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
-
The Epithelial-Stromal Interaction as a Potential Target for Breast Cancer Treatment
Clinical Cancer Drugs Pharmacotherapy of Schizophrenia: The Past, Present and Future
Current Drug Therapy Current Concepts and Controversies in the Use of Antenatal Corticosteroid Therapy for Prevention of Neonatal Morbidities
Current Women`s Health Reviews Identification of Novel Scaffolds for IκB Kinase Beta Inhibitor via a High Throughput Screening TR-FRET Assay
Combinatorial Chemistry & High Throughput Screening Anti-Inflammatory/Analgesic
Current Bioactive Compounds Pain-Relief Medication and Risk of Fractures
Current Drug Safety Challenges in the Management of Paediatric Febrile Neutropenia
Current Pediatric Reviews Analgesic and Anti-Inflammatory Properties of Arylnitroalkenes
Inflammation & Allergy - Drug Targets (Discontinued) The Gastrin-Releasing Peptide Receptor (GRPR) in the Spinal Cord as a Novel Pharmacological Target
Current Neuropharmacology Conference Report (42nd Annual Meeting of the Italian Society of Neurology, 22-25 October 2011, Turin, Italy)
CNS & Neurological Disorders - Drug Targets Targeting ADAM12 in Human Disease: Head, Body or Tail?
Current Pharmaceutical Design High Throughput Bioassays Using Nanoparticles
Combinatorial Chemistry & High Throughput Screening Nutrigenetics, Metabolic Syndrome Risk and Personalized Nutrition
Current Vascular Pharmacology Natural and Synthetic Furanocoumarins as Treatment for Vitiligo and Psoriasis
Current Drug Therapy Editorial (Thematic Issue: Advances in Research on Pharmacological Targets for Pain Relief)
Current Neuropharmacology Current Status of Ethosomes and Elastic Liposomes in Dermal and Transdermal Drug Delivery
Current Pharmaceutical Design Patent Selections
Recent Patents on Drug Delivery & Formulation Cellular Repressor of E1A-stimulated Genes, A New Potential Therapeutic Target for Atherosclerosis
Current Drug Targets G-protein Dependent Platelet Signaling - Perspectives for Therapy
Current Drug Targets Fibromyalgia and Related Syndromes Characterised by Stress Intolerance and Pain Hypersensitivity: Do We Need a New Nosology?
Current Rheumatology Reviews