Department of Chemistry & Chemical Biology, Rutgers, The State University of New Jersey, Piscataway, NJ, USA.
J Am Chem Soc. 2013 Oct 23;135(42):15682-15685. doi: 10.1021/ja4071738. Epub 2013 Oct 11.
Achieving a controlled and reproducible means to direct stem cell differentiation is the single most critical concern scientists have been trying to address since the discovery of stem cells. In this regard, the use of small molecules and RNA interference offers unique advantages by targeting different cellular mechanisms. Our cyclodextrin-modified dendritic polyamine construct (termed DexAM) combines the unique properties of two distinct chemical moieties in a single delivery vehicle. DexAM is a single vehicle that not only solubilizes hydrophobic small molecules in physiological solutions but also forms complexes with siRNA molecules, making it an attractive delivery system for controlling stem cell differentiation. Herein, we report the synthesis and application of DexAM to simultaneously deliver hydrophobic small molecules and siRNA into neural stem cells to significantly enhance their neuronal differentiation.
实现对干细胞分化的可控和可重复的指导是自干细胞发现以来科学家们一直试图解决的最关键问题。在这方面，小分子和 RNA 干扰的使用通过靶向不同的细胞机制提供了独特的优势。我们的环糊精修饰的树枝状多胺构建体（称为 DexAM）将两种不同化学部分的独特性质结合在单个输送载体中。DexAM 不仅是一种单一载体，可在生理溶液中溶解疏水分子，还可与 siRNA 分子形成复合物，使其成为控制干细胞分化的有吸引力的输送系统。在这里，我们报告了 DexAM 的合成和应用，以将疏水分子和 siRNA 同时递送到神经干细胞中，从而显著增强其神经元分化。
