| 主权项 |
1. A method of inhibiting expression of a target gene in a subject, the method comprising administering to the subject a composition comprising:
a small interfering ribonucleic acid (siRNA) in a pharmaceutically acceptable delivery vehicle, wherein the delivery vehicle comprises a lipid-cationic peptoid conjugate of the formula:
L-X—[N(CH2CH2NH2)CH2(C═O)—N(CH2CH2R)CH2(C═O)—N(CH2CH2R)CH2(C═O)]3—NH2 and positional isomers where
L is selected from a non-sterol lipid moiety comprising at least one fatty alkyl or alkenyl chain between about 8 and 24 carbon atoms in length and a sterol moiety, wherein when L is a non-sterol lipid moiety, the peptoid conjugate is a lipitoid and when L is a sterol moiety, the peptoid conjugate is a cholesteroid;each group R is independently selected from alkyl, aminoalkyl, and aralkyl, andX is selected from the group consisting of a direct bond, an oligopeptide, a substantially linear alkyl chain from 2 to about 30 bonds in length, and a substantially linear chain from 2 to about 30 bonds in length consisting of alkyl bonds and one or more linkages selected from the group consisting of ester, amide, carbonate, carbamate, disulfide, peptide, and ether; wherein the delivery vehicle comprises a combination of lipitoid and cholesteroid peptoid conjugates; and wherein one strand of the siRNA duplex has a nucleotide sequence comprised in an mRNA derived from the target gene. |
