Development and Clinical Applications of Nucleic Acid Therapeutics
Identifieur interne : 000B75 ( Istex/Corpus ); précédent : 000B74; suivant : 000B76Development and Clinical Applications of Nucleic Acid Therapeutics
Auteurs : Veenu Aishwarya ; Anna Kalota ; Alan M. GewirtzSource :
- Advances in Polymer Science [ 0065-3195 ]
Abstract
Abstract: In the past decade, our new understanding of post-transcriptional gene silencing (PTGS) has opened up new vistas that can help us to decipher the function of genes and thus to unravel the basic mechanistic principles and genetic networks in biology. This enormous potential of PTGS to selectively turn off genes either by antisense or RNA interference pathways has made a huge impact on research in basic science. Further, we can apply this knowledge and new set of rules in biomedical research for the development of more potent therapeutics. With a plethora of advantages of PTGS, there are still certain obstacles that need to be addressed to enable translation of this technology to the clinic. This article will focus on a brief history of the discovery and evolution of PTGS and its potential as a technology. We will also discuss the importance of target availability and some strategies for identification of the target sequence. Further, we will give an overview of some past experiences in the development of this technology in the clinic for cancer therapy, with a recent update on clinical development and trials on nucleic acid therapeutics.
Url:
DOI: 10.1007/12_2011_146
Links to Exploration step
ISTEX:269031B1C37D96CF8E22831ED2E9B38F2955F420Le document en format XML
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Further, we will give an overview of some past experiences in the development of this technology in the clinic for cancer therapy, with a recent update on clinical development and trials on nucleic acid therapeutics.</p></abstract><textClass ana="keyword"><keywords xml:lang="en"><term>Antisense</term><term>Cancer therapy</term><term>Clinical trials</term><term>Oligodeoxynucleotides</term><term>Post-transcriptional gene silencing</term><term>RNA interference</term><term>RNase H</term><term>siRNA</term></keywords></textClass><textClass ana="subject"><keywords scheme="book-subject-collection"><list><label>SUCO11644</label><item><term>Chemistry and Materials Science</term></item></list></keywords></textClass><textClass ana="subject"><keywords scheme="book-subject"><list><label>SCC</label><item><term type="Primary">Chemistry</term></item><label>SCC22008</label><item><term type="Secondary" subtype="priority-1">Polymer Sciences</term></item><label>SCC28000</label><item><term type="Secondary" subtype="priority-2">Medicinal Chemistry</term></item><label>SCF00008</label><item><term type="Secondary" subtype="priority-3">Pharmacy</term></item><label>SCL14005</label><item><term type="Secondary" subtype="priority-4">Biochemistry, general</term></item><label>SCC21010</label><item><term type="Secondary" subtype="priority-5">Electrochemistry</term></item></list></keywords></textClass><langUsage><language ident="EN"></language></langUsage></profileDesc></teiHeader></istex:fulltextTEI><json:item><extension>txt</extension><original>false</original><mimetype>text/plain</mimetype><uri>https://api.istex.fr/ark:/67375/HCB-MLX9QK2F-X/fulltext.txt</uri></json:item></fulltext><metadata><istex:metadataXml wicri:clean="corpus springer-ebooks not found" wicri:toSee="no header"><istex:xmlDeclaration>version="1.0" encoding="UTF-8"</istex:xmlDeclaration><istex:docType PUBLIC="-//Springer-Verlag//DTD A++ V2.4//EN" URI="http://devel.springer.de/A++/V2.4/DTD/A++V2.4.dtd" name="istex:docType"></istex:docType><istex:document><Publisher><PublisherInfo><PublisherName>Springer Berlin Heidelberg</PublisherName><PublisherLocation>Berlin, Heidelberg</PublisherLocation><PublisherImprintName>Springer</PublisherImprintName></PublisherInfo><Series><SeriesInfo ID="Series_1" SeriesType="Series" TocLevels="0"><SeriesID>12</SeriesID><SeriesPrintISSN>0065-3195</SeriesPrintISSN><SeriesElectronicISSN>1436-5030</SeriesElectronicISSN><SeriesTitle Language="En">Advances in Polymer Science</SeriesTitle><SeriesAbbreviatedTitle>Advs Polymer Science</SeriesAbbreviatedTitle></SeriesInfo><Book Language="En" OutputMedium="All"><BookInfo BookProductType="Reviews" ContainsESM="No" Language="En" MediaType="eBook" NumberingDepth="3" NumberingStyle="ContentOnly" OutputMedium="All" TocLevels="0"><BookID>978-3-642-30463-7</BookID><BookTitle>Nucleic Acid Drugs</BookTitle><BookVolumeNumber>249</BookVolumeNumber><BookSequenceNumber>249</BookSequenceNumber><BookDOI>10.1007/978-3-642-30463-7</BookDOI><BookTitleID>190514</BookTitleID><BookPrintISBN>978-3-642-30462-0</BookPrintISBN><BookElectronicISBN>978-3-642-30463-7</BookElectronicISBN><BookEdition>2012</BookEdition><BookChapterCount>6</BookChapterCount><BookCopyright><CopyrightHolderName>Springer-Verlag Berlin Heidelberg</CopyrightHolderName><CopyrightYear>2012</CopyrightYear></BookCopyright><BookSubjectGroup><BookSubject Code="SCC" Type="Primary">Chemistry</BookSubject><BookSubject Code="SCC22008" Priority="1" Type="Secondary">Polymer Sciences</BookSubject><BookSubject Code="SCC28000" Priority="2" Type="Secondary">Medicinal Chemistry</BookSubject><BookSubject Code="SCF00008" Priority="3" Type="Secondary">Pharmacy</BookSubject><BookSubject Code="SCL14005" Priority="4" Type="Secondary">Biochemistry, general</BookSubject><BookSubject Code="SCC21010" Priority="5" Type="Secondary">Electrochemistry</BookSubject><SubjectCollection Code="SUCO11644">Chemistry and Materials Science</SubjectCollection></BookSubjectGroup><BookContext><SeriesID>12</SeriesID></BookContext></BookInfo><BookHeader><EditorGroup><Editor AffiliationIDS="AffID1"><EditorName DisplayOrder="Western"><GivenName>Akira</GivenName><FamilyName>Murakami</FamilyName></EditorName><Contact><Email>akiram2@kit.ac.jp</Email></Contact></Editor><Affiliation ID="AffID1"><OrgDivision>Dept. Biomolecular Engineering</OrgDivision><OrgName>Kyoto Institute of Technology</OrgName><OrgAddress><Street>Matsugasaki</Street><City>Kyoto</City><Postcode>606-8585</Postcode><Country>Japan</Country></OrgAddress></Affiliation></EditorGroup></BookHeader><Chapter ID="b12_2011_146" Language="En" OutputMedium="All"><ChapterInfo ChapterType="OriginalPaper" ContainsESM="No" Language="En" NumberingDepth="3" NumberingStyle="ContentOnly" OutputMedium="All" TocLevels="2"><ChapterID>146</ChapterID><ChapterDOI>10.1007/12_2011_146</ChapterDOI><ChapterSequenceNumber>6</ChapterSequenceNumber><ChapterTitle Language="En">Development and Clinical Applications of Nucleic Acid Therapeutics</ChapterTitle><ChapterFirstPage>153</ChapterFirstPage><ChapterLastPage>176</ChapterLastPage><ChapterCopyright><CopyrightHolderName>Springer-Verlag Berlin Heidelberg</CopyrightHolderName><CopyrightYear>2011</CopyrightYear></ChapterCopyright><ChapterHistory><RegistrationDate><Year>2011</Year><Month>6</Month><Day>21</Day></RegistrationDate><OnlineDate><Year>2011</Year><Month>9</Month><Day>21</Day></OnlineDate></ChapterHistory><ChapterContext><SeriesID>12</SeriesID><BookID>978-3-642-30463-7</BookID><BookTitle>Nucleic Acid Drugs</BookTitle></ChapterContext></ChapterInfo><ChapterHeader><AuthorGroup><Author AffiliationIDS="Aff1" CorrespondingAffiliationID="Aff1"><AuthorName DisplayOrder="Western"><GivenName>Veenu</GivenName><FamilyName>Aishwarya</FamilyName></AuthorName><Contact><Email>veenu@mail.med.upenn.edu</Email></Contact></Author><Author AffiliationIDS="Aff1"><AuthorName DisplayOrder="Western"><GivenName>Anna</GivenName><FamilyName>Kalota</FamilyName></AuthorName></Author><Author AffiliationIDS="Aff1"><AuthorName DisplayOrder="Western"><GivenName>Alan</GivenName><GivenName>M.</GivenName><FamilyName>Gewirtz</FamilyName></AuthorName></Author><Affiliation ID="Aff1"><OrgDivision>Division of Hematology/Oncology, Department of Medicine</OrgDivision><OrgName>University of Pennsylvania School of Medicine</OrgName><OrgAddress><City>Philadelphia</City><State>PA</State><Country>USA</Country></OrgAddress></Affiliation></AuthorGroup><Abstract ID="Abs1" Language="En" OutputMedium="All"><Heading>Abstract</Heading><Para>In the past decade, our new understanding of post-transcriptional gene silencing (PTGS) has opened up new vistas that can help us to decipher the function of genes and thus to unravel the basic mechanistic principles and genetic networks in biology. This enormous potential of PTGS to selectively turn off genes either by antisense or RNA interference pathways has made a huge impact on research in basic science. Further, we can apply this knowledge and new set of rules in biomedical research for the development of more potent therapeutics. With a plethora of advantages of PTGS, there are still certain obstacles that need to be addressed to enable translation of this technology to the clinic. This article will focus on a brief history of the discovery and evolution of PTGS and its potential as a technology. We will also discuss the importance of target availability and some strategies for identification of the target sequence. Further, we will give an overview of some past experiences in the development of this technology in the clinic for cancer therapy, with a recent update on clinical development and trials on nucleic acid therapeutics.</Para></Abstract><KeywordGroup Language="En" OutputMedium="All"><Heading>Keywords</Heading><Keyword>Antisense</Keyword><Keyword>Cancer therapy</Keyword><Keyword>Clinical trials</Keyword><Keyword>Oligodeoxynucleotides</Keyword><Keyword>Post-transcriptional gene silencing</Keyword><Keyword>RNA interference</Keyword><Keyword>RNase H</Keyword><Keyword>siRNA</Keyword></KeywordGroup><ArticleNote Type="Misc"><SimplePara>Dedicated to the memory of our mentor and a visionary – Dr. Alan M. Gewirtz.</SimplePara></ArticleNote></ChapterHeader><NoBody></NoBody></Chapter></Book></Series></Publisher></istex:document></istex:metadataXml><mods version="3.6"><titleInfo lang="en"><title>Development and Clinical Applications of Nucleic Acid Therapeutics</title></titleInfo><titleInfo type="alternative" contentType="CDATA"><title>Development and Clinical Applications of Nucleic Acid Therapeutics</title></titleInfo><name type="personal" displayLabel="corresp"><namePart type="given">Veenu</namePart><namePart type="family">Aishwarya</namePart><affiliation>Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania School of Medicine, Philadelphia, PA, USA</affiliation><affiliation>E-mail: veenu@mail.med.upenn.edu</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Anna</namePart><namePart type="family">Kalota</namePart><affiliation>Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania School of Medicine, Philadelphia, PA, USA</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Alan</namePart><namePart type="given">M.</namePart><namePart type="family">Gewirtz</namePart><affiliation>Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania School of Medicine, Philadelphia, PA, USA</affiliation><role><roleTerm type="text">author</roleTerm></role></name><typeOfResource>text</typeOfResource><genre displayLabel="OriginalPaper" authority="ISTEX" authorityURI="https://content-type.data.istex.fr" type="research-article" valueURI="https://content-type.data.istex.fr/ark:/67375/XTP-1JC4F85T-7">research-article</genre><originInfo><publisher>Springer Berlin Heidelberg</publisher><place><placeTerm type="text">Berlin, Heidelberg</placeTerm></place><dateIssued encoding="w3cdtf">2011</dateIssued><copyrightDate encoding="w3cdtf">2011</copyrightDate></originInfo><language><languageTerm type="code" authority="rfc3066">en</languageTerm><languageTerm type="code" authority="iso639-2b">eng</languageTerm></language><abstract lang="en">Abstract: In the past decade, our new understanding of post-transcriptional gene silencing (PTGS) has opened up new vistas that can help us to decipher the function of genes and thus to unravel the basic mechanistic principles and genetic networks in biology. This enormous potential of PTGS to selectively turn off genes either by antisense or RNA interference pathways has made a huge impact on research in basic science. Further, we can apply this knowledge and new set of rules in biomedical research for the development of more potent therapeutics. With a plethora of advantages of PTGS, there are still certain obstacles that need to be addressed to enable translation of this technology to the clinic. This article will focus on a brief history of the discovery and evolution of PTGS and its potential as a technology. We will also discuss the importance of target availability and some strategies for identification of the target sequence. Further, we will give an overview of some past experiences in the development of this technology in the clinic for cancer therapy, with a recent update on clinical development and trials on nucleic acid therapeutics.</abstract><subject lang="en"><genre>Keywords</genre><topic>Antisense</topic><topic>Cancer therapy</topic><topic>Clinical trials</topic><topic>Oligodeoxynucleotides</topic><topic>Post-transcriptional gene silencing</topic><topic>RNA interference</topic><topic>RNase H</topic><topic>siRNA</topic></subject><relatedItem type="host"><titleInfo><title>Nucleic Acid Drugs</title></titleInfo><name type="personal"><namePart type="given">Akira</namePart><namePart type="family">Murakami</namePart><affiliation>Dept. Biomolecular Engineering, Kyoto Institute of Technology, Matsugasaki, 606-8585, Kyoto, Japan</affiliation><affiliation>E-mail: akiram2@kit.ac.jp</affiliation><role><roleTerm type="text">editor</roleTerm></role></name><genre type="book-series" authority="ISTEX" authorityURI="https://publication-type.data.istex.fr" valueURI="https://publication-type.data.istex.fr/ark:/67375/JMC-0G6R5W5T-Z">book-series</genre><originInfo><publisher>Springer</publisher><copyrightDate encoding="w3cdtf">2012</copyrightDate><issuance>monographic</issuance><edition>2012</edition></originInfo><subject><genre>Book-Subject-Collection</genre><topic authority="SpringerSubjectCodes" authorityURI="SUCO11644">Chemistry and Materials Science</topic></subject><subject><genre>Book-Subject-Group</genre><topic authority="SpringerSubjectCodes" authorityURI="SCC">Chemistry</topic><topic authority="SpringerSubjectCodes" authorityURI="SCC22008">Polymer Sciences</topic><topic authority="SpringerSubjectCodes" authorityURI="SCC28000">Medicinal Chemistry</topic><topic authority="SpringerSubjectCodes" authorityURI="SCF00008">Pharmacy</topic><topic authority="SpringerSubjectCodes" authorityURI="SCL14005">Biochemistry, general</topic><topic authority="SpringerSubjectCodes" authorityURI="SCC21010">Electrochemistry</topic></subject><identifier type="DOI">10.1007/978-3-642-30463-7</identifier><identifier type="ISBN">978-3-642-30462-0</identifier><identifier type="eISBN">978-3-642-30463-7</identifier><identifier type="ISSN">0065-3195</identifier><identifier type="eISSN">1436-5030</identifier><identifier type="BookTitleID">190514</identifier><identifier type="BookID">978-3-642-30463-7</identifier><identifier type="BookChapterCount">6</identifier><identifier type="BookVolumeNumber">249</identifier><identifier type="BookSequenceNumber">249</identifier><part><date>2012</date><detail type="volume"><number>249</number><caption>vol.</caption></detail><extent unit="pages"><start>153</start><end>176</end></extent></part><recordInfo><recordOrigin>Springer-Verlag Berlin Heidelberg, 2012</recordOrigin></recordInfo></relatedItem><relatedItem type="series"><titleInfo><title>Advances in Polymer Science</title></titleInfo><originInfo><publisher>Springer</publisher><copyrightDate encoding="w3cdtf">2012</copyrightDate><issuance>serial</issuance></originInfo><identifier type="ISSN">0065-3195</identifier><identifier type="eISSN">1436-5030</identifier><identifier type="SeriesID">12</identifier><recordInfo><recordOrigin>Springer-Verlag Berlin Heidelberg, 2012</recordOrigin></recordInfo></relatedItem><identifier type="istex">269031B1C37D96CF8E22831ED2E9B38F2955F420</identifier><identifier type="ark">ark:/67375/HCB-MLX9QK2F-X</identifier><identifier type="DOI">10.1007/12_2011_146</identifier><identifier type="ChapterID">146</identifier><identifier type="ChapterID">b12_2011_146</identifier><accessCondition type="use and reproduction" contentType="copyright">Springer-Verlag Berlin Heidelberg, 2011</accessCondition><recordInfo><recordContentSource authority="ISTEX" authorityURI="https://loaded-corpus.data.istex.fr" valueURI="https://loaded-corpus.data.istex.fr/ark:/67375/XBH-RLRX46XW-4">springer</recordContentSource><recordOrigin>Springer-Verlag Berlin Heidelberg, 2011</recordOrigin></recordInfo></mods><json:item><extension>json</extension><original>false</original><mimetype>application/json</mimetype><uri>https://api.istex.fr/ark:/67375/HCB-MLX9QK2F-X/record.json</uri></json:item></metadata><annexes><json:item><extension>txt</extension><original>true</original><mimetype>text/plain</mimetype><uri>https://api.istex.fr/ark:/67375/HCB-MLX9QK2F-X/annexes.txt</uri></json:item></annexes></istex></record>Pour manipuler ce document sous Unix (Dilib)
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