Advanced Gene Delivery
CRC Press, Sep 2, 2003 - Science - 328 pages
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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1 PRESENT AND FUTURE STATUS OF GENE THERAPY
2 EXPRESSION PLASMIDS FOR NONVIRAL GENE THERAPY
3 DNA PACKAGING IN NONVIRAL SYSTEMS
4 BIOLOGICAL BARRIERS TO GENE TRANSFER
5 CATIONIC LIPIDBASED GENE DELIVERY SYSTEMS
6 THERAPEUTIC APPLICATIONS OF LIPIDBASED GENE DELIVERY SYSTEMS
7 POLYMERIC GENE DELIVERY SYSTEMS FOR IN VIVO GENE THERAPY
8 POLYCATIONBASED DELIVERY SYSTEMS FOR RECEPTORMEDIATED GENE DELIVERY
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Acad activity adenovirus administration airway antibodies antigen barriers binding Biol biological cancer cationic lipids cationic liposomes cellular CFTR charge ratio Chem chitosan clinical trials cystic fibrosis cytokine cytoplasm dendrimers disease DNA complexes DNA condensation DNA delivery DNA vaccines dose Drug effect encoding endocytosis endosome enhance expression plasmids Felgner formulations gene delivery gene delivery systems gene expression Gene Ther gene therapy gene transfer genetic genosomes Huang human immune response injection interactions intravenous intron Lasic levels lipid-based lipoplexes liposomes liver luciferase lung mechanism mediated membrane mice molecular molecules mouse mRNA muscle nasal epithelium Natl non-viral nuclear Nucleic Acids particles patients pDNA peptides plasmid plasmid DNA polyelectrolyte polymers Proc promoter protein receptor receptor-mediated recombinant sequences serum specific splice structure studies synthetic Szoka target therapeutic tissue toxicity transcription transfection transfection efficiency transgene transgene expression tumor cells uptake viral vectors virus vitro transfection Zabner