Design of a Novel Allosteric Ribozyme “Maxizyme” with a Sensor Function

Abstract

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 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. 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.