Polymer Design for Nonviral Gene Delivery
Identifieur interne : 002252 ( Istex/Corpus ); précédent : 002251; suivant : 002253Polymer Design for Nonviral Gene Delivery
Auteurs : Kam W. LeongSource :
Abstract
Abstract: Gene therapy continues to hold promise in treating a variety of inherited and acquired diseases. The great majority of gene therapy trials rely on viral vectors for gene transduction because of their high efficiency. Viruses remain the vectors of choice in achieving high efficiency of gene transfer in vivo. Viral vectors, however, pose safety concerns unlikely to abate in the near future [1–3]. Issues of immunogenicity and toxicity remain a challenge. Limitations of cell mitosis for retrovirus, contamination of adenovirus, and packaging constraints of adeno-associated virus (AAV) also lessen their appeal. Non-viral vectors, although achieving only transient and lower gene expression level, may be able to compete on potential advantages of ease of synthesis, low immune response, and unrestricted plasmid size [4–9]. They have the potential to be administered repeatedly with minimal host immune response. They can also satisfy many of the pharmaceutical issues better than the viral vectors, such as scale-up, storage stability, and quality control. However, non-viral gene delivery is still too inefficient to be therapeutic for many applications. Development of safe and effective non-viral gene carriers is still critical to the ultimate success of gene therapy.
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DOI: 10.1007/978-0-387-25842-3_9
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Leong</name><affiliation><mods:affiliation>Department of Biomedical Engineering, Johns Hopkins School of Medicine, 21205, Baltimore, MD</mods:affiliation></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">ISTEX</idno><idno type="RBID">ISTEX:7295D4F4611349199345F9DFFEBA6ED5897248BA</idno><date when="2006" year="2006">2006</date><idno type="doi">10.1007/978-0-387-25842-3_9</idno><idno type="url">https://api.istex.fr/ark:/67375/HCB-PN334NL0-9/fulltext.pdf</idno><idno type="wicri:Area/Istex/Corpus">002252</idno><idno type="wicri:explorRef" wicri:stream="Istex" wicri:step="Corpus" wicri:corpus="ISTEX">002252</idno></publicationStmt><sourceDesc><biblStruct><analytic><title level="a" type="main" xml:lang="en">Polymer Design for Nonviral Gene Delivery</title><author><name sortKey="Leong, Kam W" sort="Leong, Kam W" uniqKey="Leong K" first="Kam W." last="Leong">Kam W. 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Non-viral vectors, although achieving only transient and lower gene expression level, may be able to compete on potential advantages of ease of synthesis, low immune response, and unrestricted plasmid size [4–9]. They have the potential to be administered repeatedly with minimal host immune response. They can also satisfy many of the pharmaceutical issues better than the viral vectors, such as scale-up, storage stability, and quality control. However, non-viral gene delivery is still too inefficient to be therapeutic for many applications. 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Non-viral vectors, although achieving only transient and lower gene expression level, may be able to compete on potential advantages of ease of synthesis, low immune response, and unrestricted plasmid size [4–9]. They have the potential to be administered repeatedly with minimal host immune response. They can also satisfy many of the pharmaceutical issues better than the viral vectors, such as scale-up, storage stability, and quality control. However, non-viral gene delivery is still too inefficient to be therapeutic for many applications. Development of safe and effective non-viral gene carriers is still critical to the ultimate success of gene therapy.</abstract><qualityIndicators><refBibsNative>false</refBibsNative><abstractWordCount>190</abstractWordCount><abstractCharCount>1285</abstractCharCount><keywordCount>0</keywordCount><score>9.28</score><pdfWordCount>10979</pdfWordCount><pdfCharCount>70255</pdfCharCount><pdfVersion>1.3</pdfVersion><pdfPageCount>25</pdfPageCount><pdfPageSize>468 x 711 pts</pdfPageSize></qualityIndicators><title>Polymer Design for Nonviral Gene Delivery</title><chapterId><json:string>9</json:string><json:string>Chap9</json:string></chapterId><genre><json:string>chapter</json:string></genre><host><title>BioMEMS and Biomedical Nanotechnology</title><language><json:string>unknown</json:string></language><copyrightDate>2006</copyrightDate><doi><json:string>10.1007/b136237</json:string></doi><eisbn><json:string>978-0-387-25842-3</json:string></eisbn><bookId><json:string>978-0-387-25842-3</json:string></bookId><isbn><json:string>978-0-387-25563-7</json:string></isbn><pages><first>239</first><last>263</last></pages><genre><json:string>book</json:string></genre><editor><json:item><name>Mauro Ferrari Ph.D.</name><affiliations><json:string>Department of Biomedical Engineering, University of Texas Health Science Center, Houston, TX</json:string><json:string>University of Texas M.D. Anderson Cancer Center, Houston, TX</json:string><json:string>Rice University, Houston, TX</json:string><json:string>University of Texas Medical Branch, Galveston, TX</json:string><json:string>Texas Alliance for NanoHealth, Houston, TX</json:string></affiliations></json:item><json:item><name>Abraham P. 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type="ark">ark:/67375/HCB-PN334NL0-9</idno><idno type="DOI">10.1007/978-0-387-25842-3_9</idno></analytic><monogr><title level="m" type="main">BioMEMS and Biomedical Nanotechnology</title><title level="m" type="sub">Volume I Biological and Biomedical Nanotechnology</title><idno type="DOI">10.1007/b136237</idno><idno type="book-id">978-0-387-25842-3</idno><idno type="ISBN">978-0-387-25563-7</idno><idno type="eISBN">978-0-387-25842-3</idno><idno type="chapter-id">Chap9</idno><editor><persName><forename type="first">Mauro</forename><surname>Ferrari</surname></persName><affiliation><orgName type="department">Department of Biomedical Engineering</orgName><orgName type="institution">University of Texas Health Science Center</orgName><address><settlement>Houston</settlement><region>TX</region></address></affiliation><affiliation><orgName type="institution">University of Texas M.D. Anderson Cancer Center</orgName><address><settlement>Houston</settlement><region>TX</region></address></affiliation><affiliation><orgName type="institution">Rice University</orgName><address><settlement>Houston</settlement><region>TX</region></address></affiliation><affiliation><orgName type="institution">University of Texas Medical Branch</orgName><address><settlement>Galveston</settlement><region>TX</region></address></affiliation><affiliation><orgName type="institution">Texas Alliance for NanoHealth</orgName><address><settlement>Houston</settlement><region>TX</region></address></affiliation></editor><editor><persName><forename type="first">Abraham</forename><forename type="first">P.</forename><surname>Lee</surname></persName><affiliation><orgName type="department">Biomedical Engineering</orgName><orgName type="institution">University of California</orgName><address><settlement>Irvine</settlement></address></affiliation></editor><editor><persName><forename type="first">L.</forename><forename type="first">James</forename><surname>Lee</surname></persName><affiliation><orgName type="department">Chemical and Biomolecular Engineering</orgName><orgName type="institution">The Ohio State University</orgName><address><country key="US">UNITED STATES</country></address></affiliation></editor><imprint><biblScope unit="page" from="239">239</biblScope><biblScope unit="page" to="263">263</biblScope><biblScope unit="chapter-count">16</biblScope></imprint></monogr></biblStruct></sourceDesc></fileDesc><profileDesc><abstract xml:lang="en"><head>Abstract</head><p>Gene therapy continues to hold promise in treating a variety of inherited and acquired diseases. The great majority of gene therapy trials rely on viral vectors for gene transduction because of their high efficiency. Viruses remain the vectors of choice in achieving high efficiency of gene transfer in vivo. Viral vectors, however, pose safety concerns unlikely to abate in the near future [1–3]. Issues of immunogenicity and toxicity remain a challenge. Limitations of cell mitosis for retrovirus, contamination of adenovirus, and packaging constraints of adeno-associated virus (AAV) also lessen their appeal. Non-viral vectors, although achieving only transient and lower gene expression level, may be able to compete on potential advantages of ease of synthesis, low immune response, and unrestricted plasmid size [4–9]. They have the potential to be administered repeatedly with minimal host immune response. They can also satisfy many of the pharmaceutical issues better than the viral vectors, such as scale-up, storage stability, and quality control. However, non-viral gene delivery is still too inefficient to be therapeutic for many applications. Development of safe and effective non-viral gene carriers is still critical to the ultimate success of gene therapy.</p></abstract><textClass ana="subject"><keywords scheme="book-subject-collection"><list><label>SUCO11647</label><item><term>Engineering</term></item></list></keywords></textClass><textClass ana="subject"><keywords scheme="book-subject"><list><label>SCT</label><item><term type="Primary">Engineering</term></item><label>SCT18000</label><item><term type="Secondary" subtype="priority-1">Nanotechnology and Microengineering</term></item><label>SCT2700X</label><item><term type="Secondary" subtype="priority-2">Biomedical Engineering</term></item><label>SCP27008</label><item><term type="Secondary" subtype="priority-3">Biophysics and Biological Physics</term></item><label>SCZ14000</label><item><term type="Secondary" subtype="priority-4">Nanotechnology</term></item><label>SCB0000X</label><item><term type="Secondary" subtype="priority-5">Biomedicine 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LLC</CopyrightHolderName><CopyrightYear>2006</CopyrightYear></ChapterCopyright><ChapterGrants Type="Regular"><MetadataGrant Grant="OpenAccess"></MetadataGrant><AbstractGrant Grant="OpenAccess"></AbstractGrant><BodyPDFGrant Grant="Restricted"></BodyPDFGrant><BodyHTMLGrant Grant="Restricted"></BodyHTMLGrant><BibliographyGrant Grant="Restricted"></BibliographyGrant><ESMGrant Grant="Restricted"></ESMGrant></ChapterGrants><ChapterContext><BookID>978-0-387-25842-3</BookID><BookTitle>BioMEMS and Biomedical Nanotechnology</BookTitle></ChapterContext></ChapterInfo><ChapterHeader><AuthorGroup><Author AffiliationIDS="Aff8"><AuthorName DisplayOrder="Western"><GivenName>Kam</GivenName><GivenName>W.</GivenName><FamilyName>Leong</FamilyName></AuthorName></Author><Affiliation ID="Aff8"><OrgDivision>Department of Biomedical Engineering</OrgDivision><OrgName>Johns Hopkins School of Medicine</OrgName><OrgAddress><City>Baltimore</City><State>MD</State><Postcode>21205</Postcode></OrgAddress></Affiliation></AuthorGroup><Abstract ID="Abs1" Language="En"><Heading>Abstract</Heading><Para TextBreak="No">Gene therapy continues to hold promise in treating a variety of inherited and acquired diseases. The great majority of gene therapy trials rely on viral vectors for gene transduction because of their high efficiency. Viruses remain the vectors of choice in achieving high efficiency of gene transfer in vivo. Viral vectors, however, pose safety concerns unlikely to abate in the near future [1–3]. Issues of immunogenicity and toxicity remain a challenge. Limitations of cell mitosis for retrovirus, contamination of adenovirus, and packaging constraints of adeno-associated virus (AAV) also lessen their appeal. Non-viral vectors, although achieving only transient and lower gene expression level, may be able to compete on potential advantages of ease of synthesis, low immune response, and unrestricted plasmid size [4–9]. They have the potential to be administered repeatedly with minimal host immune response. They can also satisfy many of the pharmaceutical issues better than the viral vectors, such as scale-up, storage stability, and quality control. However, non-viral gene delivery is still too inefficient to be therapeutic for many applications. Development of safe and effective non-viral gene carriers is still critical to the ultimate success of gene therapy.</Para></Abstract></ChapterHeader><NoBody></NoBody></Chapter></Book></Publisher></istex:document></istex:metadataXml><mods version="3.6"><titleInfo lang="en"><title>Polymer Design for Nonviral Gene Delivery</title></titleInfo><titleInfo type="alternative" contentType="CDATA"><title>Polymer Design for Nonviral Gene Delivery</title></titleInfo><name type="personal"><namePart type="given">Kam</namePart><namePart type="given">W.</namePart><namePart type="family">Leong</namePart><affiliation>Department of Biomedical Engineering, Johns Hopkins School of Medicine, 21205, Baltimore, MD</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Mauro</namePart><namePart type="family">Ferrari</namePart><namePart type="termsOfAddress">Ph.D.</namePart><affiliation>Department of Biomedical Engineering, University of Texas Health Science Center, Houston, TX</affiliation><affiliation>University of Texas M.D. Anderson Cancer Center, Houston, TX</affiliation><affiliation>Rice University, Houston, TX</affiliation><affiliation>University of Texas Medical Branch, Galveston, TX</affiliation><affiliation>Texas Alliance for NanoHealth, Houston, TX</affiliation><role><roleTerm type="text">editor</roleTerm></role><description>Editor-in-Chief, Professor, Brown Institute of Molecular Medicine Chairman, Professor of Experimental Therapeutics, Professor of Bioengineering, Professor of Biochemistry and Molecular Biology, President</description></name><name type="personal"><namePart type="given">Abraham</namePart><namePart type="given">P.</namePart><namePart type="family">Lee</namePart><affiliation>Biomedical Engineering, University of California, Irvine</affiliation><role><roleTerm type="text">editor</roleTerm></role></name><name type="personal"><namePart type="given">L.</namePart><namePart type="given">James</namePart><namePart type="family">Lee</namePart><affiliation>Chemical and Biomolecular Engineering, The Ohio State University, USA</affiliation><role><roleTerm type="text">editor</roleTerm></role></name><typeOfResource>text</typeOfResource><genre type="chapter" displayLabel="chapter" authority="ISTEX" authorityURI="https://content-type.data.istex.fr" valueURI="https://content-type.data.istex.fr/ark:/67375/XTP-CGT4WMJM-6"></genre><originInfo><publisher>Springer US</publisher><place><placeTerm type="text">Boston, MA</placeTerm></place><dateIssued encoding="w3cdtf">2006</dateIssued><copyrightDate encoding="w3cdtf">2006</copyrightDate></originInfo><language><languageTerm type="code" authority="rfc3066">en</languageTerm><languageTerm type="code" authority="iso639-2b">eng</languageTerm></language><abstract lang="en">Abstract: Gene therapy continues to hold promise in treating a variety of inherited and acquired diseases. The great majority of gene therapy trials rely on viral vectors for gene transduction because of their high efficiency. Viruses remain the vectors of choice in achieving high efficiency of gene transfer in vivo. Viral vectors, however, pose safety concerns unlikely to abate in the near future [1–3]. Issues of immunogenicity and toxicity remain a challenge. Limitations of cell mitosis for retrovirus, contamination of adenovirus, and packaging constraints of adeno-associated virus (AAV) also lessen their appeal. Non-viral vectors, although achieving only transient and lower gene expression level, may be able to compete on potential advantages of ease of synthesis, low immune response, and unrestricted plasmid size [4–9]. They have the potential to be administered repeatedly with minimal host immune response. They can also satisfy many of the pharmaceutical issues better than the viral vectors, such as scale-up, storage stability, and quality control. However, non-viral gene delivery is still too inefficient to be therapeutic for many applications. Development of safe and effective non-viral gene carriers is still critical to the ultimate success of gene therapy.</abstract><relatedItem type="host"><titleInfo><title>BioMEMS and Biomedical Nanotechnology</title><subTitle>Volume I Biological and Biomedical Nanotechnology</subTitle></titleInfo><name type="personal"><namePart type="given">Mauro</namePart><namePart type="family">Ferrari</namePart><namePart type="termsOfAddress">Ph.D.</namePart><affiliation>Department of Biomedical Engineering, University of Texas Health Science Center, Houston, TX</affiliation><affiliation>University of Texas M.D. Anderson Cancer Center, Houston, TX</affiliation><affiliation>Rice University, Houston, TX</affiliation><affiliation>University of Texas Medical Branch, Galveston, TX</affiliation><affiliation>Texas Alliance for NanoHealth, Houston, TX</affiliation><role><roleTerm type="text">editor</roleTerm></role><description>Editor-in-Chief, Professor, Brown Institute of Molecular Medicine Chairman, Professor of Experimental Therapeutics, Professor of Bioengineering, Professor of Biochemistry and Molecular Biology, President</description></name><name type="personal"><namePart type="given">Abraham</namePart><namePart type="given">P.</namePart><namePart type="family">Lee</namePart><affiliation>Biomedical Engineering, University of California, Irvine</affiliation><role><roleTerm type="text">editor</roleTerm></role></name><name type="personal"><namePart type="given">L.</namePart><namePart type="given">James</namePart><namePart type="family">Lee</namePart><affiliation>Chemical and Biomolecular Engineering, The Ohio State University, USA</affiliation><role><roleTerm type="text">editor</roleTerm></role></name><genre type="book" authority="ISTEX" authorityURI="https://publication-type.data.istex.fr" valueURI="https://publication-type.data.istex.fr/ark:/67375/JMC-5WTPMB5N-F">book</genre><originInfo><publisher>Springer</publisher><copyrightDate encoding="w3cdtf">2006</copyrightDate><issuance>monographic</issuance></originInfo><subject><genre>Book-Subject-Collection</genre><topic authority="SpringerSubjectCodes" authorityURI="SUCO11647">Engineering</topic></subject><subject><genre>Book-Subject-Group</genre><topic authority="SpringerSubjectCodes" authorityURI="SCT">Engineering</topic><topic authority="SpringerSubjectCodes" authorityURI="SCT18000">Nanotechnology and Microengineering</topic><topic authority="SpringerSubjectCodes" authorityURI="SCT2700X">Biomedical Engineering</topic><topic authority="SpringerSubjectCodes" authorityURI="SCP27008">Biophysics and Biological Physics</topic><topic authority="SpringerSubjectCodes" authorityURI="SCZ14000">Nanotechnology</topic><topic authority="SpringerSubjectCodes" authorityURI="SCB0000X">Biomedicine general</topic><topic authority="SpringerSubjectCodes" authorityURI="SCC12002">Biotechnology</topic></subject><identifier type="DOI">10.1007/b136237</identifier><identifier type="ISBN">978-0-387-25563-7</identifier><identifier type="eISBN">978-0-387-25842-3</identifier><identifier type="BookTitleID">117264</identifier><identifier type="BookID">978-0-387-25842-3</identifier><identifier type="BookChapterCount">16</identifier><part><date>2006</date><detail type="chapter"><number>9</number><caption>chapter</caption></detail><extent unit="pages"><start>239</start><end>263</end></extent></part><recordInfo><recordOrigin>Springer Science + Business Media, LLC, 2006</recordOrigin></recordInfo></relatedItem><identifier type="istex">7295D4F4611349199345F9DFFEBA6ED5897248BA</identifier><identifier type="ark">ark:/67375/HCB-PN334NL0-9</identifier><identifier type="DOI">10.1007/978-0-387-25842-3_9</identifier><identifier type="ChapterID">9</identifier><identifier type="ChapterID">Chap9</identifier><accessCondition type="use and reproduction" contentType="copyright">Springer Science + Business Media, LLC, 2006</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 Science + Business Media, LLC, 2006</recordOrigin></recordInfo></mods><json:item><extension>json</extension><original>false</original><mimetype>application/json</mimetype><uri>https://api.istex.fr/ark:/67375/HCB-PN334NL0-9/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-PN334NL0-9/annexes.txt</uri></json:item></annexes><serie></serie></istex></record>Pour manipuler ce document sous Unix (Dilib)
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