Sullivan S M
Ribozyme Pharmaceuticals, Inc., Boulder, Colorado 80301.
J Invest Dermatol. 1994 Nov;103(5 Suppl):85S-89S. doi: 10.1038/jid.1994.15.
Ribozymes are a class of RNA molecules that can perform catalytically in the absence of protein. Specifically, they can hybridize to and cleave target RNA molecules independent of cellular proteins. The cleaved target RNA can not be translated thereby preventing synthesis of a specific protein. The therapeutic application is to target the ribozyme to the mRNA of a key protein or proteins involved in maintaining a disease state resulting in a cure. The ribozymes can be chemically synthesized and delivered to cells or they can be expressed from an expression vector following either permanent or transient transfection. Therapeutic applications of ribozymes have been in the areas of AIDS and cancer. The following article describes the ribozymes in more detail with respect to optimizing the design to obtain the maximal cleavage rate, identifying cleavage sites within the target RNA and delivering the ribozymes to cells of interest both in vitro and in vivo.
核酶是一类RNA分子，能够在没有蛋白质的情况下发挥催化作用。具体而言，它们可以与靶RNA分子杂交并将其切割，而无需细胞蛋白质的参与。被切割的靶RNA无法进行翻译，从而阻止特定蛋白质的合成。其治疗应用是将核酶靶向参与维持疾病状态的一种或多种关键蛋白质的mRNA，从而实现治愈。核酶可以通过化学合成并递送至细胞，或者在永久或瞬时转染后从表达载体中表达。核酶的治疗应用已涉及艾滋病和癌症领域。以下文章将更详细地描述核酶，包括优化设计以获得最大切割率、识别靶RNA内的切割位点以及在体外和体内将核酶递送至感兴趣的细胞。
