Gewirtz Alan M
Division of Hematology/Oncology, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania 19104-6061, USA.
J Clin Invest. 2007 Dec;117(12):3612-4. doi: 10.1172/JCI34274.
Small molecules and antibodies have revolutionized the treatment of malignant diseases and appear promising for the treatment of many others. Nonetheless, there are many candidate therapeutic targets that are not amenable to attack by the current generation of targeted therapies, and in a small but growing number of patients, resistance to initially successful treatments evolves. This Review Series on the medicinal promise of posttranscriptional gene silencing with small interfering RNA and other molecules capable of inducing RNA interference (RNAi) is motivated by the hypothesis that effectors of RNAi can be developed into effective drugs for treating malignancies as well as many other types of disease. As this Review Series points out, there is still much to do, but many in the field now hope that the time has finally arrived when "antisense" therapies will finally come of age and fulfill their promise as the magic bullets of the 21st century.
小分子和抗体已经彻底改变了恶性疾病的治疗方式,并且在治疗许多其他疾病方面也显示出前景。然而,仍有许多候选治疗靶点无法被当前一代靶向疗法所攻克,而且在一小部分但数量不断增加的患者中,对最初成功的治疗产生了耐药性。关于小分子干扰RNA和其他能够诱导RNA干扰(RNAi)的分子在转录后基因沉默方面的药用前景的本综述系列,其动机基于这样一种假设,即RNAi效应物可以被开发成治疗恶性肿瘤以及许多其他类型疾病的有效药物。正如本综述系列所指出的,仍有许多工作要做,但该领域的许多人现在希望,“反义”疗法最终走向成熟并兑现其作为21世纪神奇子弹的承诺的时刻终于到来了。