抄録
Ribozymes are potential tools for future gene therapy. Recently, we designed an allosterically controllable novel ribozyme (designated maxizyme) that can be transcribed in vivo under the control of a human tRNAVal-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. The maxizyme (but not conventional ribozymes) has extremely high specificity and high-level activity, not only in vitro but also in vivo. To the best of our knowledge, our novel maxizyme is superior to other nucleic acid-based drugs reported to date because of its extremely high substrate-specificity and high cleavage activity.
