成功的基因治療(gene therapy)都有一個良好的傳遞載體 。一般用於傳遞基因的載體可分為兩大類:病毒型載體(viral carrier)及非病毒型載體(non-viral carrier)。本研究目的在設計及合成一個低細胞毒性及生物可分解性的新樹枝狀陽離子型高分子載體，使其能讓帶負電的質體DNA 分子藉由靜電作用力自組裝的形成奈米大小的複合物，這樣的複合物就能使細胞以胞飲的方式進入到細胞內而達到治療效果。接著一方面評斷所合成材料性質是否適合處於人體內而且不讓人體造成負擔的載體，另一方面評估合成的載體是否能有效的保護質體 DNA。我們藉由以下實驗來評估其相關性質：pH 緩衝能力的分析、粒徑大小及表面電位分析、電泳分析、細胞毒性分析、以及轉染能力的分析。 The successful gene therapy has an ideal gene delivery vector. Two popular vectors for gene delivery under intensively studied are viral carriers and non viral ones. In non-viral vector, the most important condition is the low cytotoxicity and high transfection efficiency. The purpose of this study is to design and synthesize new cationic dendrimers with a low cell toxicity and high biodegradability. The new material can make negatively charged plasmid DNA by electrostatic forces to self-assembly of nano-sized complexes. This compound can make the endocytosis to achieve the therapeutic effect of intracellular. We use Acid – base titration, Zeta-potential, DNA gel retardation, MTT assay, and transfection efficiency to evaluate the novel gene vector.
