Stephens Alick C, Rivers Rodney P A
Academic Department of Paediatrics, Imperial College London, St Mary's Campus, Norfolk Place, Paddington, London, W2 1PG, UK.
Curr Opin Mol Ther. 2003 Apr;5(2):118-22.
The sequencing of the human genome has highlighted some of the genes that are of importance in disease states. This has provided opportunities for the development of new therapeutics to target a wide range of human diseases. These new drugs are intended to be highly specific; antisense oligonucleotides (ONs) are one such class of new drugs. ONs are short pieces of DNA which hybridize to a specific target mRNA blocking its translation to protein, thereby inhibiting the action of the gene. Several genes known to be of importance in the regulation of apoptosis, cell growth, metastasis and angiogenesis provide a tantalizing prospect for the development of anticancer agents. The phosphorothioate antisense ONs are the current choice for antisense therapy. This article reviews the current strategies for antisense targets in cancer therapy.
人类基因组测序突出了一些在疾病状态中具有重要意义的基因。这为开发针对多种人类疾病的新疗法提供了机会。这些新药旨在具有高度特异性;反义寡核苷酸(ONs)就是这类新药中的一种。ONs是短片段DNA,可与特定的靶mRNA杂交,阻止其翻译成蛋白质,从而抑制基因的作用。已知在细胞凋亡、细胞生长、转移和血管生成调节中具有重要意义的几个基因,为抗癌药物的开发提供了诱人的前景。硫代磷酸酯反义ONs是目前反义治疗的选择。本文综述了癌症治疗中反义靶点的当前策略。