阳离子α-环糊精:聚乙二醇聚轮烷用于 siRNA 递送。
Cationic α-cyclodextrin:poly(ethylene glycol) polyrotaxanes for siRNA delivery.
机构信息
Department of Chemistry, Purdue University, 560 Oval Drive, West Lafayette, Indiana 47907, USA.
出版信息
Mol Pharm. 2013 Apr 1;10(4):1299-305. doi: 10.1021/mp300449t. Epub 2013 Mar 13.
Abstract
RNA interference has broad therapeutic potential due to its high specificity and ability to potentially evade drug resistance. Three cationic α-cyclodextrin:poly(ethylene glycol) polyrotaxanes derived from polymer axles of different sizes (MW 2,000, 3,400, and 10,000) have been synthesized for delivering siRNA. These polyrotaxanes are able to condense siRNA into positively charged particles that are <200 nm in diameter, enabling their facile internalization into mammalian cells. The cationic polyrotaxanes display cytotoxicity profiles that are >10(2)-fold lower than the commercial standard bPEI and gene silencing efficiencies that are comparable to those of both Lipofectamine 2000 and bPEI. Our findings suggest that the cationic polyrotaxanes display a size-activity relationship, wherein the higher molecular weight polyrotaxanes (PEG3,400 and 10,000) are able to condense and deliver siRNA better than the lower molecular weight material (PEG2,000).
摘要
RNA 干扰因其高度特异性和潜在的抗药性规避能力而具有广泛的治疗潜力。三种阳离子α-环糊精：聚乙二醇聚轮烷是由不同大小的聚合物轴（MW2000、3400 和 10000）衍生而来的，用于递送 siRNA。这些聚轮烷能够将 siRNA 凝聚成带正电荷的颗粒，直径小于 200nm，使其易于进入哺乳动物细胞。阳离子聚轮烷的细胞毒性谱比商业标准 bPEI 低 10^2 倍以上，基因沉默效率与 Lipofectamine 2000 和 bPEI 相当。我们的研究结果表明，阳离子聚轮烷显示出大小与活性的关系，其中高分子量聚轮烷（PEG3400 和 10000）比低分子量材料（PEG2000）更能有效地凝聚和递送 siRNA。
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