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.

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

Peptide nucleic acids (PNA): synthesis, properties and potential applications

2′-deoxy-2′-alkylnucleotides useful for stabilizing enzymatic nucleic acid molecules and antisense molecules.

2'-deoxy-2'alkylnucleotide containing nucleic acid

Developing artificial hydrolytic metalloenzymes by a unified mechanistic approach

Recent developments in the synthesis of C-glycosides

Amides as a New Type of Backbone Modification in Oligonucleotides

Stereoselective C-C bond formation in carbohydrates by radical cyclization reactions-II

Stereoselective CC bond formation in carbohydrates by radical cyclization reactions-III. Strategy for the preparation of C(1)-glycosides

Stereoselective CC bond formation in carbohydrates by radical cyclization reactions-I.

Stereocontrol in radical cyclizations on sugar templates

Pascale Hoffmann

Papers

Amides as a New Type of Backbone Modification in Oligonucleotides

Stereoselective C-C bond formation in carbohydrates by radical cyclization reactions-II

Stereoselective CC bond formation in carbohydrates by radical cyclization reactions-III. Strategy for the preparation of C(1)-glycosides

Stereoselective CC bond formation in carbohydrates by radical cyclization reactions-I.

Stereocontrol in radical cyclizations on sugar templates