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Nucleic Acid Transfection PDF

316 Pages·2010·9.133 MB·English
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296 Topics in Current Chemistry Editorial Board: A. de Meijere K.N. Houk C.A. Hunter H. Kessler l l l J.-M. Lehn S.V. Ley M. Olivucci J. Thiem B.M. Trost l l l l M. Venturi P. Vogel H. Wong H. Yamamoto l l l Topics in Current Chemistry Recently Published and Forthcoming Volumes NucleicAcidTransfection Tubulin-BindingAgents: VolumeEditors:Bielke,W.,Erbacher,C. Synthetic,StructuralandMechanisticInsights Vol.296,2010 VolumeEditor:Carlomagno,T. Vol.286,2009 CarbohydratesinSustainableDevelopmentII VolumeEditors:Rauter,A.P.,Vogel,P., STMandAFMStudieson(Bio)molecular Queneau,Y. Systems:UnravellingtheNanoworld Vol.295,2010 VolumeEditor:Samor`ı,P. Vol.285,2008 CarbohydratesinSustainableDevelopmentI VolumeEditors:Rauter,A.P.,Vogel,P., AmplificationofChirality Queneau,Y. VolumeEditor:Soai,K. Vol.294,2010 Vol.284,2008 FunctionalMetal-OrganicFrameworks:Gas AnthracyclineChemistryandBiologyII Storage,SeparationandCatalysis ModeofAction,ClinicalAspectsandNewDrugs VolumeEditor:Schro¨der,M. VolumeEditor:Krohn,K. Vol.293,2010 Vol.283,2008 C-HActivation VolumeEditors:Yu,J.-Q.,Shi,Z. AnthracyclineChemistryandBiologyI Vol.292,2010 BiologicalOccurenceandBiosynthesis, SynthesisandChemistry AsymmetricOrganocatalysis VolumeEditor:Krohn,K. VolumeEditor:List,B. Vol.282,2008 Vol.291,2010 PhotochemistryandPhotophysicsof IonicLiquids CoordinationCompoundsII VolumeEditor:Kirchner,B. VolumeEditors:Balzani,V.,Campagna,S. Vol.290,2010 Vol.281,2007 OrbitalsinChemistry PhotochemistryandPhotophysicsof VolumeEditor:Inagaki,S. CoordinationCompoundsI Vol.289,2009 VolumeEditors:Balzani,V.,Campagna,S. Vol.280,2007 GlycoscienceandMicrobialAdhesion VolumeEditors:Lindhorst,T.K.,Oscarson,S. Vol.288,2009 MetalCatalyzedReductiveC–CBond Formation TemplatesinChemistryIII ADeparturefromPreformedOrganometallic VolumeEditors:Broekmann,P.,Do¨tz,K.-H., Reagents Schalley,C.A. VolumeEditor:Krische,M.J. Vol.287,2009 Vol.279,2007 Nucleic Acid Transfection Volume Editors: (cid:1) Wolfgang Bielke Christoph Erbacher With Contributions by A. Ahmad (cid:1) M.N. Bacon (cid:1) K. Berg (cid:1) M. Berstad (cid:1) N.F. Bouxsein (cid:1) C. Bra¨uchle (cid:1) M. Bradley (cid:1) M. Caldero´n (cid:1) H.M. Evans (cid:1) K.K. Ewert (cid:1) W.Fischer(cid:1) R.Haag(cid:1) P.Hahn(cid:1) I.Hedfors(cid:1) A.Høgset(cid:1) R.Koynova(cid:1) C.S. McAllister (cid:1) P.M. McLendon (cid:1) L. Prasmickaite (cid:1) T.M. Reineke (cid:1) N.Ruthardt (cid:1) C.R.Safinya (cid:1)E.E.Salcher (cid:1)C.E. Samuel(cid:1) E.Scanlan (cid:1) P.K. Selbo (cid:1) A. Sizovs (cid:1) S. Srinivasachari (cid:1) B. Tenchov (cid:1) A. Unciti-Broceta (cid:1) E. Wagner (cid:1) A. Weyergang (cid:1) A. Zidovska Editors Dr.WolfgangBielke Dr.ChristophErbacher QIAGENGmbH QIAGENGmbH QiagenStr.1 DivisionCorporateResearch 40724Hilden QiagenStr.1 Germany 40724Hilden [email protected] Germany [email protected] ISSN0340-1022 e-ISSN1436-5049 ISBN978-3-642-16429-3 e-ISBN978-3-642-16430-9 DOI10.1007/978-3-642-16430-9 SpringerHeidelbergDordrechtLondonNewYork LibraryofCongressControlNumber:2010938948 #Springer-VerlagBerlinHeidelberg2010 Thisworkissubjecttocopyright.Allrightsarereserved,whetherthewholeorpartofthematerialis concerned,specificallytherightsoftranslation,reprinting,reuseofillustrations,recitation,broadcasting, reproductiononmicrofilmorinanyotherway,andstorageindatabanks.Duplicationofthispublication orpartsthereofispermittedonlyundertheprovisionsoftheGermanCopyrightLawofSeptember9, 1965,initscurrentversion,andpermissionforusemustalwaysbeobtainedfromSpringer.Violations areliabletoprosecutionundertheGermanCopyrightLaw. Theuseofgeneraldescriptivenames,registerednames,trademarks,etc.inthispublicationdoesnot imply, even in the absence of a specific statement, that such names are exempt from the relevant protectivelawsandregulationsandthereforefreeforgeneraluse. Coverdesign:WMXDesignGmbH,Heidelberg,Germany Printedonacid-freepaper SpringerispartofSpringerScience+BusinessMedia(www.springer.com) Volume Editors Dr.WolfgangBielke Dr.ChristophErbacher QIAGENGmbH QIAGENGmbH QiagenStr.1 DivisionCorporateResearch 40724Hilden QiagenStr.1 Germany 40724Hilden [email protected] Germany [email protected] Editorial Board Prof.Dr.ArmindeMeijere Prof.Dr.Jean-MarieLehn Institutfu¨rOrganischeChemie ISIS derGeorg-August-Universita¨t 8,alle´eGaspardMonge Tammanstr.2 BP70028 37077Go¨ttingen,Germany 67083StrasbourgCedex,France [email protected] [email protected] Prof.Dr.KendallN.Houk Prof.Dr.StevenV.Ley UniversityofCalifornia UniversityChemicalLaboratory DepartmentofChemistryandBiochemistry LensfieldRoad 405HilgardAvenue CambridgeCB21EW LosAngeles,CA90024-1589,USA GreatBritain [email protected] [email protected] Prof.Dr.ChristopherA.Hunter Prof.Dr.MassimoOlivucci DepartmentofChemistry Universita`diSiena UniversityofSheffield DipartimentodiChimica SheffieldS37HF,UnitedKingdom ViaADeGasperi2 c.hunter@sheffield.ac.uk 53100Siena,Italy [email protected] Prof.Dr.HorstKessler Prof.Dr.JoachimThiem Institutfu¨rOrganischeChemie TUMu¨nchen Institutfu¨rOrganischeChemie Lichtenbergstraße4 Universita¨tHamburg 86747Garching,Germany Martin-Luther-King-Platz6 [email protected] 20146Hamburg,Germany [email protected] vi EditorialBoard Prof.Dr.BarryM.Trost Prof.Dr.HenryWong DepartmentofChemistry TheChineseUniversityofHongKong StanfordUniversity UniversityScienceCentre Stanford,CA94305-5080,USA DepartmentofChemistry [email protected] Shatin,NewTerritories [email protected] Prof.Dr.MargheritaVenturi Prof.Dr.HisashiYamamoto DipartimentodiChimica Universita`diBologna ArthurHollyCompton viaSelmi2 DistinguishedProfessor 40126Bologna,Italy DepartmentofChemistry [email protected] TheUniversityofChicago 5735SouthEllisAvenue Prof.Dr.PierreVogel Chicago,IL60637 773-702-5059 LaboratoryofGlycochemistry USA andAsymmetricSynthesis [email protected] EPFL–Ecolepolytechniquefe´derale deLausanne EPFLSBISICLGSA BCH5307(Bat.BCH) 1015Lausanne,Switzerland pierre.vogel@epfl.ch Topics in Current Chemistry Also Available Electronically Topics in Current Chemistry is included in Springer’s eBook package Chemistry andMaterialsScience.Ifalibrarydoesnotoptforthewholepackagethebookseries may be bought on a subscription basis. Also, all back volumes are available electronically. For all customers who have a standing order to the print version of Topics in Current Chemistry, we offer free access to the electronic volumes of the Series publishedinthecurrentyearviaSpringerLink. Ifyoudonothaveaccess,youcanstillviewthetableofcontentsofeachvolume and the abstract of each article by going to the SpringerLink homepage, clicking on “Chemistry and Materials Science,” under Subject Collection, then “Book Series,”underContentTypeandfinallybyselectingTopicsinCurrentChemistry. Youwillfindinformationaboutthe –EditorialBoard –AimsandScope –InstructionsforAuthors –SampleContribution atspringer.comusingthesearchfunctionbytypinginTopicsinCurrentChemistry. ColorfiguresarepublishedinfullcolorintheelectronicversiononSpringerLink. Aims and Scope TheseriesTopics inCurrentChemistry presents criticalreviews ofthe present and futuretrendsinmodernchemicalresearch.Thescopeincludesallareasofchemical science,including theinterfaceswithrelated disciplines suchasbiology,medicine, andmaterialsscience. Theobjectiveofeachthematicvolumeistogivethenon-specialistreader,whether at the university or in industry, a comprehensive overview of an area where new insightsofinteresttoalargerscientificaudienceareemerging. vii viii TopicsinCurrentChemistryAlsoAvailableElectronically Thus each review within the volume critically surveys one aspect of that topic and places it within the context of the volume as a whole. The most significant developmentsofthelast5–10yearsarepresented,usingselectedexamplestoillus- trate the principles discussed. A description of the laboratory procedures involved is often useful to the reader. The coverage is not exhaustive in data, but rather conceptual, concentrating on the methodological thinking that will allow the non- specialistreadertounderstandtheinformationpresented. Discussionofpossiblefutureresearchdirectionsintheareaiswelcome. Reviewarticlesfortheindividualvolumesareinvitedbythevolumeeditors. InreferencesTopicsinCurrentChemistryisabbreviatedTop CurrChemandis citedasajournal. ImpactFactor2009:4.291;Section“Chemistry,Multidisciplinary”:Rank20of138 Preface Transfectionofmacromoleculesintovariouscelltypesortissues,eitherinvitroor in vivo, has become an indispensable technique for most fields of biomedical research. Furthermore, future applications will likely lead to an increase in the importance of transfection technologies since, with the exception of using certain drugs, there is currentlylittle alternative to transfection for gene-specific manipu- lationofcells.Typically,transfectionsareperformedwithvarioustypesofnucleic acids such as genetic expression constructs, siRNAs, and occasionally messenger RNAs. Although many physical and viral transfection methods have been devel- oped for use in different applications, the majority of protocols use chemical transfectionreagents. In this volume, we have brought together articles from various experts in thechemicaltransfectionreagentfield.Topicscoveredincludedescriptionsofthe chemistry of chemical transfection compounds and details of the fundamental parametersthatinfluencetransfectionefficiencies. ThearticlebyHahnandScanlangivesageneraloverviewofcommontransfec- tionmethodsandpresentsavaluableentrancepointtothefield. Students,butalsoadvancedresearchers,areadvisedtocontinuewiththearticle by Unciti-Broceta et al., which gives a comprehensive outline of the chemistry underlyingliquidandsolidsynthesisofcationiclipidsanddendrimers. Asummary anddiscussion ofempirical data on relationships between cationic lipid molecular structure and transfection activity are provided by Koynova and Tenchov Structural features of cationic lipids are related to their interaction with biomembranesandtheuptakeefficiencyoftransfectioncomplexes. Several authors describe distinct classes of chemical transfection reagents. Fischer et al. summarize synthesis methods, features, and applications of hyper- branchedpolyaminesascarriersfornucleicacids. A review from Sizovs et al. discusses carbohydrate-based polymeric transfec- tionreagents,turningspecialattentiontotheirpotentialapplicationsinpreclinical modelsandforthetreatmentofdiseases. Ewert et al. present recent work on structural studies of cationic lipid/nucleic acid interaction, optimized for therapeutic nucleic acid delivery. Fundamental ix

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