Gene therapy

Potential Applications of Nanotechnology

Gebonden Engels 2013 9781907568404
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Samenvatting

Gene therapy is emerging as a new class of therapeutics for the treatment of inherited and acquired diseases. However, poor cellular uptake and instability of DNA in the physiological milieu limits its therapeutic potential, hence a vector which can protect and efficiently transport DNA to the target cells must be developed. Nanotechnology-based non-viral vectors have been proposed as potential candidates. Various polymeric nanoparticles have been shown to be suitable, with high cellular uptake efficiencies and reduced cytotoxicity. These delivery vectors form condensed complexes with DNA which result in shielding against enzymatic degradation and enhanced cellular targeting. Advantages including easy manipulatibility, high stability, low cost and high payload, mean that nanoparticles from various polymers have been exploited. Gene therapy gives a systematic account of the many aspects of nanotechnology mediated gene therapy, from the preparation of nanoparticles to physicochemical characterization, and follows with applications in in vitro and in vivo models. This book emphasizes the various aspects of nanotechnology-based gene therapy, with initial chapters detailing the tools and techniques available for preparation and in vitro and in vivo characterization of nanoparticles. Later chapters provide exhaustive details on polymeric systems employed for gene therapy.

Specificaties

ISBN13:9781907568404
Taal:Engels
Bindwijze:Gebonden

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Inhoudsopgave

<p>Dedication</p> <p>List of figures and tables</p> <p>Acknowledgments</p> <p>Foreword</p> <p>Preface</p> <p>About the author</p> <p>Chapter 1: Nanotechnology: an introduction</p> <p>Abstract:</p> <p>1.1 Introduction</p> <p>1.2 Definition of nanotechnology</p> <p>1.3 Structure of the book</p> <p>Chapter 2: Methods of nanoparticle preparation</p> <p>Abstract:</p> <p>2.1 Introduction</p> <p>2.2 Preparation of nanoparticles by polymerization of monomers</p> <p>2.3 Preparation of nanoparticles using preformed polymers</p> <p>2.4 Methods of controlled release</p> <p>Chapter 3: Tools and techniques for physico-chemical characterization of nanoparticles</p> <p>Abstract:</p> <p>3.1 Introduction</p> <p>3.2 Physico-chemical characterization</p> <p>Chapter 4: Characterization of nanoparticles: in vitro and in vivo</p> <p>Abstract:</p> <p>4.1 Introduction</p> <p>4.2 In vitro characterization of nanoparticles</p> <p>4.3 In vivo characterization</p> <p>4.4 Conclusions</p> <p>Chapter 5: Theory and limitations to gene therapy</p> <p>Abstract:</p> <p>5.1 Introduction</p> <p>5.2 Mechanism of gene delivery</p> <p>5.3 Barriers to gene delivery</p> <p>5.4 Conclusions</p> <p>Chapter 6: Targeted gene delivery mediated by nanoparticles</p> <p>Abstract:</p> <p>6.1 Introduction</p> <p>6.2 Approaches for targeted gene delivery</p> <p>6.3 Conclusions</p> <p>Chapter 7: Polymeric nanoparticles for gene delivery</p> <p>Abstract:</p> <p>7.1 Introduction</p> <p>7.2 Advantages of nanoparticles</p> <p>7.3 Limitations of nanoparticles</p> <p>7.4 Conclusions</p> <p>Chapter 8: Poly-L-lysine nanoparticles</p> <p>Abstract:</p> <p>8.1 Introduction</p> <p>8.2 In vitro and in vivo applications of poly-L-lysine/DNA nanoparticles</p> <p>8.3 Polylysine-containing peptides for gene delivery</p> <p>8.4 Conclusions</p> <p>Chapter 9: Chitosan nanoparticles</p> <p>Abstract:</p> <p>9.1 Introduction</p> <p>9.2 Factors affecting transfection efficiency of chitosan nanoparticles</p> <p>9.3 Conclusions</p> <p>Chapter 10: Polyethylenimine nanoparticles</p> <p>Abstract:</p> <p>10.1 Introduction</p> <p>10.2 Derivatives of PEI for in vitro and in vivo gene delivery</p> <p>10.3 Degradable PEI for gene delivery</p> <p>10.4 Conclusions</p> <p>Chapter 11: Atelocollagen</p> <p>Abstract:</p> <p>11.1 Introduction</p> <p>11.2 Atelocollagen-mediated gene delivery</p> <p>11.3 Conclusions</p> <p>Chapter 12: Protamine nanoparticles</p> <p>Abstract:</p> <p>12.1 Introduction</p> <p>12.2 Protamine nanoparticles for gene delivery</p> <p>12.3 Liposome/protamine/ DNA complexes</p> <p>12.4 Protamine conjugation to other ligands</p> <p>12.5 Conclusions</p> <p>Chapter 13: Dendrimers</p> <p>Abstract:</p> <p>13.1 Introduction</p> <p>13.2 Dendrimers in gene delivery</p> <p>13.3 Conclusions</p> <p>Chapter 14: Cyclodextrins and cyclodextrin-containing polymers</p> <p>Abstract:</p> <p>14.1 Introduction</p> <p>14.2 Cyclodextrin-embedded polymers</p> <p>14.3 Polymers with cyclodextrins as pendant groups</p> <p>14.4 Cyclodextrins as adjuvants for enhanced gene delivery</p> <p>14.5 Cyclodextrin-based polyrotaxanes</p> <p>14.6 Conclusions</p> <p>Chapter 15: Poly(D,L-lactide-co-glycolide)-based nanoparticles</p> <p>Abstract:</p> <p>15.1 Introduction</p> <p>15.2 PLGA nanoparticles for gene delivery</p> <p>15.3 Chitosan-modified PLGA nanoparticles</p> <p>15.4 Polyethylenimine-modified PLGA nanoparticles</p> <p>15.5 Other modifications to PLGA nanoparticles</p> <p>15.6 Conclusions</p> <p>Chapter 16: Metallic and inorganic nanoparticles</p> <p>Abstract:</p> <p>16.1 Introduction</p> <p>16.2 Gold nanoparticles</p> <p>16.3 Mesoporous silica nanoparticles</p> <p>16.4 MSN for gene delivery</p> <p>16.5 Polycation-modified MSN for gene delivery</p> <p>16.6 Conclusions</p> <p>Index</p>

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        Gene therapy