Brisson M, Huang L
Department of Pharmacology, University of Pittsburgh School of Medicine, PA 15261, USA.
Curr Opin Mol Ther. 1999 Apr;1(2):140-6.
The field of non-viral vector gene therapy has become increasingly successful due to the production of improved liposomes, polymers and other formulations. These novel vectors have increased the amount of DNA delivered to cells both in vitro and in vivo, and in turn, have increased gene expression. However, DNA transport to the nucleus still remains one of the largest problems in obtaining efficient, high levels of gene expression. This review addresses the reasons why the nucleus is a major barrier to transfected DNA and introduces the cytoplasmic expression system as a possible alternative to nuclear expression.
由于改进的脂质体、聚合物和其他制剂的产生,非病毒载体基因治疗领域已变得越来越成功。这些新型载体在体外和体内都增加了递送至细胞的DNA量,进而增加了基因表达。然而,DNA转运至细胞核仍然是实现高效、高水平基因表达的最大问题之一。本文综述探讨了细胞核成为转染DNA主要障碍背后的原因,并介绍了细胞质表达系统作为核表达的一种可能替代方案。