Guide for Morpholino Users: Toward Therapeutics

Morpholino oligos are uncharged molecules for blocking sites on RNA. They are specific, soluble, non-toxic, stable, and effective antisense reagents suitable for development as therapeutics and currently in clinical trials. They are very versatile, targeting a wide range of RNA targets for outcomes such as blocking translation, modifying splicing of pre-mRNA, inhibiting miRNA maturation and activity, as well as less common biological targets and diagnostic applications. Solutions have been developed for delivery into a range of cultured cells, embryos and adult animals; with development of a non-toxic and effective system for systemic delivery, Morpholinos have potential for broad therapeutic development targeting pathogens and genetic disorders.