Gene-expressed RNA as a therapeutic: issues to consider, using ribozymes and small hairpin RNA as specific examples.
In recent years there has been a greater appreciation of both the role of RNA in intracellular gene regulation and the potential to use RNA in therapeutic modalities. In the latter case, RNA can be used as a therapeutic target or a drug. The chapters in this volume cover the varied and potent actions of RNA as antisense, ribozymes, aptamers, microRNA and small hairpin RNA in gene regulation, as well as their use as potential therapeutics for metabolic and infectious diseases. Our group has been involved in the development of anti-HIV gene expression constructs to treat HIV. In this chapter, we address the relevant scientific and some of the commercial issues in the use of RNA as a therapeutic. Specifically, the chapter discusses delivery, expression, potency, toxicity and commercial development using, as examples, hammerhead ribozymes and small hairpin RNA.
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Johnson Johnson Research, The Australian Technology Park, Strawberry Hills, Locked Bag 4555, 2012 Sydney NSW, Australia.
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D000818:Animals; D005786:Gene Expression Regulation / Q000187:drug effects; D006801:Humans; D009690:Nucleic Acid Conformation; D012313:RNA / Q000096:biosynthesis / Q000737:chemistry / Q000235:genetics / Q000627:therapeutic use*; D034622:RNA Interference; D016337:RNA, Catalytic / Q000627:therapeutic use*
D016337:RNA, Catalytic; D012313:RNA