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Author Schleef, Martin
Title Minicircle and Miniplasmid DNA Vectors : The Future of Non-Viral and Viral Gene Transfer
Imprint Weinheim : John Wiley & Sons, Incorporated, 2013
©2013
book jacket
Edition 1st ed
Descript 1 online resource (259 pages)
text txt rdacontent
computer c rdamedia
online resource cr rdacarrier
Note Minicircle and Miniplasmid DNA Vectors: The Future of Non-Viral and Viral Gene Transfer -- Contents -- List of Contributors -- Perface -- 1 Minicircle Patents: A Short IP Overview of Optimizing Nonviral DNA Vectors -- 2 Operator-Repressor Titration: Stable Plasmid Maintenance without Selectable Marker Genes -- 2.1 Introduction -- 2.2 Antibiotics and Metabolic Burden -- 2.3 The Mechanism of ORT -- 2.4 ORT Strain Development -- 2.5 ORT Miniplasmids -- 2.6 DNA Vaccine and Gene Therapy Vectors -- 2.7 ORT-VAC: Plasmid-Based Vaccine Delivery Using Salmonella enterica -- 2.8 Recombinant Protein Expression -- 2.9 Conclusions and Future Developments -- References -- 3 Selection by RNA-RNA Interaction: Maximally Minimized Antibiotic Resistance-Free Plasmids -- 3.1 Gene Therapy and DNA Vaccines: Emerging Technologies -- 3.1.1 Therapeutic Plasmids: General Design Principles -- 3.2 Therapeutic Plasmids: Novel Design and the Problem of Selection -- 3.2.1 Replication Control of ColE1-Type Plasmids as an Alternative Selection Marker -- 3.2.2 The MINIback Concept: Selection by RNA-RNA Interaction -- 3.2.3 Improved Production Processes by MINIback Plasmids -- 3.2.4 Improving Sequence Composition -- 3.2.5 Efficient Gene Transfer -- 3.3 Conclusions -- Acknowledgments -- References -- 4 Plasmid-Based Medicinal Products - Focus on pFAR: A Miniplasmid Free of Antibiotic Resistance Markers -- 4.1 Introduction: Rationale for the Development of Biosafe DNA Plasmid Vectors -- 4.2 Specific Requirements for the Use of DNA Product as Medicines -- 4.2.1 Requirements for Plasmid Quality and Purity -- 4.2.2 Requirements for the Removal of Antibiotic Resistance Markers from Plasmid DNA -- 4.2.2.1 Requirements for Biosafe Plasmids -- 4.2.2.2 Positive Impact on the Removal of Antibiotic Resistance Markers
4.2.2.3 Effect of Plasmid Size on Gene Transfer Efficiency In Vitro and In Vivo -- 4.3 Nonviral Gene Vectors Devoid of Antibiotic Resistance Markers -- 4.3.1 Generalities -- 4.3.2 Selection Systems Devoid of Antibiotic Resistance Markers -- 4.3.2.1 Complementation of Host Auxotrophy by a Function-Encoded Plasmid -- 4.3.2.2 The Operator-Repressor Titration (ORT) System -- 4.3.2.3 Protein-Based Antidote/Poison Selection Systems -- 4.3.2.4 RNA-Based Selection Marker -- 4.3.2.5 Suppression of a Nonsense Mutation -- 4.4 The pFAR Plasmid Family -- 4.4.1 Description of the Antibiotic-Free Selection System -- 4.4.2 pFAR Vectors Promote Efficient Expression in Several Types of Mammalian Cells -- 4.4.2.1 In Vitro Transfection Study -- 4.4.2.2 In Vivo Transfection Studies -- 4.4.3 Concluding Remarks on the pFAR4 Biosafe Miniplasmid -- 4.5 Concluding Remarks and Perspectives -- Acknowledgments -- References -- 5 Plasmid DNA Concatemers: Influence of Plasmid Structure on Transfection Efficiency -- 5.1 Introduction -- 5.2 Plasmid DNA Topology and Size -- 5.3 Plasmid DNA Concatemers -- 5.4 Conclusions -- Acknowledgments -- References -- 6 Analytical Tools in Minicircle Production -- 6.1 Introduction -- 6.1.1 Gene Transfer for Therapy, Vaccination, and Stem Cells -- 6.1.2 Plasmids -- 6.1.3 Minicircle Systems -- 6.2 Production of Minicircles -- 6.2.1 The Parental Plasmid -- 6.2.2 Cultivation and Induction -- 6.2.3 Minicircle Preparation -- 6.3 Analytics of Minicircle Production -- 6.3.1 In-Process Control -- 6.3.1.1 Atomic Force Microscopy -- 6.3.1.2 Capillary Gel Electrophoresis -- 6.3.1.3 Continuous Flow Separation in Microfluidic Channels -- 6.3.2 Finished Product Control -- 6.4 Future Goals -- Acknowledgments -- References -- 7 Utilizing Minicircle Vectors for the Episomal Modification of Cells -- 7.1 Introduction
7.2 Studies that Show Passive Episomal Maintenance of Minicircles In Vivo -- 7.3 Principles of Generating Minicircle Vectors Able to Support Episomal Maintenance -- 7.3.1 Episomal Maintenance of Minicircle S/MAR Vectors Generated by Flp Recombinase In Vitro -- 7.3.2 Episomal Maintenance of Minicircle S/MAR Vectors Generated Using Cre Recombinase In Vitro -- 7.3.3 Episomal Maintenance of S/MAR Vectors in Bovine and Murine Zygotes -- 7.4 Episomal Maintenance of S/MAR Minicircles In Vivo -- 7.5 Potential of Episomal Replication of S/MAR Minicircle Vectors -- 7.6 Possible Mechanisms Promoting the Episomal Maintenance of Minicircle Vectors -- 7.6.1 Histone Modifications -- 7.6.2 CpG Dinucleotide Content Reduction -- 7.6.3 Vector Establishment in the Correct Nuclear Compartment -- 7.6.4 Access to Replication Machinery by S/MARs -- 7.7 Conclusions -- References -- 8 Replicating Minicircles: Overcoming the Limitations of Transient and Stable Expression Systems -- 8.1 Gene Therapy: The Advent of Novel Vector Vehicles -- 8.1.1 Nonviral Vectors Avoiding Genomic Disturbances -- 8.1.2 Independent Expression Units: Chromatin Domains -- 8.1.2.1 S/MARs: a Unifying Principle -- 8.1.2.2 S/MAR Actions Are Multifold and Context Dependent -- 8.1.2.3 Stress-Induced Duplex Destabilization: a Unifying Property of S/MARs -- 8.1.2.4 Chromosome-Based Expression Strategies: Episomes and/or Predetermined Integration Sites (RMCE) -- 8.2 Replicating Nonviral Episomes -- 8.2.1 Can the Yeast ARS Principle Be Verified for Mammalian Cells? -- 8.2.2 ARS and S/MARs: Common (SIDD-) Properties -- 8.2.3 S/MAR Plasmids: Verification of the Concept -- 8.2.3.1 Transcription into the S/MAR: Directionality and Rate -- 8.2.3.2 Cell and Nuclear Permeation -- 8.2.3.3 Nuclear Association Sites -- 8.2.3.4 RMCE-Based Elaboration Following Establishment
8.2.4 Remaining Shortcomings and Their Solution -- 8.2.4.1 Establishment and Maintenance: the EBV Paradigm -- 8.2.4.2 Vector Size Limitations -- 8.3 Minimalization Approaches -- 8.3.1 Oligomerizing S/MAR Modules: pMARS and Its Properties -- 8.3.2 Replicating Minicircles: a Solution with Great Promise -- 8.3.2.1 Establishment and Maintenance Parameters -- 8.3.2.2 Clonal Behavior -- 8.3.2.3 Bi-MC Systems -- 8.3.2.4 MC Size Reduction: "In Vivo Evolution" -- 8.3.2.5 Transcriptional Termination and Polyadenylation: an Intricate Interplay -- 8.3.2.6 Episomal Status: Proof and Persistence -- 8.3.3 Emerging Extensions and Refinements -- 8.3.3.1 Combination of Excision and RMCE Strategies -- 8.3.3.2 MC Withdrawal at Will -- 8.3.3.3 Pronuclear Injection and Somatic Cell Nuclear Transfer -- 8.3.3.4 From Cells to Organs -- 8.4 Summary and Outlook -- Acknowledgments -- References -- 9 Magnetofection of Minicircle DNA Vectors -- 9.1 Introduction -- 9.2 Overview of Magnetofection Principles -- 9.3 Cellular Uptake -- 9.4 Diffusion through the Cytoplasm -- 9.5 Transgene Expression -- 9.6 Conclusions -- References -- 10 Minicircle-Based Vectors for Nonviral Gene Therapy: In Vitro Characterization and In Vivo Application -- 10.1 Minicircle Technology for Nonviral Gene Therapy -- 10.2 Current Status of In Vivo Application of Minicircle Vectors -- 10.3 Jet Injection Technology for In Vivo Transfer of Naked DNA -- 10.4 Comparative Performance Analyses of Minicircle Vectors -- 10.5 In Vivo Application of Minicircle DNA by Jet Injection -- References -- 11 Episomal Expression of Minicircles and Conventional Plasmids in Mammalian Embryos -- 11.1 Introduction -- 11.2 Fate of Plasmids and Minicircles After Injection into Mammalian Embryos -- 11.2.1 Minicircle- and Plasmid-Mediated Expression in Early Embryos and Fetuses
11.2.2 Expression of Functional Genes in Preimplantation Embryos -- 11.3 Discussion -- References -- 12 Tissue-Targeted Gene Electrodelivery of Minicircle DNA -- 12.1 Introduction -- 12.2 Plasmid DNA Electrotransfer: From Principle to Technical Design -- 12.2.1 Mechanism of Gene Electrotransfer -- 12.2.2 Preclinical Applications -- 12.3 Implementation for Efficient Tissue-Targeted Gene Delivery -- 12.3.1 Design of DNA Vector -- 12.3.2 In Vitro Minicircle Electrotransfer -- 12.3.3 In Vivo MC Electrotransfer -- 12.3.3.1 Muscle -- 12.3.3.2 Tumor -- 12.3.3.3 Skin -- 12.4 Conclusions -- Acknowledgments -- References -- 13 Increased Efficiency of Minicircles Versus Plasmids Under Gene Electrotransfer Suboptimal Conditions: an Influence of the Extracellular Matrix -- 13.1 Introduction -- 13.2 Methods -- 13.2.1 Cell Culture and Animals -- 13.2.2 Minicircle and Plasmid -- 13.2.3 Electrotransfer -- 13.2.4 Determination of the Reporter Gene (Luciferase) Activity -- 13.2.5 Data Analysis -- 13.3 Results -- 13.3.1 In Vitro -- 13.3.2 In Vivo -- 13.4 Discussion -- 13.5 Conclusions -- Acknowledgments -- References -- Index
Dr. Martin Schleef studied Biology at the Universities of Würzburg and Bielefeld, Germany and holds a PhD from the University of Bielefeld. Martin Schleef received post-doctoral training from the Institut Pasteur Paris, France. He joined QIAGEN GmbH, Hilden, Germany in 1994 and is co-founder and CEO of PlasmidFactory in Bielefeld since 2000
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Electronic reproduction. Ann Arbor, Michigan : ProQuest Ebook Central, 2020. Available via World Wide Web. Access may be limited to ProQuest Ebook Central affiliated libraries
Link Print version: Schleef, Martin Minicircle and Miniplasmid DNA Vectors : The Future of Non-Viral and Viral Gene Transfer Weinheim : John Wiley & Sons, Incorporated,c2013 9783527324569
Subject Gene therapy.;Genetic vectors.;Plasmids
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