Antisense oligonucleotide in the treatment of brain diseases:research advances / 国际药学研究杂志

Antisense oligonucleotide(ASON)has attracted a growing attention with the rapid development of biological tech?nology. ASON can be designed to target mRNA 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 specifici?ty 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.