Gao X, Huang L
Department of Pharmacology, University of Pittsburgh School of Medicine, PA 15261, USA.
Gene Ther. 1995 Dec;2(10):710-22.
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.
用于治疗人类疾病的直接基因转移需要一种能够高效、安全且可重复给药的载体。阳离子脂质体是少数能够满足这些要求的例子之一。目前，已经报道了十几种阳离子脂质体制剂。这些脂质体能够自发地结合并浓缩DNA，形成与细胞膜具有高亲和力的复合物。复合物通过内吞作用进入细胞，随后内体膜破裂，这似乎是基因传递的主要机制。许多研究已经证实了这种DNA传递方法的有效性和安全性。基于这些结果，已经开展了两项使用阳离子脂质体的人类基因治疗临床试验，并且在不久的将来还会启动更多试验。阳离子脂质体的简单性、高效性和安全性使其成为人类基因治疗中一种有吸引力的载体。
