Micro- and nanoscale delivery systems for macromolecules: gene delivery

基因疗法——将基因插入个体细胞和组织以治疗疾病——最大的挑战之一是成功地将DNA送入细胞，然后将DNA表达出来。在我们寻找合适的材料来包装DNA的过程中，我们已经确定了一组被称为gemini表面活性剂的化合物，它们可以浓缩、保护和运输DNA到细胞中。利用这些带电化合物，我们开发了一种基于非病毒纳米粒子的技术，可以通过皮肤传递基因。非病毒传递系统相对于病毒传递系统具有显著的优势，因为它们更安全、更便宜且更容易制备。然而，它们的主要缺点是效率不高，这是本研究将解决的一个缺陷。

One of the biggest challenges in gene therapy -- inserting genes into an individual's cells and tissues to treat a disease -- is successfully delivering DNA into cells, followed by the DNA being expressed. In our search for suitable materials to package DNA, we have identified a group of compounds known as gemini surfactants that can condense, protect and transport DNA into cells. Using these electrically charged compounds, we have developed a non-viral nanoparticle-based technology to deliver genes through the skin. Non-viral delivery systems have significant advantages relative to viral systems, as they are safer, cheaper and easier to prepare. Their major disadvantage, however, is that they are not as efficient, a deficiency this proposed research will address.