Phosphorodiamidate morpholino oligomers: favorable properties for sequence-specific gene inactivation.
The phosphorodiamidate morpholino oligomers (PMOs) are a distinct class of oligonucleotide analogs. They bind to RNA and efficiently interfere with gene expression in a sequence-specific manner. Their non-ionic character combined with resistance to degradation in the body offer efficacy with minimal toxicity. Hence, the PMOs are emerging as an excellent approach to therapeutics that can be rationally designed based on target gene sequence data.
Iversen
P L
PL
AVI BioPharma Inc, Corvallis, OR 97333, USA. piversen@avibio.com
D000818:Animals; D020868:Gene Silencing; D015894:Genome, Human; D006801:Humans; D009025:Morpholines / Q000737:chemistry / Q000493:pharmacokinetics / Q000627:therapeutic use; D016376:Oligonucleotides, Antisense / Q000737:chemistry / Q000235:genetics / Q000493:pharmacokinetics / Q000627:therapeutic use; D013873:Thionucleotides / Q000737:chemistry / Q000235:genetics / Q000493:pharmacokinetics / Q000627:therapeutic use
D009025:Morpholines; D016376:Oligonucleotides, Antisense; D013873:Thionucleotides