Lipid-based systems for the intracellular delivery of genetic drugs.

摘要: Summary Currently available delivery systems for genetic drugs have limited utility systemic applications. Cationic liposome/ plasmid DNA or oligonucleotide complexes are rapidly cleared from circulation, and the highest levels of activity observed infirst pass' organs, such as lungs, spleen liver. Engineered viruses can generate an immune response, which compromises transfection resulting subsequent injec- tions lack target specificity. A carrier, accumu- late at sites diseases infections, inflammations tumours, has to be a small, neutral highly serum-stable particle, is not readily recognized by fixed free macrophages reticuloendothelial system (RES). This review summarizes lipid-based technologies nucleic acid-based introduces new class carrier systems, solve, least in part, conflicting demands circulation longevity intracellular delivery. Plasmid oligonucleotides entrapped into lipid particles that contain small amounts positively charged stabilized presence polyethylene glycol (PEG) coating. These carriers protect degradation nucleases, on average 70 nm diameter, achieve long lifetimes capable transfect- ing cells.