title: Development of hammerhead ribozymes to modulate endogenous gene expression for functional studies.
abstract: Hammerhead ribozymes are catalytic RNAs that are being used to inhibit endogenous gene expression to study key components of basic biochemical pathways such as angiogenesis. In addition, these ribozymes have the potential to be used as components of gene therapy protocols for the treatment of disease states. We detail here a set of protocols for the design and testing of hammerhead ribozymes that will efficiently inhibit gene expression both in cell culture and in vivo.
authors.affiliation: Department of Molecular Genetics and Microbiology, University of Florida, College of Medicine, Box 100267, Gainesville, FL 32610, USA.
authors.forename: Jason Jon
authors.initials: JJ
authors.lastname: Fritz
authors.forename: Alfred
authors.initials: A
authors.lastname: Lewin
authors.forename: William
authors.initials: W
authors.lastname: Hauswirth
authors.forename: Anupam
authors.initials: A
authors.lastname: Agarwal
authors.forename: Maria
authors.initials: M
authors.lastname: Grant
authors.forename: Lynn
authors.initials: L
authors.lastname: Shaw
mesh_terms: D000256:Adenoviridae / Q000235:genetics; D002138:Calibration; D002478:Cells, Cultured; D004727:Endothelium / Q000166:cytology; D005786:Gene Expression Regulation; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics; D006801:Humans; D066298:In Vitro Techniques; D009690:Nucleic Acid Conformation; D010957:Plasmids / Q000235:genetics; D016337:RNA, Catalytic / Q000737:chemistry / Q000235:genetics*; D014162:Transfection / Q000379:methods*
chemical_list: D016337:RNA, Catalytic; C427204:hammerhead ribozyme