Akhtar S, Juliano R L
Pharmaceutical Sciences Institute, Aston University, Aston Triangle, Birmingham B4 7ET, UK.
Trends Cell Biol. 1992 May;2(5):139-44. doi: 10.1016/0962-8924(92)90100-2.
Antisense oligonucleotides with sequences complementary to a given genetic target can enter cells in sufficient quantities to selectively inhibit gene expression. Thus, they have a potential therapeutic use in preventing undesirable gene expression in diseases such as cancer and AIDS. However, it is remarkable that these molecules, which have high molecular weights and are often charged, gain entry to cells at all. In this article, we review the possible mechanisms by which oligonucleotides enter cells and their subsequent intracellular fates. We also discuss current approaches for improving cellular uptake and delivery of antisense nucleic acids to their intended targets.
具有与特定基因靶点互补序列的反义寡核苷酸能够以足够的量进入细胞，从而选择性地抑制基因表达。因此，它们在预防癌症和艾滋病等疾病中不良基因表达方面具有潜在的治疗用途。然而，值得注意的是，这些分子量高且通常带电荷的分子竟然能够进入细胞。在本文中，我们综述了寡核苷酸进入细胞的可能机制及其随后在细胞内的命运。我们还讨论了目前改善细胞摄取以及将反义核酸递送至其靶标的方法。
