Phosphorodiamidate morpholino oligomers: favorable properties for sequence-specific gene inactivation.

PL Iversen - Current opinion in molecular therapeutics, 2001 - europepmc.org
PL Iversen
Current opinion in molecular therapeutics, 2001europepmc.org
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.
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.
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