Gene Therapy of Cancer. Methods and Protocols PDF
Preview Gene Therapy of Cancer. Methods and Protocols
Methods in Molecular Biology 2521 Wolfgang Walther Editor Gene Therapy of Cancer Methods and Protocols Third Edition M M B ETHODS IN OLECULAR IO LO GY SeriesEditor JohnM.Walker School of Lifeand MedicalSciences University ofHertfordshire Hatfield, Hertfordshire, UK Forfurther volumes: http://www.springer.com/series/7651 For over 35 years, biological scientists have come to rely on the research protocols and methodologiesinthecriticallyacclaimedMethodsinMolecularBiologyseries.Theserieswas thefirsttointroducethestep-by-stepprotocolsapproachthathasbecomethestandardinall biomedicalprotocolpublishing.Eachprotocolisprovidedinreadily-reproduciblestep-by- step fashion, opening with an introductory overview, a list of the materials and reagents neededtocompletetheexperiment,andfollowedbyadetailedprocedurethatissupported with a helpful notes section offering tips and tricks of the trade as well as troubleshooting advice. These hallmark features were introduced by series editor Dr. John Walker and constitutethekeyingredientineachandeveryvolumeoftheMethodsinMolecularBiology series. Tested and trusted, comprehensive and reliable, all protocols from the series are indexedinPubMed. Gene Therapy of Cancer Methods and Protocols Third Edition Edited by Wolfgang Walther Max-Delbrück-Center for Molecular Medicine, Berlin, Berlin, Germany Editor WolfgangWalther Max-Delbru¨ck-CenterforMolecular Medicine Berlin,Berlin,Germany ISSN1064-3745 ISSN1940-6029 (electronic) MethodsinMolecularBiology ISBN978-1-0716-2440-1 ISBN978-1-0716-2441-8 (eBook) https://doi.org/10.1007/978-1-0716-2441-8 ©TheEditor(s)(ifapplicable)andTheAuthor(s),underexclusivelicensetoSpringerScience+BusinessMedia,LLC,part ofSpringerNature2022 Thisworkissubjecttocopyright.AllrightsaresolelyandexclusivelylicensedbythePublisher,whetherthewholeorpart of the material is concerned, specifically the rights of translation, reprinting, reuse of illustrations, recitation, broadcasting,reproductionon microfilmsorinanyotherphysicalway,andtransmissionorinformation storageand retrieval,electronicadaptation, computersoftware,orbysimilar ordissimilar methodologynow knownorhereafter developed. Theuseofgeneraldescriptivenames,registerednames,trademarks,servicemarks,etc.inthispublicationdoesnotimply, evenintheabsenceofaspecificstatement,thatsuchnamesareexemptfromtherelevantprotectivelawsandregulations andthereforefreeforgeneraluse. Thepublisher,theauthorsandtheeditorsaresafetoassumethattheadviceandinformationinthisbookarebelievedto betrueandaccurateatthedateofpublication.Neitherthepublishernortheauthorsortheeditorsgiveawarranty, expressedorimplied,withrespecttothematerialcontainedhereinorforanyerrorsoromissionsthatmayhavebeen made.Thepublisherremainsneutralwithregardtojurisdictionalclaimsinpublishedmapsandinstitutionalaffiliations. ThisHumanaimprintispublishedbytheregisteredcompanySpringerScience+BusinessMedia,LLC,partofSpringer Nature. Theregisteredcompanyaddressis:1NewYorkPlaza,NewYork,NY10004,U.S.A. Preface This book on protocols for Gene Therapy of Cancer is meanwhile the third edition of this successfulseries.Thesethreeeditionscover20yearsinevolutionofcancergenetherapyand represent a tribute to the accelerated speed of development and still growing attention to this field. Although the main topics in cancer gene therapy, such as immunogen therapy, suicidegenetherapy,genereplacement,vectordevelopmentandrefinement,etc.,arestillin focus,themajoractivitieshavesignificantlyshiftedovertheyears.Currentlymoreeffortsare made for improved immunogene therapy, based on TCR and CAR engineering, tumor vaccination using DNA or RNA vaccines, and on immune stimulation at different levels. Engineering and use of oncolytic viruses is experiencing a renaissance, stimulated by accu- mulating knowledge in mechanisms of oncolysis action and its interplay with the immune system.Vectordevelopment(viralandnonviral)andgenedeliveryaretakingincreasingparts ofresearchincancergenetherapy.Farmoreclinicaltrialsforcancergenetherapyareunder waytoimprovetheoutcomeofcancerpatients,tolearnmoreaboutclinicalapplicabilityand the needs of optimized and affordable generation of gene therapeutic products as nucleic acid-basedpharmaceuticals. Thus,followingthislongandmuchappreciatedtraditionofthe“MethodsinMolecular Biology”editions,hereagainchaptersareprovidedfornoveldetailedstep-by-stepprotocols of particular gene therapeutic procedures of quite different strategies. This is presented in combinationwithhelpfulandvaluedtechnicalnotestoaidreproducibilityofthedescribed protocols in any laboratory on the globe. This book edition complements the protocol chapterswithbrief,butveryinformativeintroductionstospecificapproaches,explainingthe scientificbackgroundandprovidingsomeoverviewforspecificresearchfieldsofcancergene therapy. As the other, previous editions, this third edition is also motivated by the strong persuasionthatcancergenetherapyhasbecomeanapplicableandclinicallyrelevantreality. Therefore,thiseditionwillprovidesomeinsightonnewdevelopmentsinthefieldandwill alsoprovidethedescriptionanddetailsofhowthisistechnicallydone.Thisistightlylinked to the hope ofbeing helpful forall researchers and cliniciansworking inthe field of cancer genetherapyor forallthosereaders,whoarejustatthebeginningtogetintouchwiththis stillexcitingandinnovativeresearcharea. Berlin,Germany WolfgangWalther v Contents Preface ..................................................................... v Contributors................................................................. ix 1 ABriefIntroductiontoCurrentCancerGeneTherapy...................... 1 DennisKobelt,JessicaPahle,andWolfgangWalther PART I STRATEGIES IN CANCER GENE THERAPY 2 MinicirclesforCARTCellProductionbySleepingBeautyTransposition: ATechnologicalOverview ............................................... 25 SabrinaPrommersberger,RaziehMonjezi,RamShankar, MarcoSchmeer,MichaelHudecek,Zolta´nIvics,andMartinSchleef 3 GenerationofCAR-TCellswithSleepingBeautyTransposon GeneTransfer .......................................................... 41 SabrinaPrommersberger,RaziehMonjezi,LacramioaraBotezatu, CsabaMiskey,MaximilianAmberger,KatrinMestermann, MichaelHudecek,andZolta´nIvics 4 GenerationofRedirectedEngineeredHumanChimericAntigen Receptor(CAR)TCells ................................................. 67 MarioBunseandUtaE.H¨opken 5 TCRandCAREngineeringofPrimaryHumanTCells...................... 85 InanEdes,JulianClauss,RainerStahn,AlbertoSadaJapp, andFelixK.M.Lorenz 6 TCRGeneTherapyforCancer ........................................... 95 SimoneRheinandNes¸eC¸akmak-G¨oru¨r 7 GeneGunHer2/neuDNAVaccination:EvaluationofVaccineEfficacy inaSyngeneicHer2/neuMouseTumorModel ............................ 129 TamNguyen-Hoai,OliverHohn,AntonioPezzutto, andJ¨orgWestermann PART II SUICIDE GENE THERAPY 8 ConstructionofMinicircleSuicideGenesCodingforRibosome-Inactivating Proteins................................................................ 157 AlexanderSonntag,HardyMitdank,andAlexanderWeng 9 Claudin-TargetedSuicideGeneTherapyforClaudin-OverexpressingTumor CellsbyUsingModifiedClostridiumperfringensEnterotoxin(CPE).......... 173 Laura-SophieBeier,J¨orgPiontek,AnnaPiontek, JonasProtze,DennisKobelt,andWolfgangWalther vii viii Contents PART III GENE SUPPRESSION AND SIGNALING MODULATION THERAPY FOR CANCER 10 BioengineeredRNATherapyinPatient-DerivedOrganoids andXenograftMouseModels ............................................ 191 Mei-JuanTu,ColleenM.Yi,GavinM.Traber,andAi-MingYu 11 ApplicationofTranscriptionFactorDecoyOligodeoxynucleotides (ODNs)forCancerTherapy ............................................. 207 BehroozJohariandMohammadMoradi PART IV ONCOLYTIC THERAPY FOR CANCER 12 NKCellEffectorFunctionsandBystanderTumorCellKilling inImmunovirotherapy .................................................. 233 AlessiaFloerchingerandChristineE.Engeland 13 AqPCR-BasedMethodforQuantificationofReplication CompetentAdenovirus(RCA)inConditionallyReplicating OncolyticAdenoviruses.................................................. 249 ErikYngve,MenghanGao,andDiYu 14 GenerationofamicroRNA-RegulatedOncolyticCoxsackievirusB3 .......... 259 BabetteDieringer,LeslieElsner,AhmetHazini, JensKurreck,andHenryFechner 15 VisualizedCancerImmunotherapywithEngineered Salmonellatyphimurium................................................. 283 YujieSunandJinHaiZheng PART V TRANSLATIONAL ASPECTS FOR CANCER GENE THERAPY 16 CancerGeneTherapy:DevelopmentandProductionofLentiviral VectorsforGeneTherapy................................................ 297 AnaS.Coroadinha 17 CapillaryGelElectrophoresis(CGE)forQualityControlofPlasmid DNAinGeneTherapy:QualityControlof20YearsStoredGMP-Grade PlasmidDNA .......................................................... 317 MarcoSchmeer,MartinSchleef,RamShankar,DennisKobelt, andWolfgangWalther 18 EfficientGenerationofSuperiorDumbbell-ShapedNonviralDNA DeliveryVectorsUsing1-2-3Gap-PrimerPCR ............................ 329 PeiSheLohandVolkerPatzel Index ...................................................................... 339 Contributors MAXIMILIANAMBERGER • DivisionofMedicalBiotechnology,PaulEhrlichInstitute,Langen, Germany LAURA-SOPHIEBEIER • DivisionofGastroenterology,Infectiology,Rheumatology,Clinical Physiology/NutritionalMedicine,MedicalDepartment,Charite`-Universita€tsmedizin Berlin,Berlin,Germany;DivisionofCellandDevelopmentalBiology,InstituteofBiology, UniversityofLeipzig,Leipzig,Germany LACRAMIOARABOTEZATU • DivisionofMedicalBiotechnology,PaulEhrlichInstitute, Langen,Germany MARIOBUNSE • DepartmentofMicroenvironmentalRegulationinAutoimmunityand Cancer,Max-Delbru¨ck-Center forMolecularMedicine(MDC),Berlin,Germany NES¸EC¸AKMAK-GO¨RU¨R • Charite´-Universita€tsmedizin,corporatememberofFreie Universita€tBerlinandHumboldt-Universita€tzuBerlin,Berlin,Germany JULIANCLAUSS • Max-Delbru¨ck-CenterforMolecularMedicineintheHelmholtzAssociation (MDC),Berlin,Germany ANAS.COROADINHA • iBET,InstitutodeBiologiaExperimentaleTecnol(cid:2)ogica,Oeiras, Portugal;InstitutodeTecnologiaQuı´micaeBiol(cid:2)ogicaAnt(cid:2)onioXavier,UniversidadeNova deLisboa,Oeiras,Portugal BABETTEDIERINGER • DepartmentofAppliedBiochemistry,InstituteofBiotechnology, TechnischeUniversita€tBerlin,Berlin,Germany INANEDES • Max-Delbru¨ck-Center forMolecularMedicineintheHelmholtzAssociation (MDC),Berlin,Germany LESLIE ELSNER • DepartmentofAppliedBiochemistry,InstituteofBiotechnology,Technische Universita€tBerlin,Berlin,Germany CHRISTINEE.ENGELAND • CenterforBiomedicalResearchandEducation(ZBAF),Schoolof Medicine,InstituteofVirologyandMicrobiology,FacultyofHealth,Witten/Herdecke University,Witten,Germany;ClinicalCooperationUnitVirotherapy,GermanCancer ResearchCenter(DKFZ),NationalCenterforTumorDiseases(NCT)andDepartmentof MedicalOncology,UniversityHospitalHeidelberg,Heidelberg,Germany HENRYFECHNER • DepartmentofAppliedBiochemistry,InstituteofBiotechnology, TechnischeUniversita€tBerlin,Berlin,Germany ALESSIA FLOERCHINGER • Center forBiomedicalResearchandEducation(ZBAF),Schoolof Medicine,InstituteofVirologyandMicrobiology,FacultyofHealth,Witten/Herdecke University,Witten,Germany MENGHANGAO • DepartmentofImmunology,GeneticsandPathology;ScienceforLife Laboratory,UppsalaUniversity,Uppsala,Sweden AHMETHAZINI • DepartmentofAppliedBiochemistry,InstituteofBiotechnology,Technische Universita€tBerlin,Berlin,Germany;DepartmentofOncology,UniversityofOxford, Oxford,Oxfordshire,UK OLIVERHOHN • RobertKochInstitute,Berlin,Germany UTAE.HO¨PKEN • DepartmentofMicroenvironmentalRegulationinAutoimmunityand Cancer,Max-Delbru¨ck-Center forMolecularMedicine(MDC),Berlin,Germany MICHAELHUDECEK • DepartmentofInternalMedicineII,UniversityHospitalofWu¨rzburg, Wu¨rzburg,Germany ix x Contributors ZOLTA´NIVICS • DivisionofMedicalBiotechnology,PaulEhrlichInstitute,Langen,Germany ALBERTOSADAJAPP • Max-Delbru¨ck-Center forMolecularMedicineintheHelmholtz Association(MDC),Berlin,Germany BEHROOZJOHARI • CancerGeneTherapyResearchCenter,ZanjanUniversityofMedical Sciences,Zanjan,Iran;DepartmentofMedicalBiotechnology,SchoolofMedicine,Zanjan UniversityofMedicalSciences,Zanjan,Iran DENNISKOBELT • Max-Delbru¨ck-Center forMolecularMedicine,Berlin,Germany; ExperimentalandClinicalResearchCenter,Charite´-Universita€tsmedizinBerlin,Berlin, Germany;GermanCancerConsortium(DKTK),DeutschesKrebsforschungzentrum (DKFZ),Heidelberg,Germany JENS KURRECK • DepartmentofAppliedBiochemistry,InstituteofBiotechnology,Technische Universita€tBerlin,Berlin,Germany PEISHELOH • HealthyLongevityTranslationalResearchProgramme,YongLooLinSchool ofMedicine,NationalUniversityofSingapore,Singapore,Singapore;Departmentof Microbiology&Immunology,YongLooLinSchoolofMedicine,NationalUniversityof Singapore,Singapore,Singapore FELIXK.M.LORENZ • Max-Delbru¨ck-Center forMolecularMedicineintheHelmholtz Association(MDC),Berlin,Germany KATRINMESTERMANN • DepartmentofInternalMedicineII,UniversityHospitalof Wu¨rzburg,Wu¨rzburg,Germany CSABAMISKEY • DivisionofMedicalBiotechnology,PaulEhrlichInstitute,Langen,Germany HARDYMITDANK • InstituteofPharmacy,FreieUniversita€tBerlin,Berlin,Germany RAZIEHMONJEZI • DepartmentofInternalMedicineII,UniversityHospitalofWu¨rzburg, Wu¨rzburg,Germany MOHAMMADMORADI • DepartmentofBiotechnology,FacultyofBiologicalScienceand Technology,UniversityofIsfahan,Isfahan,Iran;BloodTransfusionResearchCenter,High InstituteforResearchandEducationinTransfusionMedicine,Tehran,Iran TAMNGUYEN-HOAI • ExperimentalandClinicalResearchCenter,Charite´- Universita€tsmedizinBerlin,Berlin,Germany JESSICAPAHLE • ExperimentalandClinicalResearchCenter,Charite´-Universita€tsmedizin Berlin,Berlin,Germany VOLKERPATZEL • HealthyLongevityTranslationalResearchProgramme,YongLooLin SchoolofMedicine,NationalUniversityofSingapore,Singapore,Singapore;Departmentof Microbiology&Immunology,YongLooLinSchoolofMedicine,NationalUniversityof Singapore,Singapore,Singapore ANTONIO PEZZUTTO • Charite´-Universita€tsmedizinBerlinandMax-Delbru¨ck-Center for MolecularMedicine,Berlin,Germany ANNAPIONTEK • Leibniz-Forschungsinstitutfu¨rMolekularePharmakologie(FMP),Berlin, Germany JO¨RGPIONTEK • DivisionofGastroenterology,Infectiology,Rheumatology,Clinical Physiology/NutritionalMedicine,MedicalDepartment,Charite`-Universita€tsmedizin Berlin,Berlin,Germany SABRINAPROMMERSBERGER • DepartmentofInternalMedicineII,UniversityHospitalof Wu¨rzburg,Wu¨rzburg,Germany JONAS PROTZE • Leibniz-Forschungsinstitutfu¨rMolekularePharmakologie(FMP),Berlin, Germany SIMONERHEIN • MaxDelbru¨ckCenterforMolecularMedicineintheHelmholtzAssociation, Berlin,Germany
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