NOVEL LOW MOLECULAR WEIGHT CATIONIC LIPIDS FOR OLIGONUCLEOTIDE DELIVERY
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机译:新型低分子量阳离子脂质体,可用于寡核苷酸的输送
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摘要
The present invention provides 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 present invention to provide cationic lipid scaffolds which exhibit enhanced efficacy with lower liver toxicity as a result of lower lipid levels in the liver. The present invention uses low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerance of in vivo delivery of siRNAs.
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