Non‐viral gene transfer: Applications in developmental biology and gene therapy
Non‐viral gene transfer: Applications in developmental biology and gene therapy
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DOI：
10.1111/j.1768-322x.1995.tb00937.x
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发表时间：
1995
影响因子：
2.7
通讯作者：
Bassima Abdallah;Laurent Sachs;B. A. Demeneix
中科院分区：
文献类型：
--
作者：
Bassima Abdallah;Laurent Sachs;B. A. Demeneix
非病毒基因转移方法的主要局限性是其体内效率相对较低。然而，可以采取许多方法来提高它们的性能，无论是研究发育过程中的基因功能还是将这些技术用于基因治疗。描述了我们特别参与开发的三种非病毒递送系统：阳离子脂质，双十八烷基氨基甘氨酰精胺（DOGS），阳离子聚合物聚乙烯亚胺（PEI）和游离DNA。这些方法中的每一种在不同的体内情况下的应用：使用狗的胚胎和发育中的哺乳动物的神经系统;最近的应用PEI的神经系统;以及如何裸DNA可以采用不同的肌肉和大脑的胚胎。基于在每个组织中用最合适的载体系统评估的荧光素酶报告基因表达，比较相对效率。最后，对构建安全与效率相结合的复合向量的前景进行了简要的思考。
Summary— The main limitation of non‐viral gene transfer methods is their relatively low efficiency in vivo. However, a number of approaches can be taken to improve their performances, whether the aim is studying gene function during development or employing these techniques for gene therapy. Three non‐viral delivery systems that we have been particularly involved in in developing are described: the cationic lipid, dioctadecylamidoglycylspermine (DOGS), the cationic polymer polyethylenimine (PEI) and free DNA. The application of each of these methods to different in vivo situations is presented: the use of DOGS for transfecting embryos and the developing mammalian nervous system; the recent application of PEI to the nervous system; and how naked DNA can be employed for transfecting different muscles and brain. The relative efficiencies are compared on the basis of luciferase reporter gene expression assessed in each tissue with the most appropriate vector system. Finally, the perspectives for constructing composite vectors combining safety and efficiency are considered briefly.