Synthesis of AZT-based cationic lipids and in vitro evaluation of siRNA delivery

摘要

New types of cationic lipids based on AZT were evaluated for their biological effects in terms of their cytotoxicity and their cellular delivery of siRNA. After the success of the human genome project, nucleic acid-based therapies have become promising tools to expand the range of disease treatment options. The results of many conceptually simple nucleic acid-based therapies [antisense oligonucleotides, siRNAs (small interfering RNA), ribozymes] are very promising, yet it remains difficult to deliver therapeutic genes into specific cells, both safely and efficiently.