abstract |
PROBLEM TO BE SOLVED: To provide an oligonucleotide analog having a modified subunit-to-subunit bond and / or a terminal group. The compounds of the present invention address these issues and provide improvements beyond existing antisense molecules in the art. Modification of the subunit-to-subunit binding and / or end portion of the oligonucleotide analog (eg, morpholino oligonucleotide) to the 5'and / or 3'end provides an antisense oligomer with excellent properties. For example, in certain embodiments, the disclosed oligomers have enhanced cell delivery, potency, and / or tissue distribution compared to other oligonucleotide analogs, and / or are efficiently delivered to the target organ. Can be done. These superior properties provide a favorable therapeutic index, reduce clinical administration and lower product costs. [Selection diagram] None |