SHORT INTEFERING RNA (siRNA) AS A NOVEL THERAPEUTIC
Identifieur interne : 000756 ( Istex/Corpus ); précédent : 000755; suivant : 000757SHORT INTEFERING RNA (siRNA) AS A NOVEL THERAPEUTIC
Auteurs : Peter N. Pushparaj ; Alirio J. MelendezSource :
- Clinical and Experimental Pharmacology and Physiology [ 0305-1870 ] ; 2006-05.
English descriptors
- Teeft :
- Acad, Acute liver failure, Adult mice, Angiogenesis, Antisense, Antisense oligonucleotides, Antisense strands, Authors journal compilation, Bace1, Biol, Blackwell publishing asia, Caenorhabditis elegans, Cancer gene, Cell culture, Cell death, Clinical trials, Dominant gene disorders, Dsrna, Effective delivery systems, Elegans, Expression vectors, Functional genomics, Fusion protein, Gene, Gene expression, Gene function, Growth factor, Human cells, Human diseases, Human genome, Human virus, Intense research efforts, Interference rnas, Larger dsrnas, Major obstacle, Mammalian cells, Mirna molecules, Mirnas, Mouse model, Mrna, Multiple genes, Multiple sirnas, Mutation, Natl, Natl acad, Neurodegenerative disorders, Neuron, Nucleic, Nucleic acids, Ocular neovascularization, Pathway, Present review, Proc, Rna, Rnai, Rnai technologies, Rnai vectors, Sequence homology, Short interference, Short rnas, Sirna, Sirna delivery, Sirna therapeutics, Sirna therapy, Sirnas, Synthetic sirnas, Tail vein injections, Target mrna, Target tissues, Therapeutic intervention, Therapeutic purposes, Therapeutic strategy, Therapeutics, Various research groups, Vegf, Vegf receptor, Viral, Viral infections, Viral vectors, Vivo, Vivo delivery.
Abstract
1 RNA interference (RNAi) is a robust method of post‐transcriptional silencing of genes using double‐stranded RNA (dsRNA) with sequence homology driven specificity. The dsRNA can be between 21 and 23 nucleotides long: this is converted to small interfering RNA (siRNA), which then mediates gene silencing by degradation/blocking of translation of the target mRNA. 2 RNA interference provides a simple, fast and cost‐effective alternative to existing gene targeting approaches both in vitro and in vivo. The discovery of siRNAs that cause RNAi in mammalian cells opened the door to the therapeutic use of siRNAs. Highly intense research efforts are now aimed at developing siRNAs for therapeutic purposes. 3 Recent advances in the design and delivery of targeting molecules now allow efficient and highly specific gene silencing in mammalian systems. Synthetic siRNA libraries targeting thousands of mammalian genes are publicly available for high‐throughput genetic screens for target discovery and validation. Recent studies have demonstrated the clinical potential of aptly designed siRNAs in various types of viral infections, cancer and renal and neurodegenerative disorders. 4 The present review provides insight into the novel therapeutic strategies of siRNA technology, which is the latest development in nucleic acid‐based tools for knocking down gene expression, and its potential for silencing genes associated with various human diseases.
Url:
DOI: 10.1111/j.1440-1681.2006.04399.x
Links to Exploration step
ISTEX:69811272A7DC4A9B533E0A8A551493D3BA417829Le document en format XML
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The dsRNA can be between 21 and 23 nucleotides long: this is converted to small interfering RNA (siRNA), which then mediates gene silencing by degradation/blocking of translation of the target mRNA. 2 RNA interference provides a simple, fast and cost‐effective alternative to existing gene targeting approaches both in vitro and in vivo. The discovery of siRNAs that cause RNAi in mammalian cells opened the door to the therapeutic use of siRNAs. Highly intense research efforts are now aimed at developing siRNAs for therapeutic purposes. 3 Recent advances in the design and delivery of targeting molecules now allow efficient and highly specific gene silencing in mammalian systems. Synthetic siRNA libraries targeting thousands of mammalian genes are publicly available for high‐throughput genetic screens for target discovery and validation. Recent studies have demonstrated the clinical potential of aptly designed siRNAs in various types of viral infections, cancer and renal and neurodegenerative disorders. 4 The present review provides insight into the novel therapeutic strategies of siRNA technology, which is the latest development in nucleic acid‐based tools for knocking down gene expression, and its potential for silencing genes associated with various human diseases.</div></front></TEI><istex><corpusName>wiley</corpusName><keywords><teeft><json:string>sirna</json:string><json:string>sirnas</json:string><json:string>rnai</json:string><json:string>therapeutics</json:string><json:string>viral</json:string><json:string>rna</json:string><json:string>antisense</json:string><json:string>vegf</json:string><json:string>mrna</json:string><json:string>dsrna</json:string><json:string>mirnas</json:string><json:string>natl</json:string><json:string>acad</json:string><json:string>proc</json:string><json:string>mammalian 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<ref target="http://www.tei-c.org/">We use TEI</ref></desc></application></appInfo></encodingDesc><profileDesc><abstract xml:lang="en" style="main"><head>SUMMARY</head><p><list xml:id="l1" style="custom"><item n="1"><p>RNA interference (RNAi) is a robust method of post‐transcriptional silencing of genes using double‐stranded RNA (dsRNA) with sequence homology driven specificity. The dsRNA can be between 21 and 23 nucleotides long: this is converted to small interfering RNA (siRNA), which then mediates gene silencing by degradation/blocking of translation of the target mRNA.</p></item><item n="2"><p>RNA interference provides a simple, fast and cost‐effective alternative to existing gene targeting approaches both <hi rend="italic">in vitro</hi> and <hi rend="italic">in vivo</hi>. The discovery of siRNAs that cause RNAi in mammalian cells opened the door to the therapeutic use of siRNAs. Highly intense research efforts are now aimed at developing siRNAs for therapeutic purposes.</p></item><item n="3"><p>Recent advances in the design and delivery of targeting molecules now allow efficient and highly specific gene silencing in mammalian systems. Synthetic siRNA libraries targeting thousands of mammalian genes are publicly available for high‐throughput genetic screens for target discovery and validation. Recent studies have demonstrated the clinical potential of aptly designed siRNAs in various types of viral infections, cancer and renal and neurodegenerative disorders.</p></item><item n="4"><p>The present review provides insight into the novel therapeutic strategies of siRNA technology, which is the latest development in nucleic acid‐based tools for knocking down gene expression, and its potential for silencing genes associated with various human diseases.</p></item></list></p></abstract><textClass><keywords xml:lang="en"><term xml:id="k1">cancer</term><term xml:id="k2">gene knockdown</term><term xml:id="k3">mammalian cells</term><term xml:id="k4">neurodegenerative disorders</term><term xml:id="k5">renal disorders</term><term xml:id="k6">RNA interference</term><term xml:id="k7">short interference RNA (siRNA) library</term><term xml:id="k8">short interference RNA (siRNA) technology</term><term xml:id="k9">viral infection</term></keywords><keywords rend="tocHeading1"><term>Frontiers in Research Reviews: Cutting‐Edge Molecular Approaches to Therapeutics</term></keywords></textClass><langUsage><language ident="en"></language></langUsage></profileDesc><revisionDesc><change when="2019-12-20" who="#istex" xml:id="pub2tei">formatting</change></revisionDesc></teiHeader></istex:fulltextTEI><json:item><extension>txt</extension><original>false</original><mimetype>text/plain</mimetype><uri>https://api.istex.fr/ark:/67375/WNG-VH1SL6HW-S/fulltext.txt</uri></json:item></fulltext><metadata><istex:metadataXml wicri:clean="Wiley, elements deleted: body"><istex:xmlDeclaration>version="1.0" encoding="UTF-8" standalone="yes"</istex:xmlDeclaration><istex:document><component version="2.0" type="serialArticle" xml:lang="en"><header><publicationMeta level="product"><publisherInfo><publisherName>Blackwell Publishing Asia</publisherName><publisherLoc>Melbourne, Australia</publisherLoc></publisherInfo><doi origin="wiley" registered="yes">10.1111/(ISSN)1440-1681</doi><issn type="print">0305-1870</issn><issn type="electronic">1440-1681</issn><idGroup><id type="product" value="CEP"></id><id type="publisherDivision" value="ST"></id></idGroup><titleGroup><title type="main" sort="CLINICAL EXPERIMENTAL PHARMACOLOGY PHYSIOLOGY">Clinical and Experimental Pharmacology and Physiology</title></titleGroup></publicationMeta><publicationMeta level="part" position="05005"><doi origin="wiley">10.1111/cep.2006.33.issue-5-6</doi><numberingGroup><numbering type="journalVolume" number="33">33</numbering><numbering type="journalIssue">5‐6</numbering></numberingGroup><coverDate startDate="2006-05">May/June 2006</coverDate></publicationMeta><publicationMeta level="unit" type="reviewArticle" position="15" status="forIssue"><doi origin="wiley">10.1111/j.1440-1681.2006.04399.x</doi><idGroup><id type="unit" value="CEP4399"></id></idGroup><countGroup><count type="pageTotal" number="7"></count></countGroup><titleGroup><title type="tocHeading1">Frontiers in Research Reviews: Cutting‐Edge Molecular Approaches to Therapeutics</title></titleGroup><eventGroup><event type="firstOnline" date="2006-05-12"></event><event type="publishedOnlineFinalForm" date="2006-05-12"></event><event type="xmlConverted" agent="Converter:BPG_TO_WML3G version:2.3.2 mode:FullText source:FullText result:FullText" date="2010-03-02"></event><event type="xmlConverted" agent="Converter:WILEY_ML3G_TO_WILEY_ML3GV2 version:3.8.8" date="2014-01-09"></event><event type="xmlConverted" agent="Converter:WML3G_To_WML3G version:4.1.7 mode:FullText,remove_FC" date="2014-10-16"></event></eventGroup><numberingGroup><numbering type="pageFirst" number="504">504</numbering><numbering type="pageLast" number="510">510</numbering></numberingGroup><correspondenceTo> Dr Alirio J Melendez, Department of Physiology, Yong Loo Lin School of Medicine, 2 Medical Drive, MD9 #01–05, Singapore 117597. Email: <email>phsmraj@nus.edu.sg</email></correspondenceTo><linkGroup><link type="toTypesetVersion" href="file:CEP.CEP4399.pdf"></link></linkGroup></publicationMeta><contentMeta><unparsedEditorialHistory>Received 14 October 2005; revision 29 November 2005; accepted 18 January 2006.</unparsedEditorialHistory><countGroup><count type="figureTotal" number="1"></count><count type="tableTotal" number="1"></count><count type="formulaTotal" number="0"></count><count type="referenceTotal" number="84"></count><count type="wordTotal" number="4088"></count></countGroup><titleGroup><title type="main">SHORT INTEFERING RNA (siRNA) AS A NOVEL THERAPEUTIC</title><title type="shortAuthors"><i>PN Pushparaj and AJ Melendez</i></title><title type="short"><i>siRNA therapeutics</i></title></titleGroup><creators><creator creatorRole="author" xml:id="cr1" affiliationRef="#aff-1-1"><personName><givenNames>Peter N</givenNames><familyName>Pushparaj</familyName></personName></creator><creator creatorRole="author" xml:id="cr2" affiliationRef="#aff-1-1"><personName><givenNames>Alirio J</givenNames><familyName>Melendez</familyName></personName></creator></creators><affiliationGroup><affiliation xml:id="aff-1-1" countryCode="SG"><unparsedAffiliation>Department of Physiology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore</unparsedAffiliation></affiliation></affiliationGroup><keywordGroup xml:lang="en"><keyword xml:id="k1">cancer</keyword><keyword xml:id="k2">gene knockdown</keyword><keyword xml:id="k3">mammalian cells</keyword><keyword xml:id="k4">neurodegenerative disorders</keyword><keyword xml:id="k5">renal disorders</keyword><keyword xml:id="k6">RNA interference</keyword><keyword xml:id="k7">short interference RNA (siRNA) library</keyword><keyword xml:id="k8">short interference RNA (siRNA) technology</keyword><keyword xml:id="k9">viral infection</keyword></keywordGroup><abstractGroup><abstract type="main" xml:lang="en"><title type="main">SUMMARY</title><p><list xml:id="l1" style="custom"><listItem><label>1</label><p>RNA interference (RNAi) is a robust method of post‐transcriptional silencing of genes using double‐stranded RNA (dsRNA) with sequence homology driven specificity. The dsRNA can be between 21 and 23 nucleotides long: this is converted to small interfering RNA (siRNA), which then mediates gene silencing by degradation/blocking of translation of the target mRNA.</p></listItem><listItem><label>2</label><p>RNA interference provides a simple, fast and cost‐effective alternative to existing gene targeting approaches both <i>in vitro</i> and <i>in vivo</i>. The discovery of siRNAs that cause RNAi in mammalian cells opened the door to the therapeutic use of siRNAs. Highly intense research efforts are now aimed at developing siRNAs for therapeutic purposes.</p></listItem><listItem><label>3</label><p>Recent advances in the design and delivery of targeting molecules now allow efficient and highly specific gene silencing in mammalian systems. Synthetic siRNA libraries targeting thousands of mammalian genes are publicly available for high‐throughput genetic screens for target discovery and validation. Recent studies have demonstrated the clinical potential of aptly designed siRNAs in various types of viral infections, cancer and renal and neurodegenerative disorders.</p></listItem><listItem><label>4</label><p>The present review provides insight into the novel therapeutic strategies of siRNA technology, which is the latest development in nucleic acid‐based tools for knocking down gene expression, and its potential for silencing genes associated with various human diseases.</p></listItem></list></p></abstract></abstractGroup></contentMeta><noteGroup><note xml:id="n-fnt-1" numbered="no"><p>This paper has been peer reviewed.</p></note></noteGroup></header></component></istex:document></istex:metadataXml><mods version="3.6"><titleInfo lang="en"><title>SHORT INTEFERING RNA (siRNA) AS A NOVEL THERAPEUTIC</title></titleInfo><titleInfo type="abbreviated" lang="en"><title>siRNA therapeutics</title></titleInfo><titleInfo type="alternative" contentType="CDATA" lang="en"><title>SHORT INTEFERING RNA (siRNA) AS A NOVEL THERAPEUTIC</title></titleInfo><name type="personal"><namePart type="given">Peter N</namePart><namePart type="family">Pushparaj</namePart><affiliation>Department of Physiology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Alirio J</namePart><namePart type="family">Melendez</namePart><affiliation>Department of Physiology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore</affiliation><role><roleTerm type="text">author</roleTerm></role></name><typeOfResource>text</typeOfResource><genre type="review-article" displayLabel="reviewArticle" authority="ISTEX" authorityURI="https://content-type.data.istex.fr" valueURI="https://content-type.data.istex.fr/ark:/67375/XTP-L5L7X3NF-P">review-article</genre><originInfo><publisher>Blackwell Publishing Asia</publisher><place><placeTerm type="text">Melbourne, Australia</placeTerm></place><dateIssued encoding="w3cdtf">2006-05</dateIssued><edition>Received 14 October 2005; revision 29 November 2005; accepted 18 January 2006.</edition><copyrightDate encoding="w3cdtf">2006</copyrightDate></originInfo><language><languageTerm type="code" authority="rfc3066">en</languageTerm><languageTerm type="code" authority="iso639-2b">eng</languageTerm></language><physicalDescription><extent unit="figures">1</extent><extent unit="tables">1</extent><extent unit="formulas">0</extent><extent unit="references">84</extent><extent unit="words">4088</extent></physicalDescription><abstract lang="en">1 RNA interference (RNAi) is a robust method of post‐transcriptional silencing of genes using double‐stranded RNA (dsRNA) with sequence homology driven specificity. The dsRNA can be between 21 and 23 nucleotides long: this is converted to small interfering RNA (siRNA), which then mediates gene silencing by degradation/blocking of translation of the target mRNA. 2 RNA interference provides a simple, fast and cost‐effective alternative to existing gene targeting approaches both in vitro and in vivo. The discovery of siRNAs that cause RNAi in mammalian cells opened the door to the therapeutic use of siRNAs. Highly intense research efforts are now aimed at developing siRNAs for therapeutic purposes. 3 Recent advances in the design and delivery of targeting molecules now allow efficient and highly specific gene silencing in mammalian systems. Synthetic siRNA libraries targeting thousands of mammalian genes are publicly available for high‐throughput genetic screens for target discovery and validation. Recent studies have demonstrated the clinical potential of aptly designed siRNAs in various types of viral infections, cancer and renal and neurodegenerative disorders. 4 The present review provides insight into the novel therapeutic strategies of siRNA technology, which is the latest development in nucleic acid‐based tools for knocking down gene expression, and its potential for silencing genes associated with various human diseases.</abstract><subject lang="en"><genre>keywords</genre><topic>cancer</topic><topic>gene knockdown</topic><topic>mammalian cells</topic><topic>neurodegenerative disorders</topic><topic>renal disorders</topic><topic>RNA interference</topic><topic>short interference RNA (siRNA) library</topic><topic>short interference RNA (siRNA) technology</topic><topic>viral infection</topic></subject><relatedItem type="host"><titleInfo><title>Clinical and Experimental Pharmacology and Physiology</title></titleInfo><genre type="journal" authority="ISTEX" authorityURI="https://publication-type.data.istex.fr" valueURI="https://publication-type.data.istex.fr/ark:/67375/JMC-0GLKJH51-B">journal</genre><identifier type="ISSN">0305-1870</identifier><identifier type="eISSN">1440-1681</identifier><identifier type="DOI">10.1111/(ISSN)1440-1681</identifier><identifier type="PublisherID">CEP</identifier><part><date>2006</date><detail type="volume"><caption>vol.</caption><number>33</number></detail><detail type="issue"><caption>no.</caption><number>5‐6</number></detail><extent unit="pages"><start>504</start><end>510</end><total>7</total></extent></part></relatedItem><relatedItem type="references" displayLabel="cit1"><titleInfo><title>The rest is silence</title></titleInfo><name type="personal"><namePart type="given">E</namePart><namePart type="family">Bernstein</namePart><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">AM</namePart><namePart type="family">Denli</namePart><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">GJ</namePart><namePart type="family">Hannon</namePart><role><roleTerm type="text">author</roleTerm></role></name><genre>journal-article</genre><note type="citation/reference">Bernstein E, Denli AM, Hannon GJ. 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