Glossary
Morpholino (PMO)
An uncharged oligo analogue that blocks its target by steric hindrance rather than cutting.
A morpholino, or phosphorodiamidate morpholino oligomer, replaces the natural sugar phosphate backbone with a neutral morpholine and phosphorodiamidate structure. Because it carries no charge and is highly stable, it works by physically blocking its target rather than recruiting an enzyme.
Morpholinos are well suited to splice switching applications, including approved treatments for Duchenne muscular dystrophy.
Related terms
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