Gene therapy

Gene therapy

Potential Applications of Nanotechnology

1st Edition - October 31, 2013

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  • Author: Surendra Nimesh
  • Hardcover ISBN: 9781907568404
  • eBook ISBN: 9781908818645

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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.

Key Features

  • 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


Academics and students in the fields of gene therapy and the cure for genetic disorders

Table of Contents

  • Dedication

    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


Product details

  • No. of pages: 380
  • Language: English
  • Copyright: © Woodhead Publishing 2013
  • Published: October 31, 2013
  • Imprint: Woodhead Publishing
  • Hardcover ISBN: 9781907568404
  • eBook ISBN: 9781908818645

About the Author

Surendra Nimesh

Surendra Nimesh, UGC Assistant Professor at Central University of Rajasthan, Rajasthan, India. Surendra received his M.S. in Biomedical Science from the Dr. B.R. Ambedkar Center for Biomedical Research (ACBR), University of Delhi. He completed his PhD. In Nanotechnology at the Institute of Genomics and Integrative Biology (CSIR), Delhi. After completing his postdoctoral studies at the Ecole Polyetchnique of Montreal, Surendra joined the Clinical Research Institute of Montreal (IRCM), Canada as Postdoctoral Fellow. After completing his commitment at IRCM, he joined McGill University for short time. He also worked as NSERC visiting fellow at Health Canada, Canada. His research interests include nanoparticles-mediated gene, siRNA and drug delivery.

Affiliations and Expertise

UGC Assistant Professor, Central University of Rajasthan, Rajasthan, India

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