Morille Marie, Passirani Catherine, Vonarbourg Arnaud, Clavreul Anne, Benoit Jean-Pierre
Inserm U646, Ingénierie de la Vectorisation Particulaire, Université d'Angers, 10, rue André Boquel, 49100 Angers, France.
Biomaterials. 2008 Aug-Sep;29(24-25):3477-96. doi: 10.1016/j.biomaterials.2008.04.036. Epub 2008 May 21.
Initially, gene therapy was viewed as an approach for treating hereditary diseases, but its potential role in the treatment of acquired diseases such as cancer is now widely recognized. The understanding of the molecular mechanisms involved in cancer and the development of nucleic acid delivery systems are two concepts that have led to this development. Systemic gene delivery systems are needed for therapeutic application to cells inaccessible by percutaneous injection and for multi-located tumor sites, i.e. metastases. Non-viral vectors based on the use of cationic lipids or polymers appear to have promising potential, given the problems of safety encountered with viral vectors. Using these non-viral vectors, the current challenge is to obtain a similarly effective transfection to viral ones. Based on the advantages and disadvantages of existing vectors and on the hurdles encountered with these carriers, the aim of this review is to describe the "perfect vector" for systemic gene therapy against cancer.
最初,基因治疗被视为一种治疗遗传性疾病的方法,但如今其在治疗诸如癌症等后天性疾病中的潜在作用已得到广泛认可。对癌症相关分子机制的理解以及核酸递送系统的发展是促成这一进展的两个概念。对于经皮注射难以触及的细胞以及多部位肿瘤(即转移灶)的治疗应用而言,需要全身性基因递送系统。鉴于病毒载体存在的安全问题,基于阳离子脂质或聚合物的非病毒载体似乎具有广阔的潜力。使用这些非病毒载体,当前面临的挑战是要获得与病毒载体同样有效的转染效果。基于现有载体的优缺点以及这些载体所面临的障碍,本综述的目的是描述用于全身性癌症基因治疗的“完美载体”。
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