title: Allosterically controllable maxizymes for molecular gene therapy.
abstract: Ribozymes are small and versatile nucleic acids that can cleave RNAs at specific sites. These molecules have great potential to be used as effective gene therapeutic agents. However, conventional ribozymes have, in some cases, failed to exhibit precise cleavage specificity because they require cleavable sequences in the target mRNA. Recently, we demonstrated that an allosterically controllable novel ribozyme, designated the maxizyme, is a powerful tool for disruption of an abnormal chimeric RNA target (BCR-ABL (b2a2) mRNA) in cells and in mice. Furthermore, more than five custom-designed maxizymes have demonstrated these allosteric functions in vitro and in vivo. Thus, maxizyme technology is not limited to a specific case but may have broad general applicability in molecular biology and in molecular gene therapy.
authors.affiliation: Department of Chemistry and Biotechnology, School of Engineering, The University of Tokyo, Hongo, Japan.
authors.forename: Mayu
authors.initials: M
authors.lastname: Iyo
authors.forename: Hiroaki
authors.initials: H
authors.lastname: Kawasaki
authors.forename: Kazunari
authors.initials: K
authors.lastname: Taira
mesh_terms: D000494:Allosteric Regulation; D000818:Animals; D015316:Genetic Therapy; D051379:Mice; D016337:RNA, Catalytic / Q000378:metabolism / Q000627:therapeutic use*; D013379:Substrate Specificity
chemical_list: D016337:RNA, Catalytic