| 主权项 |
1. A sphingolipid-polyalkylamine-oligonucleotide compound having general formula I: wherein R1 is a branched or linear C7-C24 alkyl, alkenyl or polyenyl; R2, R3 and R4 each independently is hydrogen, C1-C4 alkyl, a branched or linear polyalkylamine or derivative thereof, or an oligonucleotide; R3′ is hydrogen or C1-C4 alkyl; A2, A3 and A4 each independently is present or absent but if present is one of C(O), C(O)NHX, C(O)NHR5X, C(O)R5X, C(O)R5C(O)X, R5X or R5OC(O)X; R5 is a branched or linear C1-C20 alkyl chain optionally substituted with one or more heteroatoms; X is present or absent but if present is S, P, O or NH; at least one of R2, R3 or R4 is a branched or linear polyalkylamine or derivative thereof; at least one of R2, R3 or R4 is an oligonucleotide; or a salt of such compound; for use in treating or preventing a disease or disorder in a subject; and with the proviso that the disease or disorder is other than cancer. |
