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Gene therapy. Woodhead Publishing Series in Biomedicine

  • ID: 2719651
  • Book
  • 380 Pages
  • Elsevier Science and Technology
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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

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List of figures and tables




About the author

Chapter 1: Nanotechnology: an introduction


1.1 Introduction

1.2 Definition of nanotechnology

1.3 Structure of the book

Chapter 2: Methods of nanoparticle preparation


2.1 Introduction

2.2 Preparation of nanoparticles by polymerization of monomers

2.3 Preparation of nanoparticles using preformed polymers

2.4 Methods of controlled release

Chapter 3: Tools and techniques for physico-chemical characterization of nanoparticles


3.1 Introduction

3.2 Physico-chemical characterization

Chapter 4: Characterization of nanoparticles: in vitro and in vivo


4.1 Introduction

4.2 In vitro characterization of nanoparticles

4.3 In vivo characterization

4.4 Conclusions

Chapter 5: Theory and limitations to gene therapy


5.1 Introduction

5.2 Mechanism of gene delivery

5.3 Barriers to gene delivery

5.4 Conclusions

Chapter 6: Targeted gene delivery mediated by nanoparticles


6.1 Introduction

6.2 Approaches for targeted gene delivery

6.3 Conclusions

Chapter 7: Polymeric nanoparticles for gene delivery


7.1 Introduction

7.2 Advantages of nanoparticles

7.3 Limitations of nanoparticles

7.4 Conclusions

Chapter 8: Poly-L-lysine nanoparticles


8.1 Introduction

8.2 In vitro and in vivo applications of poly-L-lysine/DNA nanoparticles

8.3 Polylysine-containing peptides for gene delivery

8.4 Conclusions

Chapter 9: Chitosan nanoparticles


9.1 Introduction

9.2 Factors affecting transfection efficiency of chitosan nanoparticles

9.3 Conclusions

Chapter 10: Polyethylenimine nanoparticles


10.1 Introduction

10.2 Derivatives of PEI for in vitro and in vivo gene delivery

10.3 Degradable PEI for gene delivery

10.4 Conclusions

Chapter 11: Atelocollagen


11.1 Introduction

11.2 Atelocollagen-mediated gene delivery

11.3 Conclusions

Chapter 12: Protamine nanoparticles


12.1 Introduction

12.2 Protamine nanoparticles for gene delivery

12.3 Liposome/protamine/ DNA complexes

12.4 Protamine conjugation to other ligands

12.5 Conclusions

Chapter 13: Dendrimers


13.1 Introduction

13.2 Dendrimers in gene delivery

13.3 Conclusions

Chapter 14: Cyclodextrins and cyclodextrin-containing polymers


14.1 Introduction

14.2 Cyclodextrin-embedded polymers

14.3 Polymers with cyclodextrins as pendant groups

14.4 Cyclodextrins as adjuvants for enhanced gene delivery

14.5 Cyclodextrin-based polyrotaxanes

14.6 Conclusions

Chapter 15: Poly(D,L-lactide-co-glycolide)-based nanoparticles


15.1 Introduction

15.2 PLGA nanoparticles for gene delivery

15.3 Chitosan-modified PLGA nanoparticles

15.4 Polyethylenimine-modified PLGA nanoparticles

15.5 Other modifications to PLGA nanoparticles

15.6 Conclusions

Chapter 16: Metallic and inorganic nanoparticles


16.1 Introduction

16.2 Gold nanoparticles

16.3 Mesoporous silica nanoparticles

16.4 MSN for gene delivery

16.5 Polycation-modified MSN for gene delivery

16.6 Conclusions


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Nimesh, Surendra
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