Demeneix B, Behr J, Boussif O, Zanta MA, Abdallah B, Remy J
Laboratoire de Physiologie Générale et Comparée, U.R.A.90 CNRS, Museum National d'Histoire Naturelle, F-75231, Paris Cedex 5, France
Adv Drug Deliv Rev. 1998 Mar 2;30(1-3):85-95. doi: 10.1016/s0169-409x(97)00109-9.
It is an obvious and basic principle that to be efficient, gene therapy requires effective gene transfer followed by adequate gene expression. However, getting DNA, a pro-drug, into the cell and into the nucleus, remains a crucially limiting factor. Even recombinant viral methods still show poor performances in clinical situations and non-viral methods are considered classically to be of yet lower efficiency. Here, we consider the mode of action, the nature of the complexes formed with DNA and the transfection potentials of two categories of inert, cationic vectors, the lipospermines and polyethylenimine. Both are among the best vectors currently available for in vitro work. Moreover, polyethylenimine is proving to be a versatile and effective carrier for different in vivo situations, especially for delivering genes into the mammalian brain.
一个明显且基本的原则是,为了实现高效,基因治疗需要有效的基因传递,随后是充分的基因表达。然而,将作为前体药物的DNA导入细胞并进入细胞核,仍然是一个关键的限制因素。即使是重组病毒方法在临床情况下仍表现不佳,而非病毒方法传统上被认为效率更低。在此,我们考虑两类惰性阳离子载体——脂精胺和聚乙烯亚胺的作用方式、与DNA形成的复合物的性质以及转染潜力。这两类载体都是目前体外研究中最好的载体之一。此外,聚乙烯亚胺已被证明是一种适用于不同体内情况的通用且有效的载体,尤其是用于将基因导入哺乳动物大脑。