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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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.

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

Readership

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

Table of Contents

  • Dedication

    List of figures and tables

    Acknowledgments

    Foreword

    Preface

    About the author

    Chapter 1: Nanotechnology: an introduction

    Abstract:

    1.1 Introduction

    1.2 Definition of nanotechnology

    1.3 Structure of the book

    Chapter 2: Methods of nanoparticle preparation

    Abstract:

    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

    Abstract:

    3.1 Introduction

    3.2 Physico-chemical characterization

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

    Abstract:

    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

    Abstract:

    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

    Abstract:

    6.1 Introduction

    6.2 Approaches for targeted gene delivery

    6.3 Conclusions

    Chapter 7: Polymeric nanoparticles for gene delivery

    Abstract:

    7.1 Introduction

    7.2 Advantages of nanoparticles

    7.3 Limitations of nanoparticles

    7.4 Conclusions

    Chapter 8: Poly-L-lysine nanoparticles

    Abstract:

    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

    Abstract:

    9.1 Introduction

    9.2 Factors affecting transfection efficiency of chitosan nanoparticles

    9.3 Conclusions

    Chapter 10: Polyethylenimine nanoparticles

    Abstract:

    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

    Abstract:

    11.1 Introduction

    11.2 Atelocollagen-mediated gene delivery

    11.3 Conclusions

    Chapter 12: Protamine nanoparticles

    Abstract:

    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

    Abstract:

    13.1 Introduction

    13.2 Dendrimers in gene delivery

    13.3 Conclusions

    Chapter 14: Cyclodextrins and cyclodextrin-containing polymers

    Abstract:

    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

    Abstract:

    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

    Abstract:

    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

    Index

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

Dr. Surendra Nimesh is the author/co-author of two Elsevier books: The Design and Development of Novel Drugs and Vaccines (2021) and Gene Therapy: Potential Applications of Nanotechnology (2013) an internationally recognized expert of nanotechnology for biological applications with specialization in drug and gene delivery. He received his M.S. in Biomedical Science from Dr. B.R. Ambedkar Center for Biomedical Science Research (ACBR), University of Delhi, Delhi, India in 2001. He completed his PhD. in Nanotechnology at ACBR and Institute of Genomics and Integrative Biology (CSIR), Delhi, India in 2007. After completing his postdoctoral studies at Ecole Polyetchnique of Montreal, Montreal in 2009, he joined Clinical Research Institute of Montreal, Montreal, Canada as Postdoctoral Fellow. He worked for a short duration at McGill University, Montreal and thereafter joined Health Canada, Canada as NSERC visiting fellow in 2012. He joined Central University of Rajasthan, India as UGC-Assistant Professor at Department of Bitechnology, School of life Sciences in 2013. He has authored more than 20 research papers, 10 review articles in international peer reviewed journal, 8 book chapters and 3 books. His research interests include nanoparticles mediated gene, siRNA and drug delivery for therapeutics.

Affiliations and Expertise

UGC Assistant Professor, Department of Biotechnology, School of Life Sciences, Central University of Rajasthan, India

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