Description
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.- Provides an overview of nanotechnology applications in gene therapy, from preparation of nanoparticles to in vitro and in vivo studies- Details the tools and techniques available for preparation, characterization and in vitro and in vivo study of nanoparticles- Details the limitations of nanoparticle-mediated gene therapy and proposes ways in which they may be overcome
Table of Contents
DedicationList of figures and tablesAcknowledgmentsForewordPrefaceAbout the authorChapter 1: Nanotechnology: an introductionAbstract:1.1 Introduction1.2 Definition of nanotechnology1.3 Structure of the bookChapter 2: Methods of nanoparticle preparationAbstract:2.1 Introduction2.2 Preparation of nanoparticles by polymerization of monomers2.3 Preparation of nanoparticles using preformed polymers2.4 Methods of controlled releaseChapter 3: Tools and techniques for physico-chemical characterization of nanoparticlesAbstract:3.1 Introduction3.2 Physico-chemical characterizationChapter 4: Characterization of nanoparticles: in vitro and in vivoAbstract:4.1 Introduction4.2 In vitro characterization of nanoparticles4.3 In vivo characterization4.4 ConclusionsChapter 5: Theory and limitations to gene therapyAbstract:5.1 Introduction5.2 Mechanism of gene delivery5.3 Barriers to gene delivery5.4 ConclusionsChapter 6: Targeted gene delivery mediated by nanoparticlesAbstract:6.1 Introduction6.2 Approaches for targeted gene delivery6.3 ConclusionsChapter 7: Polymeric nanoparticles for gene deliveryAbstract:7.1 Introduction7.2 Advantages of nanoparticles7.3 Limitations of nanoparticles7.4 ConclusionsChapter 8: Poly-L-lysine nanoparticlesAbstract:8.1 Introduction8.2 In vitro and in vivo applications of poly-L-lysine/DNA nanoparticles8.3 Polylysine-containing peptides for gene delivery8.4 ConclusionsChapter 9: Chitosan nanoparticlesAbstract:9.1 Introduction9.2 Factors affecting transfection efficiency of chitosan nanoparticles9.3 ConclusionsChapter 10: Polyethylenimine nanoparticlesAbstract:10.1 Introduction10.2 Derivatives of PEI for in vitro and in vivo gene delivery10.3 Degradable PEI for gene delivery10.4 ConclusionsChapter 11: AtelocollagenAbstract:11.1 Introduction11.2 Atelocollagen-mediated gene delivery11.3 ConclusionsChapter 12: Protamine nanoparticlesAbstract:12.1 Introduction12.2 Protamine nanoparticles for gene delivery12.3 Liposome/protamine/ DNA complexes12.4 Protamine conjugation to other ligands12.5 ConclusionsChapter 13: DendrimersAbstract:13.1 Introduction13.2 Dendrimers in gene delivery13.3 ConclusionsChapter 14: Cyclodextrins and cyclodextrin-containing polymersAbstract:14.1 Introduction14.2 Cyclodextrin-embedded polymers14.3 Polymers with cyclodextrins as pendant groups14.4 Cyclodextrins as adjuvants for enhanced gene delivery14.5 Cyclodextrin-based polyrotaxanes14.6 ConclusionsChapter 15: Poly(D,L-lactide-co-glycolide)-based nanoparticlesAbstract:15.1 Introduction15.2 PLGA nanoparticles for gene delivery15.3 Chitosan-modified PLGA nanoparticles15.4 Polyethylenimine-modified PLGA nanoparticles15.5 Other modifications to PLGA nanoparticles15.6 ConclusionsChapter 16: Metallic and inorganic nanoparticlesAbstract:16.1 Introduction16.2 Gold nanoparticles16.3 Mesoporous silica nanoparticles16.4 MSN for gene delivery16.5 Polycation-modified MSN for gene delivery16.6 ConclusionsIndex
