4.6 Review

Destroying RNA as a therapeutic approach

期刊

CURRENT MEDICINAL CHEMISTRY
卷 13, 期 8, 页码 863-881

出版社

BENTHAM SCIENCE PUBL LTD
DOI: 10.2174/092986706776361021

关键词

RNA degradation; endonucleolytic cleavage; cytotoxic ribonuclease; antisense oligonucleotides; RNase P-external guide sequence; siRNA; ribozyme; DNazyme

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The ability to target RNA. mRNA and viral RNA in particular, for degradation is a powerful approach in molecular biology and pharmacology. Such approaches can be used in the Study of gene function its in functional genomics, in the identification of disease-associated genes, and for the treatment or human diseases. This review provides a comprehensive up-to-date look at all the current available technologies used for the destruction of RNA, with a focus oil their therapeutic potential. This includes approaches that utilize the activity of protein ribonucleases such as antisense oligonucleotide, small interfering RNA, RNase P-associated external guide sequence, onconase and bovine seminal RNase. Sequence-specific approaches that do not utilize activity of protein ribonucleases, such as ribozyme and DNazyme. are also reviewed and discussed. This review should provide a useful starting framework for researchers interested in using the RNA-destruction methodologies oil the bench and in the clinic, and serves as a stimulus for further development of novel and more potent RNA degradation technologies. This is particularly critical, given the anticipation of discoveries of new cellular RNA degradation machineries and human diseases that are associated with dysfunctional RNA molecules.

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