Zhou Chenguang, Yue Zhang, Lee L James, Lee Robert J
Division of Pharmaceutics, College of Pharmacy, The Ohio State University, Columbus, OH 43210, USA.
Ther Deliv. 2012 Jun;3(6):715-23. doi: 10.4155/tde.12.47.
Lack of safe and efficient delivery of siRNA remains the greatest hurdle for the therapeutic application of siRNA. This article reports synthesis and evaluation of novel lipoidal amine-based nanocarrier (LANC) formulations for siRNA delivery.
Physicochemical properties were analyzed for LANC formulations. siRNA delivery efficiency of LANC-siRNA complexes was determined using a luciferase reporter gene assay. Cytotoxicity of the LANC-siRNA complexes was measured by the MTS assay. Finally, cellular uptake and cytoplasmic release of siRNA were analyzed using flow cytometry.
The LANC formulation facilitated siRNA uptake and release into the cytoplasm, mediating significant luciferase knockdown (70% inhibition).
缺乏安全有效的小干扰RNA（siRNA）递送方式仍然是siRNA治疗应用面临的最大障碍。本文报道了用于siRNA递送的新型脂质胺基纳米载体（LANC）制剂的合成与评估。
分析了LANC制剂的物理化学性质。使用荧光素酶报告基因测定法确定LANC-siRNA复合物的siRNA递送效率。通过MTS测定法测量LANC-siRNA复合物的细胞毒性。最后，使用流式细胞术分析siRNA的细胞摄取和细胞质释放。
LANC制剂促进了siRNA摄取并释放到细胞质中，介导了显著的荧光素酶敲低（70%抑制）。
