Cationic liposome-mediated gene transfer.
Cationic liposome-mediated gene transfer.
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作者：
Xiang Gao;Leaf Huang
用于治疗人类疾病的直接基因转移需要可以有效、安全和重复施用的载体。阳离子脂质体代表了能够满足这些要求的少数实例之一。目前，已经报道了十几种阳离子脂质体制剂。这些脂质体自发地结合并浓缩DNA，形成对细胞膜具有高亲和力的复合物。复合物的内吞作用，随后内体膜的破坏似乎是基因递送的主要机制。这种DNA递送方法的有效性和安全性已在许多研究中得到证实。基于这些结果，已经进行了两项使用阳离子脂质体的人类基因治疗临床试验，并将在不久的将来开始更多的试验。阳离子脂质体因其简单、高效、安全等特点，成为人类基因治疗的理想载体。
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, more than a dozen cationic liposome formulations have been reported. 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. Based on these results, two human gene therapy clinical trials using cationic liposomes have been conducted and more trials will be started in the near future. The simplicity, efficiency and safety features have rendered the cationic liposome an attractive vehicle for human gene therapy.