| Description |
1 online resource (521 pages) |
| Contents |
Book Cover -- Half-Title -- Title -- Copyright -- Dedication -- Contents -- Color plates -- Figures -- Tables -- Contributors -- Foreword -- Preface -- 1 Basic components of gene expression plasmids -- INTRODUCTION -- ESSENTIAL ELEMENTS OF EXPRESSION PLASMID VECTORS -- TRANSCRIPTIONAL REGULATORY ELEMENTS -- CHROMOSOMAL ELEMENTS -- RNA PROCESSING -- TRANSCRIPTIONAL TERMINATION SEQUENCES -- STABILITY OF TRANSCRIPTS -- TRANSLATION CONTROL -- MULTIPLE GENE PRODUCTS FROM A SINGLE mRNA -- MULTIPLE CLONING SITE OR POLYLINKER -- PERSISTENCE OF GENE EXPRESSION -- CONCLUDING REMARKS -- REFERENCES -- 2 Inducible gene expression strategies in gene therapy -- INTRODUCTION -- GENERAL APPROACHES -- INDUCIBLE GENE EXPRESSION SYSTEMS -- STRESS INDUCIBLE PROMOTERS -- HEAT SHOCK PROMOTERS -- IONIZING RADIATION INDUCIBLE PROMOTERS -- HYPOXIA INDUCIBLE PROMOTERS -- REGULATED EXPRESSION PROMOTERS -- Metabolic genes -- Metallothionein -- Tetracycline/Doxycycline 'gene switches' -- COMBINATION STRATEGIES -- LEAKY VERSUS TIGHTLY CONTROLLED EXPRESSION -- CONCLUDING REMARKS -- ACKNOWLEDGMENT -- REFERENCES -- 3 Modulation of gene expression by antisense oligonucleotides -- INTRODUCTION -- MECHANISMS OF ACTION OF ANTISENSE OLIGONUCLEOTIDES -- Hybrid-arrest -- Cleavage of target sequences -- ANTISENSE CHEMISTRY -- Antisense RNA -- Nuclear expression of RNA by engineered antisense genes -- Microinjection of in vitro transcribed RNA -- Liposomal encapsulation of RNA -- Antisense DNA -- Unmodified phosphodiester oligodeoxynucleotides -- Oligonucleotide analogs with backbone modifications -- Oligonucleotides with terminal modification -- Mixed-backbone oligonucleotides (MBOs) -- Uptake of antisense oligonucleotides -- TARGET VALIDATION OF ANTISENSE OLIGONUCLEOTIDES -- Design of antisense oligonucleotides -- Random 'sequence-walking' approach |
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Computer-aided target selection: computer folding of mRNA -- Oligonucleotide scanning arrays -- Oligonucleotide library/RNase H digestion-based screening -- Other methods of selecting antisense oligonucleotides -- Pharmacological evaluation of antisense oligonucleotides -- ANTI-MDM2 ANTISENSE OLIGONUCLEOTIDES AS ANTITUMOR AGENTS -- MDM2 as a target for cancer therapy -- PS-oligonucleotides targeted to MDM2 -- Design of anti-MDM2 oligonucleotides -- Anti-MDM2 oligos activate RNase H -- Anti-MDM2 oligos activate p53 and induce apoptosis -- Optimization of anti-MDM2 oligos -- Mixed-backbone antisense oligonucleotides targeted to MDM2 -- Design of anti-MDM2 MBO -- In vitro and in vivo activities -- CONCLUDING REMARKS -- ACKNOWLEDGMENT -- REFERENCES -- 4 Ribozymes as therapeutic agents and genetic tools -- INTRODUCTION -- CATALYTIC MOTIFS -- RIBOZYME APPLICATIONS -- OPTIMIZING INTRACELLULAR FUNCTION OF RIBOZYMES -- RIBOZYME DELIVERY -- FUNCTIONAL GENOMICS AND TARGET VALIDATION -- TRANSGENIC ANIMALS EXPRESSING RIBOZYMES -- RIBOZYME-MEDIATED RNA REPAIR -- RIBOZYME-BASED GENE DISCOVERY -- RIBOZYME EVOLUTION -- OTHER CONSIDERATIONS -- FUTURE PROSPECTS -- ACKNOWLEDGMENTS -- REFERENCES -- 5 Peptide nucleic acids (PNA) binding-mediated target gene transcription -- INTRODUCTION -- PNA BINDING AFFINITY -- PNA BINDING SPECIFICITY -- DETECTION OF PNA BINDING-INDUCED TRANSCRIPTION IN VITRO -- DETERMINATION OF TRANSCRIPTION INITIATION SITES OF PNA BINDING-INDUCED TRANSCRIPTION -- PNA BINDING GENERATED D-LOOPS LEAD TO GFP GENE EXPRESSION IN MAMMALIAN CELLS -- PNA-INDUCED ENDOGENOUS -GLOBIN GENE EXPRESSION IN HUMAN CELLS -- CORRELATION BETWEEN PNA BINDING GENERATED D-LOOPS AND NATURAL PROMOTER IN TARGET GENE TRANSCRIPTION -- PNA LENGTH REQUIREMENT FOR INDUCING TRANSCRIPTION FROM PNA BINDING SITES |
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TRANSCRIPTION COMPONENTS INVOLVED IN PNA BINDING-INDUCED TRANSCRIPTION -- LIMITATION OF PNA BINDING-INDUCED TARGET GENE EXPRESSION -- CONCLUDING REMARKS -- REFERENCES -- 6 Aptamers for controlling gene expression -- INTRODUCTION -- SELECTING THE SHAPE -- APTAMERS TO NUCLEIC ACID-BINDING PROTEINS -- Polymerases -- Nucleases -- Transcription factors -- Translation and splicing factors -- Control of gene expression by aptamer-protein complexes -- APTAMERS TO NUCLEIC ACIDS -- CHEMICALLY-MODIFIED APTAMERS -- CONCLUDING REMARKS -- ACKNOWLEDGMENTS -- REFERENCES -- 7 Enzymes acting on DNA breaks and its relevance in nucleic acid-based therapy -- INTRODUCTION -- DNA BREAKAGE AND REPAIR -- ENZYMES MODIFYING DNA BREAKS -- Flap endonuclease-1 (FEN-1) -- 3'-Exonuclease -- DNA polymerases and DNA ligases -- POLY(ADP-RIBOSE) POLYMERASE-1 (PARP-1) -- Architecture of PARP-1 -- PARP-1 and DNA breaks -- Association of PARP-1 to RNA stem-loops and regulation of transcription in response to DNA damage -- Cell death and PARP-1 -- CONCLUDING REMARKS -- REFERENCES -- 8 Compaction and condensation of DNA -- INTRODUCTION -- SWITCHING ON THE CONFORMATION OF DNA -- COMPACTION BY MULTI-VALENT CATIONS -- COMPACTION BY POLYMER -- COMPACTION BY CATIONIC LIPIDS AND SURFACTANTS -- MORPHOLOGICAL VARIATION IN COMPACT DNA -- SINGLE CHAIN COMPACTION AND MULTI-CHAIN ASSEMBLING -- GENERATION OF LARGE SCALE COOPERATION BY NON-SPECIFIC INTERACTION -- CROSS-TALK BETWEEN FOLDING TRANSITION AND HELIX-COIL TRANSITION -- MANIPULATING INDIVIDUAL DNA -- CONCLUDING REMARKS -- REFERENCES -- 9 Structure, dispersion stability and dynamics of DNA and polycation complexes -- INTRODUCTION -- POLYCATIONS USED FOR PREPARATION OF POLYPLEXES -- FORMATION OF THE COMPLEXES -- Polyion coupling reaction -- Composition of the complex -- Disproportionation -- STABILITY OF THE COMPLEXES IN DISPERSION |
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A core-shell model for positively charged complexes -- Block ionomer complexes -- Effect of proteins -- STRUCTURE AND PROPERTIES OF DNA IN THE COMPLEXES -- Morphology and conformation in DNA/polycation complexes -- Stabilization of DNA secondary structures by polycations -- Stabilization of DNA against nuclease digestion -- POLYION INTERCHANGE AND RECOGNITION PHENOMENA -- Polyion interchange reactions in DNA containing complexes -- Recognition of DNA topology by cationic copolymers -- CONCLUDING REMARKS -- ACKNOWLEDGMENTS -- REFERENCES -- 10 Structure and structure-activity correlations of cationic lipid/DNA complexes: supramolecular assembly and gene delivery -- INTRODUCTION -- LAMELLAR PHASE OF CL/DNA COMPLEXES -- THE INVERTED HEXAGONAL PHASE OF CATIONIC LIPOSOM/DNA COMPLEXES: PATHWAYS FROM LAMELLAR PHASE -- INTERACTIONS BETWEEN LAMELLAR AND INVERTED HEXAGONAL PHASE OF CL/DNA COMPLEXES AND ANIONIC GIANT LIPOSOMES MIMICKING THE CELL PLASMA MEMBRANE -- INTERACTIONS BETWEEN LAMELLAR AND INVERTED HEXAGONAL PHASE OF CL/DNA COMPLEXES AND MOUSE FIBROBLAST CELLS -- CONCLUDING REMARKS -- ACKNOWLEDGEMENTS -- REFERENCES -- 11 Cellular uptake, metabolic stability and nuclear translocation of nucleic acids -- INTRODUCTION -- INTERNALIZATION AND ENTRAPMENT IN ENDO-LYSOSOMAL COMPARTMENT -- CYTOPLASM AS A PHYSICAL AND METABOLIC BARRIER -- Diffusional constraint of plasmid mobility in cytoplasm -- Metabolic instability of plasmid in cytoplasm -- NUCLEAR UPTAKE OF PLASMID -- NUCLEOCYTOPLASMIC TRAFFICKING OF OLIGONUCLEOTIDES -- NUCLEOCYTOPLASMIC TRANSPORT OF VIRUSES -- Virus trafficking in cytoplasm -- Nuclear entry of viruses -- CONCLUDING REMARKS -- ACKNOWLEDGMENTS -- REFERENCES -- 12 Nucleocytoplasmic trafficking -- INTRODUCTION -- BARRIERS TO NUCLEAR TRANSPORT -- Mechanisms of nuclear transport -- Nuclear envelope -- Cell cycle -- NUCLEAR TRANSPORT |
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Nuclear entry of plasmids in the absence of cell division -- Sequence specific nuclear uptake -- Nuclear import in permeabilized cells -- Requirement of nuclear import sequences in the absence of cell division -- Effect of nuclear import sequences on transfection -- Sequence-specific nuclear import of plasmids in vivo -- Cell-specific nuclear import -- Nuclear import of exogenous DNA/protein complexes -- Alternative pathways of DNA nuclear uptake -- APPROACHES TO INCREASE NUCLEAR TARGETING OF DNA -- Non-covalent attachment of proteins -- Non-covalent peptide/DNA complexes -- Covalent attachment of peptides to DNA -- Peptide nucleic acids and their use -- OTHER OBSTACLES IN TRANSPORT TO NUCLEUS -- CONCLUDING REMARKS -- ACKNOWLEDGMENTS -- REFERENCES -- 13 Optimizing RNA export for transgene expression -- INTRODUCTION -- FUNDAMENTALS OF RNA EXPORT -- hnRNPs -- TRANSPORT RECEPTORS -- NUCLEAR PORE COMPLEXES (NPC) -- NUCLEAR PORE PROTEINS (NUCLEOPORINS) -- MULTIPLE RNA EXPORT PATHWAYS -- REV-MEDIATED EXPORT OF VIRAL RNA -- CTE-MEDIATED EXPORT OF VIRAL RNAs -- NUCLEAR EXPORT OF INTRONLESS VIRAL MESSAGES -- NUCLEAR EXPORT OF mRNAs -- ROLE OF RNA EXPORT IN GENE THERAPY -- CONCLUDING REMARKS -- REFERENCES -- 14 Naked plasmid DNA delivery of a therapeutic protein -- INTRODUCTION -- IN VIVO TRANSFECTION OF MUSCLE USING NAKED pDNA -- MECHANISM OF pDNA ENTRY INTO MUSCLE TISSUE -- IN VIVO TRANSFECTION OF OTHER TISSUES BY NAKED pDNA -- CLINICAL APPLICATIONS OF NAKED pDNA -- Biological activity of naked pDNA -- pDNA therapy of viral infection -- pDNA therapy of autoimmune diseases -- pDNA therapy of cancer -- IMPROVEMENTS IN pDNA VECTORS -- Vector design and vehicles -- Electroporation -- SAFETY OF NAKED pDNA -- Preclinical safety studies -- Clinical trials of pDNA delivery of a therapeutic protein -- CONCLUDING REMARKS -- REFERENCES |
| Summary |
Gene Expression. Modulation of Gene Expression. Biophysical Aspects of Nucleic Acids and Carriers. Intracellular Trafficking. Nucleic Acid Delivery Systems. Biodistribution and Pharmacokinetics. Clinical Applications of Gene Therapy |
| Notes |
15 Cationic lipid-based gene delivery |
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Description based on publisher supplied metadata and other sources |
| Subject |
Gene therapy.
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Nucleic acids -- Therapeutic use
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Genetic Therapy
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Gene therapy
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Nucleic acids -- Therapeutic use
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| Form |
Electronic book
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| Author |
Kim, Sung Wan
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| ISBN |
9780203300961 |
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0203300963 |
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