Shippy R, Lockner R, Farnsworth M, Hampel A
Department of Biological Sciences, Northern Illinois University, DeKalb 60115, USA.
Mol Biotechnol. 1999 Aug;12(1):117-29. doi: 10.1385/MB:12:1:117.
The hairpin ribozyme is a member of a family of small RNA endonucleases, which includes hammer-head, human hepatitis delta virus, Neurospora VS, and the lead-dependent catalytic RNAs. All these catalytic RNAs reversibly cleave the phosphodiester bond of substrate RNA to generate 5'-hydroxyl and 2',3'-cyclic phosphate termini. Whereas the reaction products from family members are similar, large structural and mechanistic differences exist. Structurally the hairpin ribozyme has two principal domains that interact to facilitate catalysis. The hairpin ribozyme uses a catalytic mechanism that does not require metals for cleavage or ligation of substrate RNA. In this regard it is presently unique among RNA catalysts. Targeting rules for cleavage of substrate have been determined and required bases for catalysis have been identified. The hairpin ribozyme has been developed and used for gene therapy and was the first ribozyme to be approved for human clinical trials.
发夹状核酶是小RNA内切核酸酶家族的成员之一，该家族还包括锤头状核酶、人类丁型肝炎病毒、粗糙脉孢菌VS核酶以及铅依赖性催化RNA。所有这些催化RNA都能可逆地切割底物RNA的磷酸二酯键，生成5'-羟基和2',3'-环磷酸末端。虽然家族成员的反应产物相似，但在结构和作用机制上存在很大差异。在结构上，发夹状核酶有两个主要结构域，它们相互作用以促进催化作用。发夹状核酶使用的催化机制在切割或连接底物RNA时不需要金属。在这方面，它目前在RNA催化剂中是独一无二的。已经确定了切割底物的靶向规则，并鉴定了催化所需的碱基。发夹状核酶已被开发并用于基因治疗，是第一个被批准用于人体临床试验的核酶。
