Sharma H W, Narayanan R
Division of Oncology, Roche Research Center, Hoffmann-La Roche Inc., Nutley, NJ 07110, USA.
Bioessays. 1995 Dec;17(12):1055-63. doi: 10.1002/bies.950171210.
Specific inhibition of gene expression by antisense agents provides the basis for rational drug discovery based on molecular targets. Due to the specificity of Watson-Crick base-pair hybridization, antisense oligodeoxynucleotides have been used extensively in attempts to inhibit gene expression in both in vitro and in vivo models. Analogues modified from normal phosphodiester oligodeoxynucleotides have entered clinical trials against diseases including AIDS and cancer. Although the precise mechanism of action of these drugs has not been clarified, these oligodeoxynucleotides offer considerable promise as novel molecular therapeutics. We review the recent attempts to harness the therapeutic potential of these oligodeoxynucleotides and appraise the near-term prospects for antisense technology.
反义药物对基因表达的特异性抑制为基于分子靶点的合理药物研发提供了基础。由于沃森-克里克碱基对杂交的特异性，反义寡脱氧核苷酸已被广泛用于尝试在体外和体内模型中抑制基因表达。从正常磷酸二酯寡脱氧核苷酸修饰而来的类似物已进入针对包括艾滋病和癌症在内的疾病的临床试验。尽管这些药物的确切作用机制尚未阐明，但这些寡脱氧核苷酸作为新型分子疗法具有相当大的前景。我们回顾了近期利用这些寡脱氧核苷酸治疗潜力的尝试，并评估了反义技术的近期前景。
