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Biopharmaceutical Drug Design and Development pp 193–221Cite as

Small Nucleic Acid-Based Drugs: Successes and Pitfalls

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Abstract

Since the discovery of the application of small nucleic acid molecules as inhibitors of gene expression, literally thousands of new research papers have used this technique for functional genomics or to test these molecules as potential therapeutic agents. During this time, the field has evolved from simple phosphodiester oligonucleotide (ODN) binding to complementary mRNA in vitro, to the development of hundreds of chemically modified ODNs with improved properties and mechanisms of action. RNA-based gene inhibitors have also powerfully merged onto the scene with first ribozymes, and now siRNA, providing new approaches to gene inhibition. This chapter will review the area of small nucleic acid drugs in terms of their design, delivery, and application.

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Authors and Affiliations

  1. Abramson Research Center, Children’s Hospital of Philadelphia, Philadelphia, PA

    A. Yin Zhang PharmaD, PhD (Postdoctoral Fellow)

  2. Pharmaceutical Sciences Graduate Program, Virginia Commonwealth University School of Pharmacy, Richmond, VA

    Susanna Wu-Pong (Director)

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  1. Pharmaceutical Sciences Graduate Programs Department of Pharmaceutics, School of Pharmacy, Virginia Commonwealth University, Richmond, VA

    Susanna Wu-Pong PhD (Director) (Director)

  2. Virginia Commonwealth University, Box 980581, Medical College of Virginia Campus, Richmond, VA, 23298

    Susanna Wu-Pong PhD (Director) (Director)

  3. Department of Basic Pharmaceutical Sciences, West Virginia University, Morgantown, WV

    Yon Rojanasakul PhD (Professor of Pharmaceutical and Pharmacological Sciences) (Professor of Pharmaceutical and Pharmacological Sciences)

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Zhang, A.Y., Wu-Pong, S. (2008). Small Nucleic Acid-Based Drugs: Successes and Pitfalls. In: Wu-Pong, S., Rojanasakul, Y. (eds) Biopharmaceutical Drug Design and Development. Humana Press. https://doi.org/10.1007/978-1-59745-532-9_10

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