反义核酸药物的研究现状_李学军.pdfVIP

2000 31 2 # 115 # * 李学军( , 100083) 反义药物以其合理设计药物的可能性和精确的特异性广泛吸引了人们的注 意, 但反义药物的研究 非如人们最初预想的那样简单本文从其特异性, 稳定性, 透过靶细胞的能力, 作用强度, 活性判定, 给药途径, 安全性和毒性, 生产成本等诸方 面对反义药物的研究现状, 现存问题进行了综述相信伴随这些问题的解决, 反义药 物很可能成为药典的 一部分, 给疾病的治疗带来益处 反义寡核苷酸; 反义药物; 基因治疗 Q786; R966 Current Status of Antisense Drugs LI Xue-Jun ( D ep art ment of Phar macology , Bei- j ing M ed ical Un iv ers ity , Beijing 100083) Abstract The antisense as first imagined as the therapeutic drug at the end of 1970. After 20 years the antisense drugs have been hitting into the market from laboratory and clinical research. Antisense captures general attention ith their promise of rational drug design and exquisite specification. But antisense drugs arefar more difficult to pro- duce than ere originally anticipated. T his article revie ed the current situation and present questions involved in their specificity, stability, potency, toxicology , intracel- lular delivery, administration routes and costs of manufacture, etc. It is likely that the antisense drugs ill be a part of the pharmacopoeia in the future and benefit for treat- ment of human diseases ith the solution of present questions. Key words Antisense oligonucleotides; Antisense drugs; Gene therapy ( antisense oligonucleotide, ODNs ) mRNA DNA , , RNase H mR- [ 1] NA, , :) ) ) ; ) ) ) mRNA; ) ) ) Watson-Crick ; : ( 1) RNase H RNA ; (2) DNA , ODNs70 , Fomivirsen , ODNs , , , , , , , , [2] , , * (RF93-63# 82) # 116 # 2000 31 2 : ODNs , RNA , , RNA ODNs, ODNs; , , [2] ; , ODN , , , 17 , , , ODN 15~ 20