Advanced Gene Delivery

Front Cover
Alain Rolland
CRC Press, Sep 2, 2003 - Science - 328 pages
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A practical resource for everyone involved in the gene therapy field and in the design of effective gene delivery systems, this volume presents an overview and update of recent advances in the field of non-viral methods for the in vivo transfer of therapeutic genes to biological targets using conventional routes of administration. Methods to control the spatial and temporal modulation of gene function in vivo as well as the level, duration, specificity, and fidelity of gene expression are described.
The rational design and the applications of a variety of non-viral gene delivery systems, such as cationic lipid-, polymer-, and (poly) peptide-based systems, are exemplified for the control of location of therapeutic genes administered by various routes. Current and potential clinical applications of gene-based medicines are presented for the prevention, correction or modulation of diseases. Examples of current applications of plasmid-based systems for genetic vaccination, treatment of genetic disorders such as cystic fibrosis, and treatment of acquired diseases such as cancer are also provided.
  

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Contents

1 PRESENT AND FUTURE STATUS OF GENE THERAPY
1
2 EXPRESSION PLASMIDS FOR NONVIRAL GENE THERAPY
14
3 DNA PACKAGING IN NONVIRAL SYSTEMS
41
4 BIOLOGICAL BARRIERS TO GENE TRANSFER
59
5 CATIONIC LIPIDBASED GENE DELIVERY SYSTEMS
94
6 THERAPEUTIC APPLICATIONS OF LIPIDBASED GENE DELIVERY SYSTEMS
111
7 POLYMERIC GENE DELIVERY SYSTEMS FOR IN VIVO GENE THERAPY
130
8 POLYCATIONBASED DELIVERY SYSTEMS FOR RECEPTORMEDIATED GENE DELIVERY
160
9 DNA DELIVERY SYSTEMS BASED ON SYNTHETIC PEPTIDES
175
10 GENEBASED VACCINES
193
11 INTRAVASCULAR DELIVERY OF NAKED PLASMID DNA
212
VIRAL AND NONVIRAL APPROACHES
228
STRATEGIES AND REVIEW OF CLINICAL TRIALS
252
INDEX
280
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About the author (2003)

Rolland is Director of Vector Delivery at GeneMedicine, Inc., Houston, Texas.

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