非病毒性基因载体的合成研究

基因治疗是一种有效的治疗先天性遗传性疾病以及后天获得性疾病的手段。它通过激发细胞的生物活性或者抑制细胞非正常的功能来治疗或者预防疾病的发生,例如细胞的基因紊乱,细胞的无序增殖。目前基因治疗所面临的问题是缺乏有效的基因递送载体。基因载体主要分为病毒性基因载体和非病毒性基因载体。与病毒性基因载体相比,非病毒性基因载体具有毒性小、安全性高、易于制备、能够荷载分子量大的DNA等优点。本文综述了非病毒性基因载体的合成研究进展。

Research progress in synthesis of nonviral gene delivery vector

Gene therapy is regard as the most efficient method to treat many inheritable or acquired diseases by producing bioactive agents or preventing abnormal functions of the cells such as genetic disorder or uncontrollable proliferation of cells.However,the lack of effective vectors is a major problem for progress in human gene therapy due to several problems,such as their instability in body fluids,non-specificity to the target cells,degradation by enzymes and low transfection efficiency.The main types of vectors used in gene therapy are viral and non-viral vectors.Although viral vectors have higher transfection efficiency,they have fatal drawback such as mutagenesis,carcinogenesis,immune response and time consuming.Compared with viral vectors,nonviral vectors have several advantages including safer,easy to produce,the ability to transfer large DNA moleculars,and have been considered as potential alternatives.The research progress in synthesis of nonviral gene delivery vector was reviewed.