Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups

摘要

H:/drnenvown/P rtlDC AR/5f355..doc-2/O4/213 The invention concerns an oligomer comprising a backbone, the backbone comprising a sequence of morpholino ring structures joined by intersubunit linkages, the intersubunit linkages joining a 3' -end of one morpholino ring structure to a 5' -end of an adjacent morpholino ring structure, wherein each morpholino ring structure is bound to a basepairing moiety, such that the oligomer can bind in a sequence - specific manner to a target nucleic acid. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects. 0 cHt HO, WO 2011/150408 PCT/US2011/038459 0 rNH Ff00