基因沉默
RNA干扰
医学
神经退行性变
生物信息学
人口
神经保护
肌萎缩侧索硬化
疾病
计算生物学
神经科学
小干扰RNA
药物发现
生物
作者
Atefeh Amiri,George E. Barreto,Thozhukat Sathyapalan,Amirhossein Sahebkar
出处
期刊:Current Neuropharmacology
[Bentham Science]
日期:2021-04-01
卷期号:19
被引量:1
标识
DOI:10.2174/1570159x19666210402104054
摘要
Neurodegenerative diseases (ND), as a group of central nervous system (CNS) disorders, are among the most prominent medical problems of the 21st century. They are often associated with considerable disability, motor dysfunction and dementia and are more common in the aged population. ND imposes a psychologic, economic and social burden on the patients and their families. Currently, there is no effective treatment for ND. Since many ND result from the gain of function of a mutant allele, small interference RNA (siRNA) can be a potential therapeutic agent for ND management. Based on the RNA interference (RNAi) approach, siRNA is a powerful tool for modulating gene expression through gene silencing. However, there are some obstacles in the clinical application of siRNA, including unfavorable immune response, off-target effects, instability of naked siRNA, nuclease susceptibility and a need to develop a suitable delivery system. Since there are some issues related to siRNA delivery routes, in this review, we focus on the application of siRNA in the management of ND treatment from 2000 to 2020.
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