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Nonviral Vectors for Gene Therapy - 1st Edition - ISBN: 9780128022726, 9780128024706

Nonviral Vectors for Gene Therapy, Volume 89

1st Edition

Physical Methods and Medical Translation

Serial Volume Editors: Leaf Huang Dexi Liu Ernst Wagner
Hardcover ISBN: 9780128022726
eBook ISBN: 9780128024706
Imprint: Academic Press
Published Date: 8th January 2015
Page Count: 296
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Table of Contents

Chapter One. Physical Methods for Gene Transfer

  • 1. Introduction
  • 2. Most Commonly Used Physical Methods
  • 3. Future Perspectives

Chapter Two. Nonviral Gene Delivery Systems by the Combination of Bubble Liposomes and Ultrasound

  • 1. Introduction
  • 2. Microbubbles and Ultrasound
  • 3. Bubble Liposomes
  • 4. In vitro pDNA Delivery with BLs
  • 5. In vivo pDNA Delivery with BLs
  • 6. In vitro/In vivo Small Interfering RNA Delivery with BLs
  • 7. siRNA-Loaded BLs
  • 8. MicroRNA-Loaded BLs
  • 9. Endosomal Escape Enhanced by BLs
  • 10. Conclusions

Chapter Three. Electroporation-Mediated Gene Delivery

  • 1. Introduction
  • 2. Theory of Membrane Electroporation
  • 3. Mechanism(s) of Electroporation-Mediated Gene Delivery
  • 4. Pulse Parameters
  • 5. Electrodes
  • 6. Tissues that can be Directly Injected: Liver and Skeletal Muscle
  • 7. Electroporation of Skeletal Muscle: DNA Vaccines and Use as Bioreactors
  • 8. Electroporation of Skin: Vaccination, Wound Healing, and Cancer
  • 9. Electroporation of the Cardiovascular System
  • 10. Gene Delivery into the Lung Using Electroporation
  • 11. Conclusions

Chapter Four. Hydrodynamic Delivery

  • 1. Fundamentals of Hydrodynamic Delivery
  • 2. Applications of Hydrodynamic Gene Delivery
  • 3. Hydrodynamic Delivery in Large Animals—Toward Clinic Application
  • 4. Future Prospective

Chapter Five. Sustained Expression from DNA Vectors

  • 1. Introduction
  • 2. Immune Responses
  • 3. Preventing Epigenetic Silencing
  • 4. Position Effect
  • 5. Persistent Nonintegrating Episomal DNA Vectors
  • 6. Episomal DNA Vectors Based on Replication-Deficient Viruses
  • 7. Episomal DNA Vectors Based on Chromosomal Elements
  • 8. Mammalian Artificial Chromosomes
  • 9. Episomally Maintained pDNA Vectors
  • 10. Scaffold/Matrix Attachment Regions
  • 11. Insulator Function
  • 12. Transcription Augmentation
  • 13. Mitotic Stability
  • 14. Persistent Expression Mediated by S/MAR DNA Vectors
  • 15. Alternative Elements to Modulate Transcription Levels of DNA Vectors
  • 16. Optimization of Episomal Vectors for Gene Therapy
  • 17. Promoters and Enhancers
  • 18. The CMV Promoter
  • 19. The UbC Promoter
  • 20. The AAT Promoter
  • 21. Enhancers
  • 22. Posttranscriptional Regulation
  • 23. Effect of CpG Depletion
  • 24. Studies Demonstrating Passive Episomal State of Minicircles In vivo
  • 25. Conclusions

Chapter Six. Noncoding Oligonucleotides: The Belle of the Ball in Gene Therapy

  • 1. Introduction
  • 2. Noncoding Oligonucleotides-Based Therapeutics in Gene Therapy
  • 3. Challenges for Successful Gene Therapy Using Noncoding Oligonucleotides
  • Conflict of Interest Statement

Chapter Seven. Self-Amplifying mRNA Vaccines

  • 1. Introduction
  • 2. History of Nucleic Acid Vaccines
  • 3. Introduction to mRNA Vaccines
  • 4. Self-Amplifying mRNA
  • 5. Delivery of Self-Amplifying mRNA Vaccines
  • 6. Production and Purification of Self-Amplifying mRNA
  • 7. Stability of mRNA
  • 8. Future Prospects for Nonviral Delivery of Self-Amplifying mRNA Vaccines

Chapter Eight. Gene Electrotransfer Clinical Trials

  • 1. Introduction
  • 2. GET in Clinical Trials
  • 3. Summary and Conclusions


The field of genetics is rapidly evolving, and new medical breakthroughs are occurring as a result of advances in our knowledge of genetics. Advances in Genetics continually publishes important reviews of the broadest interest to geneticists and their colleagues in affiliated disciplines.

Key Features

  • Includes methods for testing with ethical, legal, and social implications
  • Critically analyzes future directions
  • Written and edited by recognized leaders in the field


Molecular geneticists, clinical geneticists, neurologists, neuroscientists, molecular biologists, and biochemists.


No. of pages:
© Academic Press 2015
8th January 2015
Academic Press
Hardcover ISBN:
eBook ISBN:


Praise for previous volumes in the series:
"Outstanding both in variety and in the quality of its contributions." –Nature

"Can be highly recommended to geneticists, and biologists in general...will prove to be of high importance for the development of the science of genetics." --Science

Ratings and Reviews

About the Serial Volume Editors

Leaf Huang

Leaf Huang

Affiliations and Expertise

Department of Biomedical Engineering, University of North Carolina at Chapel Hill, North Carolina, U.S.A.

Dexi Liu

Dexi Liu

Affiliations and Expertise

Department of Pharmaceutical and Biomedical Sciences, University of Georgia College of Pharmacy, Georgia, U.S.A.

Ernst Wagner

Ernst Wagner

Affiliations and Expertise

Munich Center for System-based Drug Research, Center for Nanoscience, Ludwig-Maximilians-Universität, Germany