Ribozymes: RNA as a therapeutic agent

RZs, like ASO agents, are several years from approval by the Food and Drug Administration. Although many problems must be solved before they can be used effectively as therapeutic agents, results from a wide variety of in vitro and in vivo experiments are encouraging. The advantages of RZs are their very high specificity of action, their catalytic mechanism, their potential for targeted therapy, and the lack of side effects with their use. These advantages, coupled with the wide applicability of ribozyme technology, will continue to drive the research necessary to develop RZs into useful therapeutic agents.