Ebook: Non-viral Gene Therapy: Gene Design and Delivery
- Tags: Cell Biology, Biotechnology, Molecular Medicine, Genetic Engineering, Biochemical Engineering, Medical Biochemistry
- Year: 2005
- Publisher: Springer Tokyo
- Edition: 1
- Language: English
- pdf
The development of gene medicine based on the concept of molecular therapy has opened new medical horizons. Among the many techniques in gene medicine, gene design and delivery are especially significant in clinical applications. This book presents state-of-the-art information on non-viral gene-delivery techniques, covering a broad spectrum of disciplines that include chemistry, molecular biology, cell biology, and pharmacokinetics. Major sections introduce molecules for gene delivery and their properties; technologies of controlled gene delivery in vitro and in vivo; therapeutic genes and the status of clinical applications; and the design of genes based on current RNA technology, with revolutionary developments in the world of RNAi shown to be key factors in gene medicine. This groundbreaking work is an invaluable resource for researchers and engineers in genetic engineering, molecular medicine, biochemical engineering, and biotechnology.
The development of gene medicine based on the concept of molecular therapy has opened new medical horizons. Among the many techniques in gene medicine, gene design and delivery are especially significant in clinical applications. This book presents state-of-the-art information on non-viral gene-delivery techniques, covering a broad spectrum of disciplines that include chemistry, molecular biology, cell biology, and pharmacokinetics. Major sections introduce molecules for gene delivery and their properties; technologies of controlled gene delivery in vitro and in vivo; therapeutic genes and the status of clinical applications; and the design of genes based on current RNA technology, with revolutionary developments in the world of RNAi shown to be key factors in gene medicine. This groundbreaking work is an invaluable resource for researchers and engineers in genetic engineering, molecular medicine, biochemical engineering, and biotechnology.
Content:
Front Matter....Pages I-XII
Front Matter....Pages 1-1
Recent Progress in Non-viral Gene Delivery....Pages 3-10
Physical Chemistry of DNA-Carrier Complexes....Pages 11-18
Liposomes....Pages 19-34
Polymeric Gene Carriers....Pages 35-50
Development of a Supramolecular Nanocarrier for Gene Delivery Based on Cationic Block Copolymers....Pages 51-62
Chitosan....Pages 63-74
Dendrimers as DNA Carriers....Pages 75-86
Use of Synthetic Peptides for Non-viral Gene Delivery....Pages 87-102
An Oligonucleotide Carrier Based on ?-1,3-Glucans....Pages 103-117
Biological and Chemical Hybrid Vectors....Pages 118-132
Front Matter....Pages 133-133
Pharmacokinetics of Gene Delivery in Cells....Pages 135-154
Active DNA Release from Complexes....Pages 155-164
Controlled Release of DNA Using Thermoresponsive Polymers....Pages 165-175
Active Transport of Exogenous Genes into the Nucleus....Pages 176-186
Controlled Intracellular Localization of Oligonucleotides by Chemical Conjugation....Pages 187-197
Functional Nucleotide Sequences Capable of Promoting Non-viral Genetic Transfer....Pages 198-210
Shielding of Cationic Charge of the DNA Complex to Avoid Nonspecific Interactions for In Vivo Gene Delivery....Pages 211-225
In Vivo Gene Transfer by Ligand-Modified Gene Carriers....Pages 226-236
Optimizing Polyplexes into Synthetic Viruses for Tumor-Targeted Gene Therapy....Pages 237-245
Gene Transfer and Target Diseases....Pages 246-260
Front Matter....Pages 133-133
Clinical Trials Using Non-viral Gene Delivery Systems....Pages 261-290
Front Matter....Pages 291-291
Evaluation of Size and Zeta Potential of DNA/Carrier Complexes....Pages 293-299
Observation of DNA/Carrier Complexes Under Fluorescence Microscopy....Pages 300-306
Synthesis of Chemically Modified Chitosan and a Study of Its Gene Transfection Efficiency....Pages 307-314
Newly Designed DNA Fragments for Gene Correction....Pages 315-322
Evaluation of Gene Expression In Vivo After Intravenous and Intraportal Administration of Lipoplexes....Pages 323-330
Evaluation by Southern Blot Hybridization of DNA Administered with a Gene Carrier to Organs....Pages 331-338
Evaluation of the Immune Response After Administration of Plasmid DNA-Non-viral Vector Complexes....Pages 339-347
Gateway RNAi....Pages 348-360
Front Matter....Pages 361-361
Design of Intracellularly Active Ribozymes and siRNAs....Pages 363-397
RNAi-Based Inhibition Specific for Mutant Alleles in Autosomal Dominant Diseases: Sequence-Dependent and -Independent Discrimination of Mutant and Wild-Type Alleles by siRNA....Pages 398-404
In Vivo RNA Interference: Another Tool in the Box?....Pages 405-422
Suppression of Gene Expression via Chromatin Remodeling and the siRNA-Induced Silencing of Transcription....Pages 423-440
Intracellular Delivery of Nucleic Acids: Differences Between Transfection and siFection Reflect Differences Between DNA and RNA, and Between Oligodeoxynucleotides and Oligonucleotides....Pages 441-455
In Vivo Antitumor Activity of a New Cationic Liposome siRNA Complex....Pages 456-464
Enhancing RNAi with Synthetic RNA Duplexes....Pages 465-475
Back Matter....Pages 477-487
The development of gene medicine based on the concept of molecular therapy has opened new medical horizons. Among the many techniques in gene medicine, gene design and delivery are especially significant in clinical applications. This book presents state-of-the-art information on non-viral gene-delivery techniques, covering a broad spectrum of disciplines that include chemistry, molecular biology, cell biology, and pharmacokinetics. Major sections introduce molecules for gene delivery and their properties; technologies of controlled gene delivery in vitro and in vivo; therapeutic genes and the status of clinical applications; and the design of genes based on current RNA technology, with revolutionary developments in the world of RNAi shown to be key factors in gene medicine. This groundbreaking work is an invaluable resource for researchers and engineers in genetic engineering, molecular medicine, biochemical engineering, and biotechnology.
Content:
Front Matter....Pages I-XII
Front Matter....Pages 1-1
Recent Progress in Non-viral Gene Delivery....Pages 3-10
Physical Chemistry of DNA-Carrier Complexes....Pages 11-18
Liposomes....Pages 19-34
Polymeric Gene Carriers....Pages 35-50
Development of a Supramolecular Nanocarrier for Gene Delivery Based on Cationic Block Copolymers....Pages 51-62
Chitosan....Pages 63-74
Dendrimers as DNA Carriers....Pages 75-86
Use of Synthetic Peptides for Non-viral Gene Delivery....Pages 87-102
An Oligonucleotide Carrier Based on ?-1,3-Glucans....Pages 103-117
Biological and Chemical Hybrid Vectors....Pages 118-132
Front Matter....Pages 133-133
Pharmacokinetics of Gene Delivery in Cells....Pages 135-154
Active DNA Release from Complexes....Pages 155-164
Controlled Release of DNA Using Thermoresponsive Polymers....Pages 165-175
Active Transport of Exogenous Genes into the Nucleus....Pages 176-186
Controlled Intracellular Localization of Oligonucleotides by Chemical Conjugation....Pages 187-197
Functional Nucleotide Sequences Capable of Promoting Non-viral Genetic Transfer....Pages 198-210
Shielding of Cationic Charge of the DNA Complex to Avoid Nonspecific Interactions for In Vivo Gene Delivery....Pages 211-225
In Vivo Gene Transfer by Ligand-Modified Gene Carriers....Pages 226-236
Optimizing Polyplexes into Synthetic Viruses for Tumor-Targeted Gene Therapy....Pages 237-245
Gene Transfer and Target Diseases....Pages 246-260
Front Matter....Pages 133-133
Clinical Trials Using Non-viral Gene Delivery Systems....Pages 261-290
Front Matter....Pages 291-291
Evaluation of Size and Zeta Potential of DNA/Carrier Complexes....Pages 293-299
Observation of DNA/Carrier Complexes Under Fluorescence Microscopy....Pages 300-306
Synthesis of Chemically Modified Chitosan and a Study of Its Gene Transfection Efficiency....Pages 307-314
Newly Designed DNA Fragments for Gene Correction....Pages 315-322
Evaluation of Gene Expression In Vivo After Intravenous and Intraportal Administration of Lipoplexes....Pages 323-330
Evaluation by Southern Blot Hybridization of DNA Administered with a Gene Carrier to Organs....Pages 331-338
Evaluation of the Immune Response After Administration of Plasmid DNA-Non-viral Vector Complexes....Pages 339-347
Gateway RNAi....Pages 348-360
Front Matter....Pages 361-361
Design of Intracellularly Active Ribozymes and siRNAs....Pages 363-397
RNAi-Based Inhibition Specific for Mutant Alleles in Autosomal Dominant Diseases: Sequence-Dependent and -Independent Discrimination of Mutant and Wild-Type Alleles by siRNA....Pages 398-404
In Vivo RNA Interference: Another Tool in the Box?....Pages 405-422
Suppression of Gene Expression via Chromatin Remodeling and the siRNA-Induced Silencing of Transcription....Pages 423-440
Intracellular Delivery of Nucleic Acids: Differences Between Transfection and siFection Reflect Differences Between DNA and RNA, and Between Oligodeoxynucleotides and Oligonucleotides....Pages 441-455
In Vivo Antitumor Activity of a New Cationic Liposome siRNA Complex....Pages 456-464
Enhancing RNAi with Synthetic RNA Duplexes....Pages 465-475
Back Matter....Pages 477-487
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