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- W1569668463 abstract "Publisher Summary Ribozymes represent a relatively new approach for specific inactivation of gene expression. To date, ribozymes have been reported to be effective in cell culture against a variety of RNA targets, including viral, endogenous, and transfected sequences. Ribozymes have been utilized to functionally destroy mRNAs as well as for the repair of mutant RNAs. These RNA catalysts have progressed to the stage where both hammerhead and hairpin ribozymes are currently being tested in human clinical trials. The effective use of ribozymes in vivo depends on effective target-site selection, knowledge of the rules governing intracellular localization, effective delivery to target cells, and maintaining a high and long-term intracellular expression of the engineered gene. Over the past decade, ribozymes have been widely employed in a variety of systems. Their use as tools to control gene expression is both enticing and simple in concept. Ribozymes can be designed to specifically base pair with and cleave any given RNA target, with the result of producing an irreversible inactivation of the targeted RNA. Because these catalytic RNAs can be designed to have exquisite specificity and are inherently devoid of coding capacities, there is little likelihood for undesired cellular toxicities. The combination of specificity, the possibilities of endogeneous synthesis or exogenous delivery, and the capability to be adapted for the destruction or modification of any RNA species make ribozymes extremely attractive genetic tools and potential therapeutic agents. The major limitations to effective intracellular ribozyme use are colocalization of ribozymes with target RNAs and target-site accessibility. These specific problems are discussed in this chapter. In addition, strategies and protocols for expression and delivery of ribozyme genes are addressed." @default.
- W1569668463 created "2016-06-24" @default.
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- W1569668463 date "2000-01-01" @default.
- W1569668463 modified "2023-10-16" @default.
- W1569668463 title "Intracellular expression and function of antisense catalytic RNAs" @default.
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- W1569668463 doi "https://doi.org/10.1016/s0076-6879(00)13025-3" @default.
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