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 |
- 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
-
Effects of Eugenol on the Central Nervous System: Its Possible Application to Treatment of Alzheimers Disease, Depression, and Parkinsons Disease
Current Bioactive Compounds Subject Index to Volume 4
Current Drug Targets Glucocorticoids: Structure, Signaling and Molecular Mechanisms in the Treatment of Diabetic Retinopathy and Diabetic Macular Edema
Current Molecular Medicine Protein Tyrosine Phosphatase as Potential Therapeutic Target in various Disorders
Current Molecular Pharmacology Complement: An Inflammatory Pathway Fulfilling Multiple Roles at the Interface of Innate Immunity and Development
Current Drug Targets - Inflammation & Allergy Inflammatory Cellular Phenotypes and Molecular Mechanisms of Glucocorticoid Resistance in Patients with Bronchial Asthma
Anti-Inflammatory & Anti-Allergy Agents in Medicinal Chemistry Nano-Delivery in Pediatric Tumors: Looking Back, Moving Forward
Anti-Cancer Agents in Medicinal Chemistry AIDS Related Viruses, their Association with Leukemia, and Raf Signaling
Current HIV Research Antioxidative Effects of Rhodiola Genus: Phytochemistry and Pharmacological Mechanisms against the Diseases
Current Topics in Medicinal Chemistry Antibiotic or Anti-inflammatory Agent? The Double-Edged Sword of Tetracyclines
Anti-Inflammatory & Anti-Allergy Agents in Medicinal Chemistry Aliphatic and Aromatic Oxidations, Epoxidation and S-Oxidation of Prodrugs that Yield Active Drug Metabolites
Current Medicinal Chemistry Subcellular Injuries in Alzheimer's Disease
CNS & Neurological Disorders - Drug Targets Candidiasis and HIV-Protease Inhibitors: The Expected and the Unexpected
Current Medicinal Chemistry - Immunology, Endocrine & Metabolic Agents Scorpion Toxin Polyptides as Therapeutic Agents: An Overview
Protein & Peptide Letters Nitric Oxide and Dietary Factors: Part III Minerals, Vitamins and Other Dietary and Lifestyle Factors
Vascular Disease Prevention (Discontinued) Redox-Driven Events in the Human Immunodeficiency Virus Type 1 (HIV-1) Infection and their Clinical Implications
Current HIV Research Infantile Functional Gastrointestinal Disorders and Maternal Psychological Status: A Narrative Review
Current Pediatric Reviews The Clinical Trials of Mesenchymal Stem Cell Therapy in Skin Diseases: An Update and Concise Review
Current Stem Cell Research & Therapy Plasticity and Maintenance of Hematopoietic Stem Cells During Development
Recent Patents on Biotechnology Safflor Protected Rat Cerebral Ischemia-Reperfusion Injury Through Inhibiting the Expression of NF-kB and IL-1β
Neuroscience and Biomedical Engineering (Discontinued)