Guinot P, Temsamani J
Hybridon Europe, Paris, France.
Pathol Biol (Paris). 1998 May;46(5):347-54.
The use of antisense oligonucleotides as therapeutic agents has generated considerable enthusiasm in the research and medical community. Oligonucleotides inhibit gene expression by binding to their target nucleic acid with high specificity and selectivity. The field of antisense technology has progressed enormously. Major progress has been accomplished in the synthesis and manufacturing of modified oligonucleotides. Numerous studies have demonstrated the ability of antisense oligonucleotides to modulate gene expression, in such diverse fields as infectious diseases, cancer, and inflammation. More than a dozen of clinical trials using antisense oligonucleotides have been initiated during the last three years or so. The insights gained through these ongoing clinical trials has opened the pathway to the design of more advanced chemistries which have improved safety profile and efficacy.
将反义寡核苷酸用作治疗剂已在研究和医学界引发了相当大的热情。寡核苷酸通过与靶核酸高特异性和选择性结合来抑制基因表达。反义技术领域取得了巨大进展。在修饰寡核苷酸的合成和制造方面已取得重大进展。大量研究已证明反义寡核苷酸在传染病、癌症和炎症等不同领域调节基因表达的能力。在过去三年左右的时间里，已经启动了十几项使用反义寡核苷酸的临床试验。通过这些正在进行的临床试验获得的见解为设计更先进的化学结构开辟了道路，这些化学结构具有更好的安全性和疗效。
