Small Nucleic Acid-Based Drugs: Successes and Pitfalls

TL;DRAbstract

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.

Chat with Paper

AI Agents for this Paper