Non-viral Gene Therapy: Gene Design and Delivery

Contents......Page 10
Foreword......Page 5
Preface......Page 7
1. Molecules for Gene Therapy......Page 13
Recent Progress in Non-viral Gene Delivery......Page 14
Physical Chemistry of DNA-Carrier Complexes......Page 22
Liposomes......Page 30
Polymeric Gene Carriers......Page 46
Development of a Supramolecular Nanocarrier for Gene Delivery Based on Cationic Block Copolymers......Page 62
Chitosan......Page 74
Dendrimers as DNA Carriers......Page 86
Use of Synthetic Peptides for Non-viral Gene Delivery......Page 98
An Oligonucleotide Carrier Based on β-1,3-Glucans......Page 114
Biological and Chemical Hybrid Vectors......Page 129
2. Controlled Gene Delivery......Page 144
Pharmacokinetics of Gene Delivery in Cells......Page 145
Active DNA Release from Complexes......Page 165
Controlled Release of DNA Using Thermoresponsive Polymers......Page 175
Active Transport of Exogenous Genes into the Nucleus......Page 186
Controlled Intracellular Localization of Oligonucleotides by Chemical Conjugation......Page 197
Functional Nucleotide Sequences Capable of Promoting Non-viral Genetic Transfer......Page 208
Shielding of Cationic Charge of the DNA Complex to Avoid Nonspecific Interactions for In Vivo Gene Delivery......Page 221
In Vivo Gene Transfer by Ligand-Modified Gene Carriers......Page 236
Optimizing Polyplexes into Synthetic Viruses for Tumor-Targeted Gene Therapy......Page 247
Gene Transfer and Target Diseases......Page 256
Clinical Trials Using Non-viral Gene Delivery Systems......Page 271
3. Current Protocols of Gene Delivery......Page 301
Evaluation of Size and Zeta Potential of DNA/Carrier Complexes......Page 302
Observation of DNA/Carrier Complexes Under Fluorescence Microscopy......Page 309
Synthesis of Chemically Modified Chitosan and a Study of Its Gene Transfection Efficiency......Page 316
Newly Designed DNA Fragments for Gene Correction......Page 324
Evaluation of Gene Expression In Vivo After Intravenous and Intraportal Administration of Lipoplexes......Page 332
Evaluation by Southern Blot Hybridization of DNA Administered with a Gene Carrier to Organs......Page 340
Evaluation of the Immune Response After Administration of Plasmid DNA-Non-viral Vector Complexes......Page 348
Gateway RNAi......Page 357
4. Design of Genes Based on Current RNA Technology......Page 370
Design of Intracellularly Active Ribozymes and siRNAs......Page 371
RNAi-Based Inhibition Specific for Mutant Alleles in Autosomal Dominant Diseases: Sequence-Dependent and -Independent Discrimination of Mutant and Wild-Type Alleles by siRNA......Page 406
In Vivo RNA Interference: Another Tool in the Box?......Page 413
Suppression of Gene Expression via Chromatin Remodeling and the siRNA-Induced Silencing of Transcription......Page 431
Intracellular Delivery of Nucleic Acids: Differences Between Transfection and siFection Reflect Differences Between DNA and RNA, and Between Oligodeoxynucleotides and Oligonucleotides......Page 449
In Vivo Antitumor Activity of a New Cationic Liposome siRNA Complex......Page 464
Enhancing RNAi with Synthetic RNA Duplexes......Page 473
B......Page 484
C......Page 485
E......Page 486
H......Page 487
K......Page 488
M......Page 489
P......Page 490
S......Page 492
T......Page 493
Z......Page 494