Antisense Oligonucleotides: Pharmacology and Delivery Strategies

Antisense Oligonucleotides: Strategies and Applications

Polymersome delivery of siRNA and antisense oligonucleotides.

siRNA and antisense oligonucleotides, AON, have similar size and negative charge and are often packaged for in vitro delivery with cationic lipids or polymers-but exposed positive charge is problematic in vivo. Here we demonstrate loading and functional delivery of RNAi and AON with non-ionic, nano-transforming polymersomes. These degradable carriers are taken up passively by cultured cells aft...

Antisense Oligonucleotides: Treatment Strategies and Cellular Internalization.

The clinical applicaton of antisense oligonucleotides (ASOs) is becoming more of a reality as several drugs have been approved for the treatment of human disorders and many others are in various phases in development and clinical trials. ASOs are short DNA/RNA oligos which are heavily modified to increase their stability in biological fluids and retain the properties of creating RNA-RNA and DNA...

In vitro delivery of antisense oligonucleotides.

Oligonucleotides (ODNs) do not readily get access to their desired site (cytoplasm/nucleus) of action due to their high hydrophility and high molecular weight. Therefore enhancing components (carriers) are needed. Most widely used in vitro are cationic lipids (liposomes). Other systems include peptide conjugates or complexes, polymers and nanoparticles. We used cationic lipids and polymers, wit...

Mechanisms and strategies for effective delivery of antisense and siRNA oligonucleotides

The potential use of antisense and siRNA oligonucleotides as therapeutic agents has elicited a great deal of interest. However, a major issue for oligonucleotide-based therapeutics involves effective intracellular delivery of the active molecules. In this Survey and Summary, we review recent reports on delivery strategies, including conjugates of oligonucleotides with various ligands, as well a...