Jan 9, 2010

RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform

Annual Review of Pharmacology and Toxicology
C Frank Bennett, Eric E Swayze

Abstract

Dramatic advances in understanding of the roles RNA plays in normal health and disease have greatly expanded over the past 10 years and have made it clear that scientists are only beginning to comprehend the biology of RNAs. It is likely that RNA will become an increasingly important target for therapeutic intervention; therefore, it is important to develop strategies for therapeutically modulating RNA function. Antisense oligonucleotides are perhaps the most direct therapeutic strategy to approach RNA. Antisense oligonucleotides are designed to bind to the target RNA by well-characterized Watson-Crick base pairing, and once bound to the target RNA, modulate its function through a variety of postbinding events. This review focuses on the molecular mechanisms by which antisense oligonucleotides can be designed to modulate RNA function in mammalian cells and how synthetic oligonucleotides behave in the body.

  • References184
  • Citations436

References

  • References184
  • Citations436

Mentioned in this Paper

Peptide Nucleic Acids
Chills
Monoclonal antibodies, antineoplastic
Biochemical Pathway
AGO2 gene
Urine
DNA, Catalytic
TPI ASM8
Conjugation
Antineoplastic Agents

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