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

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Nucleic acids that bind to target molecules
- Series Introduction: Emerging clinical applications of nucleic acids

The concept of using nucleic acids to bind to and inhibit the activities of target proteins grew out of early HIV gene therapy studies that employed RNA ligands, termed decoys, to competitively inhibit the activities of essential HIV proteins and in the process block viral replication (1). TAR and RRE decoys were expressed in cells to bind and squelch the activities of the HIV RNA-binding proteins tat and rev. Similarly, double-stranded DNA decoys have been employed to squelch the activities of a variety of transcription factors (2). The use of combinatorial libraries of nucleic acids and in vitro selection methods, termed SELEX, allow nucleic acid–based ligands (aptamers) to be developed as specific, high-affinity antagonists to virtually any target protein (3, 4). The ability to modify nucleic acids to enhance the stability and bioavailability of decoys and aptamers should allow these nucleic acid–based therapeutics to be administered in a manner more like traditional drug delivery than like gene therapy.

Three papers in this series describe the development of therapeutic decoys and aptamers. Hicke and Stephens discuss the potential utility of aptamers as imaging reagents, and White, Sullenger, and Rusconi review the development of therapeutic aptamers. Finally, Mann and Dzau provide the reader with an overview of the transcription factor decoy approach and discuss results from early clinical trials using this therapeutic strategy.

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