title: Designing ribozymes for the inhibition of gene expression.
abstract: Ribozymes are being increasingly used for the sequence-specific inhibition of gene expression by the cleavage of mRNAs encoding proteins of interest. However, particular attention must be paid to the following points: the identification of regions on the mRNA accessible to the ribozyme; the delivery of ribozymes to cells by either exogenous or endogenous delivery; colocalization of the ribozyme with the target RNA in the cell; and differentiation between closely related sequences. This field is advancing rapidly, and results obtained with transgenic animals demonstrate the power of this strategy for the inhibition of gene expression.
authors.affiliation: Max-Planck-Institut für Experimentelle Medizin, Göttingen, Germany.
authors.forename: B
authors.initials: B
authors.lastname: Bramlage
authors.forename: E
authors.initials: E
authors.lastname: Luzi
authors.forename: F
authors.initials: F
authors.lastname: Eckstein
mesh_terms: D000818:Animals; D004337:Drug Carriers; D016503:Drug Delivery Systems; D005786:Gene Expression Regulation / Q000187:drug effects*; D005822:Genetic Vectors / Q000235:genetics* / Q000494:pharmacology; D006801:Humans; D012313:RNA / Q000187:drug effects; D016337:RNA, Catalytic / Q000737:chemistry* / Q000235:genetics / Q000494:pharmacology*
chemical_list: D004337:Drug Carriers; D016337:RNA, Catalytic; D012313:RNA