Mansouri Sania, Lavigne Patrick, Corsi Karin, Benderdour Mohamed, Beaumont Eric, Fernandes Julio C
Laboratoire de Recherche en Orthopédie, Hôpital du Sacré-Coeur, Montreal, Québec, Canada.
Eur J Pharm Biopharm. 2004 Jan;57(1):1-8. doi: 10.1016/s0939-6411(03)00155-3.
Currently, the major drawback of gene therapy is the gene transfection rate. The two main types of vectors that are used in gene therapy are based on viral or non-viral gene delivery systems. The viral gene delivery system shows a high transfection yield but it has many disadvantages, such as oncogenic effects and immunogenicity. However, cationic polymers, like chitosan, have potential for DNA complexation and may be useful as non-viral vectors for gene therapy applications. Chitosan is a natural non-toxic polysaccharide, it is biodegradable and biocompatible, and protects DNA against DNase degradation and leads to its condensation. The objective of this paper was to summarize the state of the art in gene therapy and particularly the use of chitosan to improve the transfection efficiency in vivo and in vitro.
目前,基因治疗的主要缺点是基因转染率。基因治疗中使用的两种主要载体类型基于病毒或非病毒基因递送系统。病毒基因递送系统显示出高转染率,但它有许多缺点,如致癌作用和免疫原性。然而,阳离子聚合物,如壳聚糖,具有与DNA复合的潜力,可能用作基因治疗应用的非病毒载体。壳聚糖是一种天然无毒多糖,可生物降解且具有生物相容性,能保护DNA免受DNase降解并使其凝聚。本文的目的是总结基因治疗的现状,特别是壳聚糖在提高体内和体外转染效率方面的应用。