title: Pseudoknot interaction-mediated activation of type I hammerhead ribozyme: a new class of gene-therapeutic agents.
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.
authors: a Laboratory of Molecular Transformation, Graduate School of Pharmaceutical Sciences , Tohoku University , Sendai , Miyagi , Japan.
authors: Mituhiro
authors: M
authors: Kuriyama
authors: Yoshinori
authors: Y
authors: Kondo
authors: Yoshiyuki
authors: Y
authors: Tanaka
mesh_terms: D020029:Base Pairing; D001483:Base Sequence; D055162:Biocatalysis; D004789:Enzyme Activation; D015316:Genetic Therapy / Q000379:methods*; D016337:RNA, Catalytic / Q000737:chemistry* / Q000378:metabolism*; D025062:Yarrowia / Q000201:enzymology
chemical_list: D016337:RNA, Catalytic; C427204:hammerhead ribozyme
keywords: Ribozymes; gene regulation; nucleoside synthesis