Abstract
A novel class of therapeutic agents based on nucleic acids has emerged and shown very promising pre-clinical results, named small interfering RNAs (siRNAs) and microRNAs (miRNAs). siRNAs are small RNA duplexes capable of silencing undesired (i.e., mutant, exogenous or aberrant) gene expression with high specificity through a mechanism known as RNA interference. These agents have called special attention to neuroscience since they have been used to experimentally treat a variety of neurological diseases with distinct etiologies such as viral, prions, genetic disorders and others. siRNAs have also been used in other scenarios as: drug-receptor blockage, inhibition of pain signaling and regulation of behavior. Although in a very initial stage, miRNAs also promise novel therapeutic approaches. In this review article we intend to introduce clinicians and researchers to the novel field of si- and miRNA-mediated gene silencing strategies, its history, use in cell and animal models, delivery methods, current status and possible applications in future clinical practice.
Central Nervous System Agents in Medicinal Chemistry
Title: Novel Nucleic Acid-Based Agents: siRNAs and miRNAs
Volume: 8 Issue: 2
Author(s): Tiago Campos Pereira and Iscia Lopes-Cendes
Affiliation:
Abstract: A novel class of therapeutic agents based on nucleic acids has emerged and shown very promising pre-clinical results, named small interfering RNAs (siRNAs) and microRNAs (miRNAs). siRNAs are small RNA duplexes capable of silencing undesired (i.e., mutant, exogenous or aberrant) gene expression with high specificity through a mechanism known as RNA interference. These agents have called special attention to neuroscience since they have been used to experimentally treat a variety of neurological diseases with distinct etiologies such as viral, prions, genetic disorders and others. siRNAs have also been used in other scenarios as: drug-receptor blockage, inhibition of pain signaling and regulation of behavior. Although in a very initial stage, miRNAs also promise novel therapeutic approaches. In this review article we intend to introduce clinicians and researchers to the novel field of si- and miRNA-mediated gene silencing strategies, its history, use in cell and animal models, delivery methods, current status and possible applications in future clinical practice.
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Cite this article as:
Pereira Campos Tiago and Lopes-Cendes Iscia, Novel Nucleic Acid-Based Agents: siRNAs and miRNAs, Central Nervous System Agents in Medicinal Chemistry 2008; 8 (2) . https://dx.doi.org/10.2174/187152408784533932
DOI https://dx.doi.org/10.2174/187152408784533932 |
Print ISSN 1871-5249 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-6166 |
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