Pharmaceutical Perspectives of Nucleic Acid-Based Therapy

by Mahato; Ram I.

• ISBN13: 9780415283854
• ISBN10: 041528385X
• Edition: 1st
• Format: Hardcover
• Copyright: 2002-06-27
• Publisher: CRC Press

Summary

Pharmaceutical Perspectives of Nucleic Acid-Based Therapy presents a comprehensive account of gene therapy, from development in the laboratory to clinical applications, with an emphasis on cancer and cardiovascular and autoimmune diseases. Internationally acclaimed scientists discuss the potential use of lipids, peptides and polymers for the in vivo delivery of nucleic acids, the genesis of structure-synthesis-function interrelationships and the evolutionary approaches of these gene carriers. The book also addresses the influence of a viable gene expression system on disease by controlling gene regulation, transcription, translation and replication.

Table of Contents

List of color plates	p. x
List of figures	p. xii
List of tables	p. xx
List of contributors	p. xxii
Foreword	p. xxvii
Preface	p. xxviii
Basic components of gene expression plasmids	p. 1
Inducible gene expression strategies in gene therapy	p. 15
Modulation of gene expression by antisense oligonucleotides	p. 33
Ribozymes as therapeutic agents and genetic tools	p. 54
Peptide nucleic acids (PNA) binding-mediated target gene transcription	p. 73
Aptamers for controlling gene expression	p. 89
Enzymes acting on DNA breaks and its relevance in nucleic acid-based therapy	p. 121
Compaction and condensation of DNA	p. 136
Structure, dispersion stability and dynamics of DNA and polycation complexes	p. 164
Structure and structure-activity correlations of cationic lipid/DNA complexes: supramolecular assembly and gene delivery	p. 190
Cellular uptake, metabolic stability and nuclear translocation of nucleic acids	p. 210
Nucleocytoplasmic trafficking	p. 229
Optimizing RNA export for transgene expression	p. 261
Naked plasmid DNA delivery of a therapeutic protein	p. 285
Cationic lipid-based gene delivery	p. 302
Peptide-based gene delivery systems	p. 334
Cationic and non-condensing polymer-based gene delivery	p. 367
Device-mediated gene delivery	p. 388
Basic pharmacokinetics of oligonucleotides and genes	p. 409
Interstitial transport of macromolecules: implication for nucleic acid delivery in solid tumors	p. 434
Hepatic delivery of nucleic acids using hydrodynamics-based procedure	p. 455
Role of CpG motifs in immunostimulation and gene expression	p. 469
Local cardiovascular gene therapy	p. 487
Gene therapy for cardiovascular disease	p. 499
Gene therapy for the prevention of autoimmune diabetes	p. 508
Index	p. 520
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