Lv Hongtao, Zhang Shubiao, Wang Bing, Cui Shaohui, Yan Jie
Dalian Institute of Chemical Physics, Chinese Academy of Sciences, Dalian 116023, Liaoning, China.
J Control Release. 2006 Aug 10;114(1):100-9. doi: 10.1016/j.jconrel.2006.04.014. Epub 2006 May 13.
Gene therapy, as a promising therapeutics to treat genetic or acquired diseases, has achieved exciting development in the past two decades. Appropriate gene vectors can be crucial for gene transfer. Cationic lipids and polymers, the most important non-viral vectors, have many advantages over viral ones as non-immunogenic, easy to produce and not oncogenic. They hold the promise to replace viral vectors to be used in clinic. However, the toxicity is still an obstacle to the application of non-viral vectors to gene therapy. For overcoming the problem, many new cationic compounds have been developed. This article provides a review with respect to toxicity of cationic lipids and polymers in gene delivery. We evaluate the structural features of cationic compounds and summarize the relationship of toxicity and structure and hope to provide available suggestions on the development of these cationic compounds.
基因治疗作为一种治疗遗传或后天疾病的有前景的疗法,在过去二十年中取得了令人振奋的进展。合适的基因载体对于基因转移至关重要。阳离子脂质和聚合物作为最重要的非病毒载体,相对于病毒载体具有许多优势,如无免疫原性、易于生产且无致癌性。它们有望取代病毒载体用于临床。然而,毒性仍然是将非病毒载体应用于基因治疗的一个障碍。为了克服这个问题,人们已经开发了许多新型阳离子化合物。本文就阳离子脂质和聚合物在基因递送中的毒性进行综述。我们评估阳离子化合物的结构特征,总结毒性与结构的关系,希望能为这些阳离子化合物的开发提供有用的建议。