We have previously introduced the use of the biomaterial chitosan to form chitosan/siRNA nanoparticles for gene silencing protocols. This present study shows that the physicochemical properties (size, zeta potential, morphology and complex stability) and in vitro gene silencing of chitosan/siRNA nanoparticles are strongly dependent on chitosan molecular weight (Mw) and degree of deacetylation (DD). High Mw and DD chitosan resulted in the formation of discrete stable nanoparticles approximately 200 nm in size. Chitosan/siRNA formulations (N:P 50) prepared with low Mw (approximately 10 kDa) showed almost no knockdown of endogenous enhanced green fluorescent protein (EGFP) in H1299 human lung carcinoma cells, whereas those prepared from higher Mw (64.8-170 kDa) and DD (approximately 80%) showed greater gene silencing ranging between 45% and 65%. The highest gene silencing efficiency (80%) was achieved using chitosan/siRNA nanoparticles at N:P 150 using higher Mw (114 and 170 kDa) and DD (84%) that correlated with formation of stable nanoparticles of approximately 200 nm. In conclusion, this work confirms the application of chitosan as a non-viral carrier for siRNA and the importance of polymeric properties for the optimisation of gene silencing using chitosan/siRNA nanoparticles.

译文

我们之前已经介绍了使用生物材料壳聚糖形成壳聚糖/siRNA纳米颗粒的基因沉默方案。本研究表明,壳聚糖/siRNA纳米颗粒的理化性质 (大小,zeta电位,形态和复合物稳定性) 和体外基因沉默强烈依赖于壳聚糖分子量 (Mw) 和脱乙酰度 (DD)。高Mw和DD壳聚糖导致形成尺寸约200 nm的离散稳定纳米颗粒。低Mw (约10 kDa) 制备的壳聚糖/siRNA制剂 (N:P 50) 在H1299人肺癌细胞中几乎没有内源性增强绿色荧光蛋白 (EGFP) 的敲除,而由较高Mw (64.8-170 kDa) 和DD (约80%) 制备的那些显示出45% 和65% 之间更大的基因沉默。使用与形成约200纳米的稳定纳米颗粒相关的较高Mw (114和170 kDa) 和DD (84%),在N:P 150使用壳聚糖/siRNA纳米颗粒获得最高的基因沉默效率 (80%)。总之,这项工作证实了壳聚糖作为siRNA的非病毒载体的应用,以及聚合物特性对于使用壳聚糖/siRNA纳米颗粒优化基因沉默的重要性。

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