Title:siRNA Therapeutics: Future Promise for Neurodegenerative Diseases
Volume: 19
Issue: 11
Author(s): Atefeh Amiri, George Barreto, Thozhukat Sathyapalan and Amirhossein Sahebkar*
Affiliation:
- Biotechnology Research Center, Pharmaceutical Technology Institute, Mashhad University of Medical Sciences, Mashhad,Iran
Keywords:
Central nervous system, neurodegenerative disorders, siRNA, RNAi, delivery system, antisense technology.
Abstract: 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.
