Minicircle and miniplasmid DNA vectors: the future of non-viral and viral gene transfer
Gespeichert in:
| Format: | Buch |
|---|---|
| Sprache: | English |
| Veröffentlicht: |
Weinheim
Wiley-Blackwell
2013
|
| Schlagworte: | |
| Online-Zugang: | Inhaltstext Inhaltsverzeichnis |
| Beschreibung: | Literaturangaben |
| Beschreibung: | XXI, 235 S. Ill., graph. Darst. 240 mm x 170 mm |
| ISBN: | 3527324569 9783527324569 |
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IMAGE 1
V I I
CONTENTS
LIST O F CONTRIBUTORS XIII PERFACE XXI
1 MINICIRCLE PATENTS: A SHORT IP OVERVIEW O F OPTIMIZING NONVIRAL DNA
VECTORS 1
MARTIN CRUND AND MARTIN SCHLEEF REFERENCES 6
2 OPERATOR-REPRESSOR TITRATION: STABLE PLASMID MAINTENANCE WITHOUT
SELECTABLE MARKER GENES 7 ROCKY M. CRANENBURGH 2.1 INTRODUCTION 7
2.2 ANTIBIOTICS A N D METABOLIC BURDEN 7
2.3 THE MECHANISM O F ORT 8
2.4 ORT STRAIN DEVELOPMENT 9
2.5 ORT MINIPLASMIDS 12
2.6 DNA VACCINE AND GENE THERAPY VECTORS 13
2.7 ORT-VAC: PLASMID-BASED VACCINE DELIVERY USING SALMONELLA ENTERICA 14
2.8 RECOMBINANT PROTEIN EXPRESSION 18
2.9 CONCLUSIONS A N D FUTURE DEVELOPMENTS 19
REFERENCES 19
3 SELECTION BY RNA-RNA INTERACTION: MAXIMALLY MINIMIZED ANTIBIOTIC
RESISTANCE-FREE PLASMIDS 23 JUERGEN MAIRHOFER AND REINGARD CRABHERR 3.1
GENE THERAPY A N D DNA VACCINES: EMERGING TECHNOLOGIES 23
3.1.1 THERAPEUTIC PLASMIDS: GENERAL DESIGN PRINCIPLES 24 3.2 THERAPEUTIC
PLASMIDS: NOVEL DESIGN AND THE PROBLEM O F SELECTION 2 5 3.2.1
REPLICATION CONTROL O F COLEL-TYPE PLASMIDS AS A N ALTERNATIVE SELECTION
MARKER 26
3.2.2 THE MINIBACK CONCEPT: SELECTION BY RNA-RNA INTERACTION 28 3.2.3
IMPROVED PRODUCTION PROCESSES BY MINIBACK PLASMIDS 3 0
HTTP://D-NB.INFO/1027115829
IMAGE 2
V I I I CONTENTS
3.2.4 IMPROVING SEQUENCE COMPOSITION 32
3.2.5 EFFICIENT GENE TRANSFER 32
3.3 CONCLUSIONS 33
ACKNOWLEDGMENTS 33 REFERENCES 33
4 PLASMID-BASED MEDICINAL PRODUCTS - FOCUS ON PFAR: A MINIPLASMID FREE O
F ANTIBIOTIC RESISTANCE MARKERS 3 7 CORINNE MARIE, MICKA'EL QUIVIGER,
HELEN FOSTER, GEORGE DICKSON, AND DANIEL SCHERMAN 4.1 INTRODUCTION:
RATIONALE FOR THE DEVELOPMENT O F BIOSAFE DNA PLASMID
VECTORS 37
4.2 SPECIFIC REQUIREMENTS FOR THE USE O F DNA PRODUCT AS MEDICINES 39
4.2.1 REQUIREMENTS FOR PLASMID QUALITY AND PURITY 39 4.2.2 REQUIREMENTS
FOR THE REMOVAL O F ANTIBIOTIC RESISTANCE MARKERS FROM PLASMID DNA 4 0
4.2.2.1 REQUIREMENTS FOR BIOSAFE PLASMIDS 4 0 4.2.2.2 POSITIVE IMPACT O
N THE REMOVAL O F ANTIBIOTIC RESISTANCE MARKERS 41 4.2.2.3 EFFECT O F
PLASMID SIZE O N GENE TRANSFER EFFICIENCY IN VITRO A N D IN VIVO 41
4.3 NONVIRAL GENE VECTORS DEVOID O F ANTIBIOTIC RESISTANCE MARKERS 43
4.3.1 GENERALITIES 43
4.3.2 SELECTION SYSTEMS DEVOID O F ANTIBIOTIC RESISTANCE MARKERS 43
4.3.2.1 COMPLEMENTATION O F HOST AUXOTROPHY BY A FUNCTION-ENCODED
PLASMID 43
4.3.2.2 THE OPERATOR-REPRESSOR TITRATION (ORT) SYSTEM 44 4.3.2.3
PROTEIN-BASED ANTIDOTE/POISON SELECTION SYSTEMS 44 4.3.2.4 RNA-BASED
SELECTION MARKER 44
4.3.2.5 SUPPRESSION O F A NONSENSE MUTATION 45 4.4 THE PFAR PLASMID
FAMILY 46
4.4.1 DESCRIPTION O F THE ANTIBIOTIC-FREE SELECTION SYSTEM 46 4.4.2 PFAR
VECTORS PROMOTE EFFICIENT EXPRESSION I N SEVERAL TYPES O F MAMMALIAN
CELLS 4 9
4.4.2.1 IN VITRO TRANSFECTION STUDY 49 4.4.2.2 IN VIVO TRANSFECTION
STUDIES 50 4.4.3 CONCLUDING REMARKS O N THE PFAR4 BIOSAFE MINIPLASMID 51
4.5 CONCLUDING REMARKS AND PERSPECTIVES 52
ACKNOWLEDGMENTS 53 REFERENCES 53
5 PLASMID DNA CONCATEMERS: INFLUENCE O F PLASMID STRUCTURE ON
TRANSFECTION EFFICIENCY 59 CHRISTOF MAUCKSCH, BRONWEN CONNOR, AND
CARSTEN RUDOLPH 5.1 INTRODUCTION 59
5.2 PLASMID DNA TOPOLOGY A N D SIZE 60
IMAGE 3
CONTENTS I X
5.3 PLASMID DNA CONCATEMERS 62
5.4 CONCLUSIONS 67
ACKNOWLEDGMENTS 6 7 REFERENCES 68
6 ANALYTICAL TOOLS IN MINICIRDE PRODUCTION 71
ANJA RISCHMULIER, MARTINA VIEJHUES, MAREIKE DIEDING, MARKUS BLAESEN,
MARCO SCHMEER, RUTH BAIER, DARIO ANSELMETTI, AND MARTIN SCHLEEF 6.1
INTRODUCTION 71
6.1.1 GENE TRANSFER FOR THERAPY, VACCINATION, A N D STEM CELLS 71 6.1.2
PLASMIDS 72
6.1.3 MINICIRCLE SYSTEMS 73
6.2 PRODUCTION O F MINICIRCLES 74
6.2.1 THE PARENTAL PLASMID 74
6.2.2 CULTIVATION A N D INDUCTION 74
6.2.3 MINICIRCLE PREPARATION 77
6.3 ANALYTICS O F MINICIRCLE PRODUCTION 79
6.3.1 IN-PROCESS CONTROL 79
6.3.1.1 ATOMIC FORCE MICROSCOPY 79 6.3.1.2 CAPILLARY GEL ELECTROPHORESIS
81 6.3.1.3 CONTINUOUS FLOW SEPARATION I N MICROFLUIDIC CHANNELS 82 6.3.2
FINISHED PRODUCT CONTROL 86
6.4 FUTURE GOALS 8 8
ACKNOWLEDGMENTS 88 REFERENCES 89
7 UTILIZING MINICIRCLE VECTORS FOR THE EPISOMAL MODIFICATION O F CELLS
93 ORESTIS ARGYROS, SUET-PING WONG, CHARLES COUTELLE, AND RICHARD P.
HARBOTTLE 7.1 INTRODUCTION 93
7.2 STUDIES THAT SHOW PASSIVE EPISOMAL MAINTENANCE O F MINICIRCLES IN
VIVO 94
7.3 PRINCIPLES O F GENERATING MINICIRCLE VECTORS ABLE TO SUPPORT
EPISOMAL MAINTENANCE 97 7.3.1 EPISOMAL MAINTENANCE O F MINICIRCLE S/MAR
VECTORS GENERATED BY FLP RECOMBINASE IN VITRO 98 7.3.2 EPISOMAL
MAINTENANCE O F MINICIRCLE S/MAR VECTORS GENERATED USING
CRE RECOMBINASE IN VITRO 99 7.3.3 EPISOMAL MAINTENANCE O F S/MAR VECTORS
I N BOVINE AND MURINE ZYGOTES 101 7 A EPISOMAL MAINTENANCE O F S/MAR
MINICIRCLES IN VIVO 102
7.5 POTENTIAL O F EPISOMAL REPLICATION O F S/MAR MINICIRCLE VECTORS 104
7.6 POSSIBLE MECHANISMS PROMOTING THE EPISOMAL MAINTENANCE O F
MINICIRCLE VECTORS 105 7.6.1 HISTONE MODIFICATIONS 106
7.6.2 CPG DINUCLEOTIDE CONTENT REDUCTION 106
IMAGE 4
X | CONTENTS
7.6.3 VECTOR ESTABLISHMENT I N THE CORRECT NUCLEAR COMPARTMENT 107
7.6.4 ACCESS TO REPLICATION MACHINERY BY S/MARS 107 7.7 CONCLUSIONS 110
REFERENCES 110
8 REPLICATING MINICIRDES: OVERCOMING T H E LIMITATIONS O F TRANSIENT AND
STABLE EXPRESSION SYSTEMS 115 KRISTINA NEHLSEN, SANDRA BROIL, RAJU
KANDIMALLA, NIELS HEINZ, MARKUS HEINE, STEFANIE BINIUS, AXEL SCHAMBACH,
ANDJURGEN BODE 8.1 GENE THERAPY; THE ADVENT O F NOVEL VECTOR VEHICLES
115 8.1.1 NONVIRAL VECTORS AVOIDING GENOMIC DISTURBANCES 116 8.1.2
INDEPENDENT EXPRESSION UNITS: CHROMATIN DOMAINS 116 8.1.2.1 S/MARS: A
UNIFYING PRINCIPLE 118 8.1.2.2 S/MAR ACTIONS ARE MULTIFOLD A N D CONTEXT
DEPENDENT 119 8.1.2.3 STRESS-INDUCED DUPLEX DESTABILIZATION: A UNIFYING
PROPERTY
O F S/MARS 121
8.1.2.4 CHROMOSOME-BASED EXPRESSION STRATEGIES: EPISOMES AND/OR
PREDETERMINED INTEGRATION SITES (RMCE) 123 8.2 REPLICATING NONVIRAL
EPISOMES 123
8.2.1 CAN THE YEAST ARS PRINCIPLE BE VERIFIED FOR MAMMALIAN CELLS? 125
8.2.2 ARS AND S/MARS: C O M M O N (SIDD-) PROPERTIES 125 8.2.3 S/MAR
PLASMIDS: VERIFICATION O F THE CONCEPT 126 8.2.3.1 TRANSCRIPTION INTO
THE S/MAR: DIRECTIONALITY A N D RATE 126 8.2.3.2 CELL A N D NUCLEAR
PERMEATION 128 8.2.3.3 NUCLEAR ASSOCIATION SITES 129 8.2.3.4 RMCE-BASED
ELABORATION FOLLOWING ESTABLISHMENT 130 8.2.4 REMAINING SHORTCOMINGS AND
THEIR SOLUTION 132
8.2.4.1 ESTABLISHMENT AND MAINTENANCE: THE EBV PARADIGM 132 8.2.4.2
VECTOR SIZE LIMITATIONS 136 8.3 MINIMALIZATION APPROACHES 137
8.3.1 OLIGOMERIZING S/MAR MODULES: PMARS A N D ITS PROPERTIES 139 8.3.2
REPLICATING MINICIRCLES: A SOLUTION WITH GREAT PROMISE 140 8.3.2.1
ESTABLISHMENT AND MAINTENANCE PARAMETERS 141 8.3.2.2 CLONAL BEHAVIOR 141
8.3.2.3 BI-MC SYSTEMS 143
8.3.2.4 MC SIZE REDUCTION: "IN VIVO EVOLUTION" 144 8.3.2.5
TRANSCRIPTIONAL TERMINATION AND POLYADENYLATION: A N INTRICATE INTERPLAY
146 8.3.2.6 EPISOMAL STATUS: PROOF A N D PERSISTENCE 147
8.3.3 EMERGING EXTENSIONS AND REFINEMENTS 149 8.3.3.1 COMBINATION O F
EXCISION AND RMCE STRATEGIES 151 8.3.3.2 MC WITHDRAWAL AT WILL 153
8.3.3.3 PRONUCLEAR INJECTION A N D SOMATIC CELL NUCLEAR TRANSFER 155
8.3.3.4 FROM CELLS TO ORGANS 155
IMAGE 5
CONTENTS X I
8.4 SUMMARY A N D OUTLOOK 156
ACKNOWLEDGMENTS 157 REFERENCES 158
9 MAGNETOFECTION O F MINICIRCLE DNA VECTORS 165
FLAVIE SICARD, CEDRIC SAPET, NICOLAS LAURENT, ELODIE BERTOSIO, MELANIE
BERTUZZI, AND OLIVIER ZELPHATI 9.1 INTRODUCTION 165
9.2 OVERVIEW O F MAGNETOFECTION PRINCIPLES 167
9.3 CELLULAR UPTAKE 168
9.4 DIFFUSION THROUGH THE CYTOPLASM 169
9.5 TRANSGENE EXPRESSION 169
9.6 CONCLUSIONS 172
REFERENCES 173
10 MINICIRCLE-BASED VECTORS FOR NONVIRAL GENE THERAPY: IN VITRO
CHARACTERIZATION AND IN VIVO APPLICATION 177 DENNIS KOBELT,JUTTA AUMANN,
MARTIN SCHLEEF, MARCO SCHMEER, ULRIKE STEIN, PETER M. SCHLAG, AND
WOLFGANG WALTHER 10.1 MINICIRCLE TECHNOLOGY FOR NONVIRAL GENE THERAPY
177 10.2 CURRENT STATUS O F IN VIVO APPLICATION O F MINICIRCLE VECTORS
178 10.3 JET INJECTION TECHNOLOGY FOR IN VIVO TRANSFER O F NAKED DNA 180
* 10.4 COMPARATIVE PERFORMANCE ANALYSES O F MINICIRCLE VECTORS 183
10.5 IN VIVO APPLICATION O F MINICIRCLE DNA BY JET INJECTION 185
REFERENCES 186
11 EPISOMAL EXPRESSION O F MINICIRCLES AND CONVENTIONAL PLASMIDS IN
MAMMALIAN EMBRYOS 189 WIEBKE CARRELS, KHURSHEED IQBAL, AND WILFRIED A.
KUES 11.1 INTRODUCTION 189
11.2 FATE O F PLASMIDS AND MINICIRCLES AFTER INJECTION INTO MAMMALIAN
EMBRYOS 191 11.2.1 MINICIRCLE- A N D PLASMID-MEDIATED EXPRESSION I N
EARLY EMBRYOS AND FETUSES 191 11.2.2 EXPRESSION O F FUNCTIONAL GENES I N
PREIMPLANTATION EMBRYOS 195
11.3 DISCUSSION 198
REFERENCES 299
12 TISSUE-TARGETED GENE ELECTRODELIVERY O F MINICIRCLE DNA 203 SOPHIE
CHABOT, MURIEL COLZIO, AND JUSTIN TEISSIE 12.1 INTRODUCTION 203
12.2 PLASMID DNA ELECTROTRANSFEN FROM PRINCIPLE TO TECHNICAL DESIGN 204
12.2.1 MECHANISM O F GENE ELECTROTRANSFER 204 12.2.2 PRECLINICAL
APPLICATIONS 205 12.3 IMPLEMENTATION FOR EFFICIENT TISSUE-TARGETED GENE
DELIVERY 206
IMAGE 6
X I I | CONTENTS
12.3.1 DESIGN O F DNA VECTOR 206
12.3.2 IN VITRO MINICIRCLE ELECTROTRANSFER 206 12.3.3 IN VIVO MC
ELECTROTRANSFER 207 12.3.3.1 MUSCLE 207
12.3.3.2 TUMOR 208
12.3.3.3 SKIN 209
12.4 CONCLUSIONS 209
ACKNOWLEDGMENTS 209 REFERENCES 210
13 INCREASED EFFICIENCY O F MINICIRDES VERSUS PLASMIDS UNDER GENE
ELECTROTRANSFER SUBOPTIMAL CONDITIONS: AN INFLUENCE O F THE
EXTRACELLULAR MATRIX 215 VANESSA JOUBERT, FRANCK M. ANDRE, MARCO
SCHMEER, MARTIN SCHLEEF,
AND LLUIS M. MIR
13.1 INTRODUCTION 215
13.2 METHODS 215
13.2.1 CELL CULTURE AND ANIMALS 215
13.2.2 MINICIRCLE AND PLASMID 216
13.2.3 ELECTROTRANSFER 216
13.2.4 DETERMINATION O F THE REPORTER GENE (LUCIFERASE) ACTIVITY 216
13.2.5 DATA ANALYSIS 217
13.3 RESULTS 217
13.3.1 IN VITRO 217
13.3.2 IN VIVO 218
13.4 DISCUSSION 220
13.5 CONCLUSIONS 223
ACKNOWLEDGMENTS 224 REFERENCES 224
INDEX 227 |
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| spelling | Minicircle and miniplasmid DNA vectors the future of non-viral and viral gene transfer ed. by Martin Schleef Weinheim Wiley-Blackwell 2013 XXI, 235 S. Ill., graph. Darst. 240 mm x 170 mm txt rdacontent n rdamedia nc rdacarrier Literaturangaben Vektor Genetik (DE-588)4279246-0 gnd rswk-swf (DE-588)4143413-4 Aufsatzsammlung gnd-content Vektor Genetik (DE-588)4279246-0 s DE-604 Schleef, Martin Sonstige (DE-588)1034891375 oth text/html http://deposit.dnb.de/cgi-bin/dokserv?id=4166012&prov=M&dok_var=1&dok_ext=htm Inhaltstext DNB Datenaustausch application/pdf http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=025999408&sequence=000001&line_number=0001&func_code=DB_RECORDS&service_type=MEDIA Inhaltsverzeichnis |
| spellingShingle | Minicircle and miniplasmid DNA vectors the future of non-viral and viral gene transfer Vektor Genetik (DE-588)4279246-0 gnd |
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| title | Minicircle and miniplasmid DNA vectors the future of non-viral and viral gene transfer |
| title_auth | Minicircle and miniplasmid DNA vectors the future of non-viral and viral gene transfer |
| title_exact_search | Minicircle and miniplasmid DNA vectors the future of non-viral and viral gene transfer |
| title_full | Minicircle and miniplasmid DNA vectors the future of non-viral and viral gene transfer ed. by Martin Schleef |
| title_fullStr | Minicircle and miniplasmid DNA vectors the future of non-viral and viral gene transfer ed. by Martin Schleef |
| title_full_unstemmed | Minicircle and miniplasmid DNA vectors the future of non-viral and viral gene transfer ed. by Martin Schleef |
| title_short | Minicircle and miniplasmid DNA vectors |
| title_sort | minicircle and miniplasmid dna vectors the future of non viral and viral gene transfer |
| title_sub | the future of non-viral and viral gene transfer |
| topic | Vektor Genetik (DE-588)4279246-0 gnd |
| topic_facet | Vektor Genetik Aufsatzsammlung |
| url | http://deposit.dnb.de/cgi-bin/dokserv?id=4166012&prov=M&dok_var=1&dok_ext=htm http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=025999408&sequence=000001&line_number=0001&func_code=DB_RECORDS&service_type=MEDIA |
| work_keys_str_mv | AT schleefmartin minicircleandminiplasmiddnavectorsthefutureofnonviralandviralgenetransfer |