Rossi J J
Department of Molecular Biology, Beckman Research Institute of the City of Hope, Duarte, CA 91010-3011, USA.
Ciba Found Symp. 1997;209:195-204; discussion 204-6. doi: 10.1002/9780470515396.ch14.
Ribozymes have progressed from an intriguing subject of scientific study to therapeutic agents for the potential treatment of a fatal and devastating viral infection. Despite this rapid road to clinical trials, there are many unexplored avenues that should be examined to improve the intracellular effectiveness of ribozymes. Since ribozymes are RNA molecules, the cellular rules governing RNA partitioning and stability can be applied to these molecules to make them more effective therapeutic agents. Future, successful therapeutic ribozyme applications will depend upon increasing our knowledge of RNA metabolism and movement, and applying this knowledge to the design of ribozymes. This chapter discusses experimental approaches towards this goal as well as recent progress in the application of a pair of hammerhead ribozymes for the clinical treatment of HIV1 infection.
核酶已从一个引人入胜的科学研究课题发展成为用于潜在治疗致命且具有破坏性的病毒感染的治疗剂。尽管在通往临床试验的道路上进展迅速，但仍有许多未探索的途径有待研究，以提高核酶在细胞内的有效性。由于核酶是RNA分子，支配RNA分配和稳定性的细胞规则可应用于这些分子，使其成为更有效的治疗剂。未来，成功的治疗性核酶应用将取决于我们对RNA代谢和移动的了解不断增加，并将这些知识应用于核酶的设计。本章讨论了实现这一目标的实验方法，以及一对锤头状核酶在临床治疗HIV-1感染中的应用进展。
