センサー機能をもつ遺伝子治療薬 : 核酸の熱力学的性質に基づくリボザイムの配列設計

概要

論文の詳細を見る
Ribozymes are potential tools for future gene therapy. Recenlty, we designed an allosterically controllable novel ribozyme (designated maxizyme) that can be transcribed in vivo under the control of a human tRNA~Val-promotor. 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 Mg~2+ 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.
日本生物物理学会の論文
2000-03-25