COMPOSITIONS FOR TARGETED DELIVERY OF SIRNA

Disclosed is a composition for delivering an oligonucleotide to a liver cell in vivo. The composition comprises: an oligonucleotide covalently linked to a hydrophobic group having at least 20 carbon atoms; a reversibly masked and targeted amphipathic membrane active polyamine comprising a terpolymer...

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Bibliographische Detailangaben
Hauptverfasser:	HADWIGER, PHILIPP, THUER, WILMA, ROZEMA, DAVID B, WAKEFIELD, DARREN H, VALIS, LINDA CHRISTINE, MOHR, PETER, HOFFMANN, TORSTEN, KITAS, ERIC A, LEWIS, DAVID L
Format:	Patent
Sprache:	eng
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	Disclosed is a composition for delivering an oligonucleotide to a liver cell in vivo. The composition comprises: an oligonucleotide covalently linked to a hydrophobic group having at least 20 carbon atoms; a reversibly masked and targeted amphipathic membrane active polyamine comprising a terpolymer synthesized from amine-containing monomers, lower hydrophobic group-containing monomers, and higher hydrophobic group-containing monomers to which a plurality of galactose derivatives and PEG groups are individually linked to said membrane active polyamine via pH labile disubstituted maleamic bonds and wherein cleavage of said pH labile disubstituted maleamic bonds yields amine groups thereby generating a membrane active polyamine; and wherein the oligonucleotide is not covalently linked to the amphipathic membrane active polyamine.