Thymidine-Methylene oxy-DMTr-Morpholino, cyanoethyl- N,N-diisopropyl di phosphoramidite

Phosphorodiamidate morpholinos (PMOs) are chemically modified oligonucleotides (ODNs) wherein the 2’-deoxyribonucleosides and phosphate linkages of canonical DNA are substituted with morpholino rings and phosphorodiamidate linkages respectively. They have been researched extensively in oligonucleotide therapeutics as potential steric blocking agents for the treatment of various genetic disorders. PMOs exhibit high hybridization affinity to complementary RNA, possess excellent enzymatic stability both in vitro and in vivo and elicit low immunogenicity leading to acceptable toxicokinetic profiles in mammalian models. The ability of PMOs to effectively function as steric blockers arises from their ability to specifically bind complementary RNA both in vitro and in vivo. Morpholinos block small (~25 base) regions of the base-pairing surfaces of ribonucleic acid (RNA).
Morpholinos are six-membered rings which may provide higher conformational rigidity when incorporated into an oligonucleotide (ODN) backbone.

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