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.
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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 |
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