Vater Axel, Klussmann Sven
NOXXON Pharma AG, Max-Dohrn Strasse 8-10, D-10589 Berlin, Germany.
Curr Opin Drug Discov Devel. 2003 Mar;6(2):253-61.
Single-stranded mirror-image oligonucleotides, which are highly resistant to nuclease degradation and are capable of tightly and specifically binding to protein targets to inhibit their function, have been developed as potential therapeutic agents. The scientific discoveries that led to the development of the Spiegelmer technology are described in this review, along with recent preclinical developments of the first therapeutic Spiegelmers.
单链镜像寡核苷酸对核酸酶降解具有高度抗性,能够紧密且特异性地结合蛋白质靶点以抑制其功能,已被开发为潜在的治疗药物。本综述介绍了促成Spiegelmer技术发展的科学发现,以及首批治疗性Spiegelmer的近期临床前进展。