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…mehr
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.Several years ago, when the discovery of catalytic RNA was recognized in a public manner,many people asked if new ?elds of therapy would soon be available. Although some tentative positive answers were given,nobody would say with certainty that RNA of various kinds was a truly promising means of altering gene expression. In fact,over the past decade,both our knowledge of RNAs with different functions and the utility of RNA in the inhibition or enhancement of gene expression have occurred with great drama. We proceeded in terms of possible therapeutic tools from RNase P and group I introns through hammerhead RNA enzymes, antisense technology, and more recently, to RNAi and its derivatives. A useful practical method of RNA delivery in animals will complete the picture. The diversity of RNA and the varied role of it inside cells and in therapy should be a tremendous challenge for young molecular biologists. This volume will make their task easier. Sidney Altman Sterling Professor of Molecular,Cellular & Devel- mental Biology,Nobel Laureate Department of Molecular,Cellular and Developm- tal Biology Yale University V NGTPR 4/23/05 1:00 PM Page VI VI Foreword Delivery of nucleic acids to cells in an animal remains a challenging problem. It is the major obstacle to success of therapeutic approaches using genes and oli- nucleotides,including siRNAs. Solutions found so far by chemists are satisfactory only for transfection of cells in culture.
Taira Kazunari, University of Tokyo, Japan / Kataoka Kazunori, University of Tokyo, Japan / Niidome Takuro, Kyushu University, Fukuoka, Japan
1 Molecules for Gene Delivery 1-1 Leaf Huang Recent Progress in Nonviral Gene Delivery 1-2 Kenichi Yoshikawa Physical Chemistry of DNA-Carrier Complex 1-3 Kazuo Maruyama Liposome-Mediated Delivery System 1-4 Tomoko Hashimoto Polymeric Gene Carriers 1-5 Kazunori Kataoka Development of Cationic Block Copolymer-Based Gene Delivery System 1-6 Toshinori Sato Chitosan-Mediated Gene Delivery 1-7 Hidetoshi Arima Dendrimers as DNA Carriers 1-8 Takuro Niidome Use of Synthetic Peptide for Non-viral Gene Delivery 1-9 Kazuo Sakurai Oligonucleotide Carrier Based on b-1,3-Glucans 1-10 Mahito Nakanishi Biological and Chemical Hybrid Vectors 2 Controlled Gene Delivery 2-1 Hideyoshi Harashima Pharmacokinetics of Gene Delivery in Cells 2-2 Takeshi Nagasaki Active DNA Release from Complex 2-3 Masayuki Yokoyama Controlled Release of DNA using Thermo-Responsive Polymers 2-4 Takeshi Nagasaki Active Transport of Exogenous Gene into Nucleus 2-5 Masayuki Fujii Controlled Intracellular Localization of Oligonucleotides by Chemical Conjugation 2-6 Osam Mazda Functional Nucleotide Sequences Capable of Promoting Nonviral Genetic Transfer 2-7 Yoshiyuki Koyama Shielding of cationic charge of the DNA complex to avoid non-specific interactions for in vivo gene delivery 2-8 Shigeru Kawakami In Vivo Gene Transfer by Ligand Modified Gene Carriers 2-9 Ernst Wagner Optimizing Polyplexes into Systemic Viruses for Tumor-Targeted Gene Therapy 2-10 Mariko Harada-Shiba Gene Transfer and Target Diseases 2-11 Hironobu Yanagie Clinical Trials Using Non-Viral Gene Delivery Systems 3 Current Protocoles of Gene Delivery 3-1 Mitsuru Hashida Evaluation of Size and Zeta-Potential of DNA/Carrier Complex 3-2 Tomoko Ito Observation of DNA / Carrier Complex under Fluorescence Microscopy 3-3 Taku Satoh Synthesis of Chemically Modified Chitosan and its Gene Transfection Efficiency 3-4 Hiroyuki Kamiya Newly Designed DNA Fragments for Gene Correcton 3-5 Tatsuya Okuda Evaluation of Gene Expression in Vivo after Intraveous and Intraportal Administration of Lipoplexes 3-6 Takuro Niidome Evaluation of DNA Administrated with Gene Carrier in Organs by Southern Blot Hybridization 3-7 Fuminori Sakurai Evaluation of Immune Response after Administration of Plasmid DNA-Nonviral Vector Complex 3-8 Kunt R. Madden Gateway RNAi 4 Design of Genes Based on Current RNA Technology 4-1 Kazunari Taira Design of Intracellulary Active Ribozymes and siRNA 4-2 Takanori Yokota siRNA-based Inhibition Specific for Mutant Allele in Autosomal Dominant Diseases: Sequence-Dependent and -Independent Discriminations of Mutant and Wild-type Alleles by siRNA 4-3 Silvere Petit In Vivo RNA Interference: another tool in the box? 4-4 Hiroaki Kawasaki Suppression of Gene Expression via Chromatin Remodeling and the siRNA-Induced Silencing of Transcription 4-5 Andrew Miller Intracellular delivery of nucleic acids: differences between transfection and siFection reflect differences between DNA and RNA, and between oligodeoxynucleotides and oligonucleotides 4-6 Junichi Yano In Vivo Antitumour Activity of a New Cationic Liposome siRNA Complex 4-7 John Rossi Enhancing RNAi with Synthetic RNA Duplexes
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