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Attempts to employ nucleic acids in gene therapy have become commonplace in …


Biology Articles » Biochemistry » Nucleic Acid Biochemistry » Series Introduction: Emerging clinical applications of nucleic acids » Nucleic acids that perform catalysis

Nucleic acids that perform catalysis
- Series Introduction: Emerging clinical applications of nucleic acids

The discovery by Cech and Altman that certain RNAs can perform catalysis dramatically changed how scientists viewed the role of nucleic acids in nature (6, 7). The observation that certain catalytic RNAs, termed ribozymes, can be made to specifically cleave (8, 9) or splice (10) target RNAs has engendered much excitement about the potential therapeutic utility of these molecules. Much of this effort originally focused upon the use of ribozymes in gene therapy strategies. More recently however, much progress has been made in the development of synthetic nuclease-resistant ribozymes for therapeutic applications. In addition, through the use of in vitro evolution techniques, DNA-based enzymes have also been obtained that cleave target RNAs (11). In this Perspective series, Usman and Blatt describe recent advances in the use of synthetic ribozymes in animals and the clinic, and Khachigian discusses the potential utility of DNA enzymes as therapeutic agents.

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