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Updated: May 15, 2026

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System
Published on: October 29, 2019
siRNA therapeutics in the treatment of diseases
Salar Bakhtiyari1, Karimeh Haghani, Gholam Basati
1Department of Clinical Biochemistry, Faculty of Medicine, Ilam University of Medical Sciences, Ilam, Iran. basati-gh@medilam.ac.ir
Abstract:
siRNAs are a class of dsRNAs, 21-23 nucleotides in length, which are able to silence their target genes through enzymatic cleavage of target mRNA. The sequence-specific gene-silencing by siRNA can be used as a new therapeutic approach for treatment of a variety of diseases that are incurable by conventional drugs. Many efforts have been made to overcome the problems related to delivery, stability, off-target gene silencing and immunostimulatory effects of siRNA. Different studies have carried out done to improve in vitro and in vivo delivery of naked or formulated siRNAs. In this review, different aspects of using siRNA as a new class of drugs will be discussed.
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