abstract |
An oligonucleotide analogue having a modified intersubunit bond and / or terminal group is provided. The compounds of the present invention address these problems and provide improvements over existing antisense molecules in the art. Modification of the intersubunit linkage and / or conjugation of the oligonucleotide moiety (eg, morpholino oligonucleotide) at the terminal portion to the 5 ′ and / or 3 ′ end results in an antisense oligomer with superior properties. For example, in certain embodiments, the disclosed oligomers have enhanced cell delivery, potency, and / or tissue distribution as compared to other oligonucleotide analogs, and / or allow for efficient delivery to target organs. Can be. These superior properties provide favorable therapeutic indices, reduce clinical dosing and lower product costs. [Selection diagram] None |