![conference banner](/images/conference-banner.jpeg)
Abstract
RNA interference consists of a sequence specific post-transcriptional gene silencing phenomenon triggered by a double strand RNA molecule homologous to the silenced gene. The dsRNA is cleaved by DICER enzyme in small dsRNA pieces, named short interfering RNAs (siRNAs). These fragments are thereafter associated to RISC complex where the cleavage of target RNA occurs. The observation that siRNAs can trigger the RNA interference mechanism in mammalian cells represents a fundamental discovery that discloses new horizons in genetic researches in that theoretically each gene can be silenced. The relative simplicity by which active short interfering RNAs can be designed and synthesized explains their widespread use in basic and applied researches, even if appropriate controls that exclude offtarget effects are strictly required. The findings that siRNAs are active even when expressed in viral vectors open the possibility that they can be very soon used for gene therapy of several human diseases.
Keywords: rna interference, short interfering rnas (sirnas), sirnas design, sirnas testing, gene expression knock down, expression vectors, functional genomics, therapeutics
Current Pharmaceutical Biotechnology
Title: RNA-Based Drugs: From RNA Interference to Short Interfering RNAs
Volume: 5 Issue: 4
Author(s): L. Poliseno, A. Mercatanti, L. Citti and G. Rainaldi
Affiliation:
Keywords: rna interference, short interfering rnas (sirnas), sirnas design, sirnas testing, gene expression knock down, expression vectors, functional genomics, therapeutics
Abstract: RNA interference consists of a sequence specific post-transcriptional gene silencing phenomenon triggered by a double strand RNA molecule homologous to the silenced gene. The dsRNA is cleaved by DICER enzyme in small dsRNA pieces, named short interfering RNAs (siRNAs). These fragments are thereafter associated to RISC complex where the cleavage of target RNA occurs. The observation that siRNAs can trigger the RNA interference mechanism in mammalian cells represents a fundamental discovery that discloses new horizons in genetic researches in that theoretically each gene can be silenced. The relative simplicity by which active short interfering RNAs can be designed and synthesized explains their widespread use in basic and applied researches, even if appropriate controls that exclude offtarget effects are strictly required. The findings that siRNAs are active even when expressed in viral vectors open the possibility that they can be very soon used for gene therapy of several human diseases.
Export Options
About this article
Cite this article as:
Poliseno L., Mercatanti A., Citti L. and Rainaldi G., RNA-Based Drugs: From RNA Interference to Short Interfering RNAs, Current Pharmaceutical Biotechnology 2004; 5 (4) . https://dx.doi.org/10.2174/1389201043376797
DOI https://dx.doi.org/10.2174/1389201043376797 |
Print ISSN 1389-2010 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-4316 |
Call for Papers in Thematic Issues
Artificial Intelligence in Bioinformatics
Bioinformatics is an interdisciplinary field that analyzes and explores biological data. This field combines biology and information system. Artificial Intelligence (AI) has attracted great attention as it tries to replicate human intelligence. It has become common technology for analyzing and solving complex data and problems and encompasses sub-fields of machine ...read more
Latest Advancements in Biotherapeutics
The scope of this thematic issue is to comprehensively explore the rapidly evolving landscape of biotherapeutics, emphasizing breakthroughs in precision medicine. Encompassing diverse therapeutic modalities, the issue will delve into the latest developments in monoclonal antibodies, CRISPR/Cas gene editing, CAR-T cell therapies, and innovative drug delivery systems, such as nanoparticle-based ...read more
![Wayfinder Image](/images/wayfinder.jpg)
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
Related Articles
-
The Breath of Life: Recent Patents on Placental and Amnion Derived Cells for Treatment of Bronchopulmonary Dysplasia
Recent Patents on Regenerative Medicine Mitochondrial Calcium Signaling as a Therapeutic Target for Alzheimer’s Disease
Current Alzheimer Research Histamine and Histaminergic Receptors in Colorectal Cancer: From Basic Science to Evidence-based Medicine
Anti-Cancer Agents in Medicinal Chemistry Meet Our Editorial Board Member
Inflammation & Allergy - Drug Targets (Discontinued) Chemokines and Relevant microRNAs in the Atherogenic Process
Mini-Reviews in Medicinal Chemistry New Compounds Targeting the Androgen Receptor for Treatment of Advanced Prostate Cancer
Current Drug Targets Brain Inflammation, Cholesterol, and Glutamate as Interconnected Participants in the Pathology of Alzheimers Disease
Current Pharmaceutical Design Evaluation of High Risk HPV (HPV-16 and -18) RNA and Integration in Cervical Neoplasms in Systemic Lupus Erythematosus
Current Women`s Health Reviews Relevance of the Neuropeptide Y System in the Biology of Cancer Progression
Current Topics in Medicinal Chemistry Prevention of Oxidative Stress and Diseases by Antioxidant Supplementation
Medicinal Chemistry MicroRNA and Multiple Myeloma: from Laboratory Findings to Translational Therapeutic Approaches
Current Pharmaceutical Biotechnology Cannabinoids Receptors in COVID-19: Perpetrators and Victims
Current Medicinal Chemistry TNF-Related Apoptosis-Inducing Ligand (TRAIL) as a Pro-Apoptotic Signal Transducer with Cancer Therapeutic Potential
Current Pharmaceutical Design Modulation Effects of Piracetam and Ginkgo biloba on the Cognitive and Working Memory Functions: Psychometric Study
Current Psychopharmacology Immune and Metabolic Interactions of Human Erythrocytes: A Molecular Perspective
Endocrine, Metabolic & Immune Disorders - Drug Targets Potential of Modulating Wnt Signaling Pathway Toward the Development of Bone Anabolic Agent
Current Molecular Pharmacology N-Heterocyclic Dronic Acids: Applications and Synthesis
Mini-Reviews in Medicinal Chemistry Multiple Roles of Transient Receptor Potential (TRP) Channels in Inflammatory Conditions and Current Status of Drug Development
Current Topics in Medicinal Chemistry Endothelial Function: A Surrogate Endpoint in Cardiovascular Studies?
Current Pharmaceutical Design Platelets and Platelet Interaction with Progenitor Cells in Vascular Homeostasis and Inflammation
Current Vascular Pharmacology