Gene Therapy of Cancer. Methods and Protocols

342 Pages·2022·10.805 MB·English

by  Wolfgang Walther (ed.)

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
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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