论文部分内容阅读
基因治疗通过基因载体将治病基因导入病患的特异细胞以治疗心血管、神经系统疾病和癌症等。寻找安全高效的非病毒基因载体一直是基因治疗以及生物材料领域中的前沿课题。聚酰胺-胺型(PAMAM)树枝状高分子作为一类三维的、结构高度有序的新型载体,由于具有安全性好、易于修饰、携带外源基因容量大等特点,已经引起了广泛的关注。但是另一方面,合成步骤相对繁琐、后期产物纯化困难以及转染效率相对较低等问题限制了这类载体的进一步发展。本文结合本课题组的研究情况,针对如何提高PAMAM的转染效率以及增强其基因传递的靶向性等相关问题,对近几年在PAMAM树枝状分子修饰改性方面所做的一些有意义的工作进行了综述,并对前景进行了展望。
Gene therapy through gene delivery of gene therapy to patients with specific cells to treat cardiovascular, neurological diseases and cancer. Looking for safe and efficient non-viral gene vectors has long been a frontier issue in the field of gene therapy and biomaterials. Polyamidoamine (PAMAM) dendrimers, as a new type of three-dimensional, highly organized and ordered carrier, have attracted a great deal of attention due to their high safety, ease of modification and high capacity of carrying foreign genes . On the other hand, however, the relative complexity of the synthesis steps, the difficulties in the purification of the final product, and the relatively low transfection efficiency have limited the further development of such vectors. Based on the research of our group, this article aims at how to improve the transfection efficiency of PAMAM and enhance its targeting of gene delivery, and so on. In this paper, we make some meaningful contributions to modification of PAMAM dendrimers in recent years The work is reviewed and the outlook is forecasted.