通过直接导入外源基因来治疗人类疾病的方法需要一种有效、安全并且可重复进行的载体,阳离子脂质体是基本满足这些条件的有限几种载体中的一员. 目前已经有十几种阳离子脂质体. 这些脂质体通过外周的电荷与DNA相结合,静电吸引形成复合物在与细胞膜相互作用后,通过细胞的内吞或融合作用使复合物进入细胞内,从而将外源基因导入细胞,这种DNA传递技术的有效性和安全性已经确立. 二例利用阳离子脂质体的人体基因治疗临床试验也已开始实施,将来会有更多的临床试验得到开展,阳离子脂质体在基因治疗领域有较好的前景.
Direct gene transfer for the treatment of human diseases requires a vector which can be administered efficiently, safely and repeatedly. Cationic liposomes represent one of the few examples that can meet these requirements. Currently, there are more than a dozen cationic liposome formulations. These liposomes bind and condense DNA spontaneously to form complexes with high affinity to cell membranes. Endocytosis of the complexes followed by disruption of the endosomal membrane appears to be the major mechanism of gene delivery. The effectiveness and safety of this DNA delivery method has been established in many studies. Two human gene therapy clinical trials using cationic liposomes have been conducted and more trials will be started in the near future. In the field of gene therapy, cationic liposome is struggling to meet expectations.
王瓞,林其谁.用于转基因的阳离子脂质体[J].生物化学与生物物理进展,1997,24(6):486-491
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