Sommer W, Heilig M
Beroendecentrum Syd och institutionen för klinisk neurovetenskap och allmänmedicin, Karolinska institutet, Huddinge.
Lakartidningen. 1999 Jan 27;96(4):348-54.
Recent advances in molecular biology have improved our understanding of basic pathophysiological mechanisms, and indicated possible strategies for their manipulation. The use of oligonucleotides occupies a prominent position among the emerging strategies, and has given rise to a new generation of pharmaceutical preparations now entering clinical usage. Antisense oligonucleotides are capable of blocking the expression of specific genes, thus permitting highly precise intervention in pathophysiology at the molecular level. Several clinical trials have recently produced highly promising results. The most likely applications of antisense agents are viral infections, malignancies, and chronic inflammatory disease. The article consists in a presentation of the antisense principle, discussion of its advantages and limitations, and a review of recent clinical developments and future prospects.
分子生物学的最新进展增进了我们对基本病理生理机制的理解，并指明了对其进行调控的可能策略。在新兴策略中，寡核苷酸的应用占据显著地位，并催生了新一代现已进入临床应用的药物制剂。反义寡核苷酸能够阻断特定基因的表达，从而在分子水平上对病理生理过程进行高度精确的干预。最近的几项临床试验已产生了非常有前景的结果。反义药物最有可能的应用领域是病毒感染、恶性肿瘤和慢性炎症性疾病。本文介绍了反义原理，讨论了其优缺点，并综述了近期的临床进展和未来前景。
