Non-viral Gene Therapy : Gene Design and Delivery / edited by Kazunari Taira, Kazunori Kataoka, Takuro Niidome.

Springer eBooks

Molecules for Gene Therapy -- Recent Progress in Non-viral Gene Delivery -- Physical Chemistry of DNA-Carrier Complexes -- Liposomes -- Polymeric Gene Carriers -- Development of a Supramolecular Nanocarrier for Gene Delivery Based on Cationic Block Copolymers -- Chitosan -- Dendrimers as DNA Carriers -- Use of Synthetic Peptides for Non-viral Gene Delivery -- An Oligonucleotide Carrier Based on ?-1,3-Glucans -- Biological and Chemical Hybrid Vectors -- Controlled Gene Delivery -- Pharmacokinetics of Gene Delivery in Cells -- Active DNA Release from Complexes -- Controlled Release of DNA Using Thermoresponsive Polymers -- Active Transport of Exogenous Genes into the Nucleus -- Controlled Intracellular Localization of Oligonucleotides by Chemical Conjugation -- Functional Nucleotide Sequences Capable of Promoting Non-viral Genetic Transfer -- Shielding of Cationic Charge of the DNA Complex to Avoid Nonspecific Interactions for In Vivo Gene Delivery -- In Vivo Gene Transfer by Ligand-Modified Gene Carriers -- Optimizing Polyplexes into Synthetic Viruses for Tumor-Targeted Gene Therapy -- Gene Transfer and Target Diseases -- Clinical Trials Using Non-viral Gene Delivery Systems -- Current Protocols of Gene Delivery -- Evaluation of Size and Zeta Potential of DNA/Carrier Complexes -- Observation of DNA/Carrier Complexes Under Fluorescence Microscopy -- Synthesis of Chemically Modified Chitosan and a Study of Its Gene Transfection Efficiency -- Newly Designed DNA Fragments for Gene Correction -- Evaluation of Gene Expression In Vivo After Intravenous and Intraportal Administration of Lipoplexes -- Evaluation by Southern Blot Hybridization of DNA Administered with a Gene Carrier to Organs -- Evaluation of the Immune Response After Administration of Plasmid DNA-Non-viral Vector Complexes -- Gateway RNAi -- Design of Genes Based on Current RNA Technology -- Design of Intracellularly Active Ribozymes and siRNAs -- RNAi-Based Inhibition Specific for Mutant Alleles in Autosomal Dominant Diseases: Sequence-Dependent and -Independent Discrimination of Mutant and Wild-Type Alleles by siRNA -- In Vivo RNA Interference: Another Tool in the Box? -- Suppression of Gene Expression via Chromatin Remodeling and the siRNA-Induced Silencing of Transcription -- Intracellular Delivery of Nucleic Acids: Differences Between Transfection and siFection Reflect Differences Between DNA and RNA, and Between Oligodeoxynucleotides and Oligonucleotides -- In Vivo Antitumor Activity of a New Cationic Liposome siRNA Complex -- Enhancing RNAi with Synthetic RNA Duplexes.

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.

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