CRISPR in Animals and Animal Models: Volume 152 PDF
Preview CRISPR in Animals and Animal Models: Volume 152
VOLU MEONEHUNDREDANDFIFTYTWO P ROGRESS IN MOLECULAR BIOLOGY AND TRANSLATIONAL SCIENCE CRISPR in Animals and Animal Models Pageleft intentionallyblank VOLU MEONEHUNDREDANDFIFTYTWO P ROGRESS IN MOLECULAR BIOLOGY AND TRANSLATIONAL SCIENCE CRISPR in Animals and Animal Models Editedby Raúl Torres-Ruiz MolecularCytogeneticsandGenomeEngineeringGroup, CentroNacionalInvestigaciones Oncológicas(CNIO), Madrid, Spain Sandra Rodriguez-Perales MolecularCytogeneticsandGenomeEngineeringGroup, CentroNacionalInvestigaciones Oncológicas(CNIO), Madrid, Spain AcademicPressisanimprintofElsevier 125LondonWall,LondonEC2Y5AS,UnitedKingdom 525BStreet,Suite1800,SanDiego,CA92101-4495,UnitedStates 50HampshireStreet,5thFloor,Cambridge,MA02139,UnitedStates TheBoulevard,LangfordLane,Kidlington,OxfordOX51GB,UnitedKingdom Firstedition2017 Copyright©2017ElsevierInc.AllRightsReserved. Nopartofthispublicationmaybereproducedortransmittedinanyformorbyany means, electronic or mechanical, including photocopying, recording, or any information storage and retrieval system, without permission in writing from thepublisher.Detailsonhowtoseekpermission,furtherinformationaboutthe Publisher’spermissionspoliciesandourarrangementswithorganizationssuchas the Copyright Clearance Center and the Copyright Licensing Agency, can be foundatourwebsite:www.elsevier.com/permissions. Thisbookandtheindividualcontributionscontainedinitareprotectedunder copyrightbythePublisher(otherthanasmaybenotedherein). Notices Knowledgeandbestpracticeinthisfieldareconstantlychanging.Asnewresearch andexperiencebroadenourunderstanding,changesinresearchmethods,profes- sionalpractices,ormedicaltreatmentmaybecomenecessary. Practitioners and researchers must always rely on their own experience and knowledge in evaluating and using any information, methods, compounds, or experimentsdescribedherein.Inusingsuchinformationormethodstheyshould bemindfuloftheirownsafetyandthesafetyofothers,includingpartiesforwhom theyhaveaprofessionalresponsibility. Tothefullestextentofthelaw,neitherthePublishernortheauthors,contributors, oreditors,assumeanyliabilityforanyinjuryand/ordamagetopersonsorproperty asamatterofproductsliability,negligenceorotherwise,orfromanyuseoroperation ofanymethods,products,instructions,orideascontainedinthematerialherein. ISBN:978-0-12-812506-9 ISSN:1877-1173 ForinformationonallAcademicPresspublications visitourwebsiteathttps://www.elsevier.com/ Publisher:ZoeKruze AcquisitionEditor:AshlieM.Jackman EditorialProjectManager:JoannaCollett ProductionProjectManager:VigneshTamil CoverDesigner:MarkRogers TypesetbyThomsonDigital CONTENTS Contributors ix 1. CRISPRHistory:Discovery,Characterization,andProsperity 1 WenyuanHan,QunxinShe 1. Introduction 2 2. DiscoveryofCRISPR-CasSystems 3 3. MolecularMechanismsofNucleicAcidInterference 9 4. DiscoveryofAnti-CRISPRSystem 13 5. IdentificationofNovelCRISPR-CasSystems 14 6. BloomingofCRISPRTechnology 16 References 17 2. CRISPR/Cas9Technology:ApplicationsandHuman DiseaseModeling 23 MartaMartinez-Lage,RaúlTorres-Ruiz,SandraRodriguez-Perales 1. GenomeEngineeringTools 24 2. CRISPRApplications 31 3. CRISPRApplicationsinBiomedicalModeling 38 4. ConcludingRemarks 41 References 42 3. DynamicsofIndelProfilesInducedbyVarious CRISPR/Cas9DeliveryMethods 49 MichaelKosicki,SandeepS.Rajan,FlaminiaC.Lorenzetti, HansH.Wandall,YoshikiNarimatsu,EmmanouilMetzakopian, EricP.Bennett 1. Introduction 50 2. MaterialsandMethods 55 3. Results 58 v vi Contents 4. ConcludingRemarks 63 Acknowledgments 65 References 66 4. CRISPRLibrariesandScreening 69 JohnT.Poirier 1. Introduction 69 2. MouseModelsofCancer 71 3. CRISPR-Cas9Systems 72 4. CRISPRLibraries 73 5. ConcludingRemarks 78 References 78 5. GenomeEngineeringUsingHaploidEmbryonicStemCells 83 TakuroHorii,IzuhoHatada 1. Introduction 84 2. GenerationofHaploidESCells 85 3. PurificationandMaintenanceofHaploidCells 85 4. GenomeEditing 87 5. HaploidCellsasGameteReplacements 90 6. GeneticScreeningUsingHaploidESCells 91 7. ConcludingRemarks 91 References 92 6. CRISPRinAnimalsandAnimalModels 95 EllenShrock,MarcGüell 1. Introduction 96 2. MethodsofProducingGeneticallyModifiedAnimals 98 3. UsesofGeneticallyModifiedAnimals 101 4. LookingForward 106 5. EthicalConsiderations 108 6. ConcludingRemarks 109 References 109 7. GeneEditingandCRISPRTherapeutics:Strategies TaughtbyCellandGeneTherapy 115 JuanC.Ramirez 1. GenomeEditorsEntertheScene 116 2. MakingAdvancedTechnologiesGround-Breaking 117 Contents vii 3. HowHaveWeReachedThisPointSoQuickly? 119 4. HowFarintotheClinicWillGenome-EditingGo? 123 5. AFutureScenario—RisksAssociatedWithGeneEditingBusinesses 125 6. Conclusions 129 References 130 Index 131 Pageleft intentionallyblank CONTRIBUTORS EricP.Bennett DepartmentsofCellularandMolecularMedicineandOdontology,CopenhagenCenterfor Glycomics,UniversityofCopenhagen,Copenhagen,Denmark MarcGu¨ell BiologicalandBiomedicalSciences,HarvardUniversity,Boston,MA,UnitedStates; HarvardMedicalSchool,Boston,MA,UnitedStates; PompeuFabraUniversity,Barcelona,Spain WenyuanHan ArchaeaCenter,UniversityofCopenhagen,CopenhagenBiocenter,Copenhagen,Denmark IzuhoHatada BiosignalGenomeResourceCenter,InstituteforMolecularandCellularRegulation, GunmaUniversity,Maebashi,Gunma,Japan TakuroHorii BiosignalGenomeResourceCenter,InstituteforMolecularandCellularRegulation, GunmaUniversity,Maebashi,Gunma,Japan MichaelKosicki WellcomeTrustSangerInstitute,Hinxton,Cambridge,UnitedKingdom FlaminiaC.Lorenzetti DepartmentsofCellularandMolecularMedicineandOdontology,CopenhagenCenter forGlycomics,UniversityofCopenhagen,Copenhagen,Denmark MartaMartinez-Lage MolecularCytogeneticsandGenomeEngineeringGroup,CentroNacionalInvestigaciones Oncolo´gicas(CNIO),Madrid,Spain EmmanouilMetzakopian WellcomeTrustSangerInstitute,Hinxton,Cambridge,UnitedKingdom YoshikiNarimatsu DepartmentsofCellularandMolecularMedicineandOdontology,CopenhagenCenterfor Glycomics,UniversityofCopenhagen,Copenhagen,Denmark; GlycoDisplayAps,Copenhagen,Denmark JohnT.Poirier DepartmentofMedicine,MemorialSloanKetteringCancerCenter,NewYork,NY, UnitedStates ix
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