Iversen P L
AVI BioPharma Inc, Corvallis, OR 97333, USA.
Curr Opin Mol Ther. 2001 Jun;3(3):235-8.
The phosphorodiamidate morpholino oligomers (PMOs) are a distinct class of oligonucleotide analogs. They bind to RNA and efficiently interfere with gene expression in a sequence-specific manner. Their non-ionic character combined with resistance to degradation in the body offer efficacy with minimal toxicity. Hence, the PMOs are emerging as an excellent approach to therapeutics that can be rationally designed based on target gene sequence data.
磷酰二胺吗啉代寡聚物(PMOs)是一类独特的寡核苷酸类似物。它们与RNA结合,并以序列特异性方式有效干扰基因表达。其非离子特性以及在体内抗降解的能力使其具有高效且低毒的特点。因此,PMOs正成为一种基于靶基因序列数据可合理设计的优秀治疗方法。