title: A potential therapeutic application of hairpin ribozymes: in vitro and in vivo studies of gene therapy for hepatitis C virus infection.
abstract: Two effective ribozymes (CR2 and CR4) that target HCV RNA 5' UTR and capsid gene regions were generated. Ribozyme cleavage was demonstrated in vitro, which can be enhanced by facilitator RNA molecules. In tissue culture cells, these two ribozymes can inhibit the expression of a cotransfected reporter gene containing HCV RNA target sequences. Furthermore, transduction of human hepatoma cells, HepG2, with retroviral vectors carrying CR2 or CR4 ribozymes enabled the cells to resist the infection by retroviral particles containing HCV target sequences. These results represent the first positive step towards the application of hairpin ribozymes in gene therapy for the treatment of HCV infection.
authors.affiliation: Immusol Inc, San Diego, CA 92121, USA.
authors.forename: P J
authors.initials: PJ
authors.lastname: Welch
authors.forename: R
authors.initials: R
authors.lastname: Tritz
authors.forename: S
authors.initials: S
authors.lastname: Yei
authors.forename: M
authors.initials: M
authors.lastname: Leavitt
authors.forename: M
authors.initials: M
authors.lastname: Yu
authors.forename: J
authors.initials: J
authors.lastname: Barber
mesh_terms: D000818:Animals; D002213:Capsid / Q000235:genetics; D002460:Cell Line; D018014:Gene Transfer Techniques; D016174:Hepacivirus / Q000235:genetics*; D006801:Humans; D051379:Mice; D010865:Pilot Projects; D016337:RNA, Catalytic / Q000235:genetics* / Q000378:metabolism; D012367:RNA, Viral / Q000378:metabolism*; D014407:Tumor Cells, Cultured
chemical_list: D016337:RNA, Catalytic; D012367:RNA, Viral