A novel allosterically trans-activated ribozyme, the maxizyme, with exceptional specificity in vitro and in vivo

Mol Cell. 1998 Nov;2(5):617-27. doi: 10.1016/s1097-2765(00)80160-4.

Abstract

We have constructed an allosterically controllable novel enzyme (designated maxizyme) that can be transcribed in vivo under the control of a human tRNA(Val) promoter. The maxizyme has sensor arms that can recognize target sequences, and in the presence of such a target sequence only, it can form a cavity that can capture catalytically indispensable Mg2+ ions. As a target for a demonstration of the potential utility of the maxizyme, we chose BCR-ABL mRNA, the translated products of which cause chronic myelogenous leukemia. Only the maxizyme (but not conventional ribozymes) had extremely high specificity and high-level activity, not only in vitro but also in cultured cells including BV173 cells derived from a patient with a Philadelphia chromosome. The maxizyme induced apoptosis only in leukemic cells with this chromosome.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Allosteric Regulation
  • Animals
  • Apoptosis / drug effects
  • Base Sequence
  • Caspase 3
  • Caspases / metabolism
  • Cell Line
  • Cell Line, Transformed
  • Cytoplasm / metabolism
  • Dimerization
  • Enzyme Precursors / metabolism
  • Fusion Proteins, bcr-abl / genetics*
  • Gene Expression
  • Humans
  • Interleukin-3 / pharmacology
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / genetics
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / metabolism*
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / therapy
  • Magnesium / pharmacology
  • Mice
  • Nucleic Acid Conformation
  • Philadelphia Chromosome
  • RNA, Catalytic / metabolism*
  • RNA, Catalytic / therapeutic use
  • RNA, Messenger / metabolism*
  • RNA, Transfer, Val / genetics
  • Substrate Specificity
  • Transfection
  • Tumor Cells, Cultured

Substances

  • Enzyme Precursors
  • Interleukin-3
  • RNA, Catalytic
  • RNA, Messenger
  • RNA, Transfer, Val
  • Fusion Proteins, bcr-abl
  • CASP3 protein, human
  • Casp3 protein, mouse
  • Caspase 3
  • Caspases
  • Magnesium