摘要
随着生物技术的快速发展,反义核酸药物倍受关注。反义寡核苷酸(ASON)通过作用于疾病相关靶m RNA,干扰基因表达发挥治疗作用,具有特异性高、毒副作用低等特点。基于脑靶向载体技术和化学修饰,ASON在脑部疾病的治疗领域显示了广阔前景。本文综述了ASON药物的研发现状,及其应用于阿尔茨海默病、帕金森病及脑肿瘤等常见脑部疾病治疗的研究进展。
Antisense oligonucleotide（ASON）has attracted a growing attention with the rapid development of biological technology. ASON can be designed to target m RNA in a sequence-specific manner to stop,alter,or induce particular disease-related gene functions. Thus,its drugs have emerged as a very promising new class of therapeutics at the molecular genetic level with high specificity and low toxicity. ASON has shown good application prospects in the treatment of brain diseases,by means of brain-targeted delivery and chemical modification. In this review,we summarize the latest development of ASON drugs and their applications on the treatment of Alzheimer disease,Parkinson′s disease and brain tumors.
出处
《国际药学研究杂志》
CSCD
北大核心
2017年第8期743-752,共10页
Journal of International Pharmaceutical Research
基金
国家自然科学基金资助项目(81573345)
北京市科技新星计划资助项目(Z171100001117095)
关键词
反义寡核苷酸
阿尔茨海默病
帕金森病
脑肿瘤
antisense oligonucleotide
Alzheimers disease
Parkinson＇s disease
brain tumors
