Related Experiment Video
Updated: Aug 16, 2026

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
Published on: September 21, 2017
Pharmacokinetics and biodistribution of phosphorodiamidate morpholino antisense oligomers
Adams Amantana1, Patrick L Iversen
1Research and Development, AVI BioPharma Inc, Corvallis, OR 97333, USA. amantana@avibio.com
Abstract:
The concept of using antisense oligonucleotides to interfere with gene expression offers a new therapeutic strategy for the treatment of diseases resulting from overexpression or dysfunction of certain genes. Phosphorodiamidate morpholino oligomers (PMOs) represent a neutral class of antisense agents that interfere with target gene expression either by binding and sterically blocking the assembly of translation machinery, resulting in inhibition of translation, or by altering splicing of pre-mRNA. Studies in animal models and human clinical trials have demonstrated a high degree of functional bioavailability in several target organs. Preclinical and clinical studies have shown that PMOs demonstrate improved efficacy, excellent kinetic behavior, biological stability, and a good safety profile. We conclude from the emerging data that PMOs display advantageous pharmaceutical properties in comparison with other antisense strategies.
Related Concept Videos
Indirect-Acting Cholinergic Agonists: Pharmacokinetics
Reversible agents containing quaternary amines, such as neostigmine and edrophonium, are not easily absorbed orally because they are...
RNA Interference
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
Biopharmaceutics and Pharmacokinetics: Overview
Measurement of Bioavailability: Pharmacodynamic Methods
Pharmaceutical Alternatives: Polymorphic Form-Related and Particle Size-Related Therapeutic Nonequivalence
Pharmacogenetic Phenotypes: Alterations in Pharmacokinetics, Drug Targets and Biologic Milieu
