Novel low molecular weight cationic lipids for oligonucleotide delivery

NOVEL LOW MOLECULAR WEIGHT CATIONIC LIPIDS FOR OLIGONUCLEOTIDE DELIVERY The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the in...

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Hauptverfasser:	LIAO, Hongbiao, STANTON, Matthew G, BEUTNER, Gregory L, BUDZIK, Brian W
Format:	Patent
Sprache:	eng
Schlagworte:	ACYCLIC OR CARBOCYCLIC COMPOUNDS CHEMISTRY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY ORGANIC CHEMISTRY PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Beschreibung
Zusammenfassung:	NOVEL LOW MOLECULAR WEIGHT CATIONIC LIPIDS FOR OLIGONUCLEOTIDE DELIVERY The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.