we examined if the newly found pseudoknot-type hammerhead ribozyme with type I topology is activated through the pseudoknot interactions. Substitutions of pseudoknot sequences into fully mismatched ones significantly reduced the activity of type I pseudoknot-type hammerhead ribozyme, while those with full-matched pseudoknot sequences were highly active. The results indicated that the pseudoknot interactions activated type I pseudoknot-type hammerhead ribozyme, making them suitable as gene-therapeutic agents.

Original languageEnglish
Pages (from-to)466-480
Number of pages15
JournalNucleosides, Nucleotides and Nucleic Acids
Volume33
Issue number7
DOIs
Publication statusPublished - 2014 Jul 3

Keywords

  • Ribozymes
  • gene regulation
  • nucleoside synthesis

ASJC Scopus subject areas

  • Biochemistry
  • Molecular Medicine
  • Genetics

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