Non-viral vectors for gene therapy : physical methods and medical translation / edited by Leaf Huang, Dexi Liu, Ernst Wagner.

Format:

Book

Contributor:

Huang, Leaf, editor.

Liu, Dexi, editor.

Wagner, Ernst, 1960- editor.

Series:

Advances in genetics ; Volume 89.

Advances in Genetics, 0065-2660 ; Volume 89

Language:

English

Subjects (All):

Genetic vectors.

Gene therapy.

Physical Description:

1 online resource (297 pages, 9 unnumbered pages of plates) : illustrations (some color).

Edition:

First edition.

Place of Publication:

London, England : Academic Press, 2015.

Language Note:

English

Summary:

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

Contents:

Front Cover

Advances in Genetics

Advances in Genetics, Volume 89

Advances in Genetics Nonviral Vectors for Gene Therapy Physical Methods and Medical TranslationEdited byLeaf HuangDivision ...

copyright

Dedication

Contents

Contributors

Physical Methods for Gene Transfer

1. Introduction

2. Most Commonly Used Physical Methods

2.1 Needle Injection

2.2 Gene Gun or "Biolistic" Gene Transfer

2.3 Electroporation

2.4 Hydrodynamic Gene Transfer "Hydroporation"

2.5 Ultrasound-Mediated Gene Transfer "Sonoporation"

2.6 Magnetofection

2.7 Laser-Mediated Gene Transfer "Optical Transfection"

2.8 Cell Squeezing "Microfluidics Gene Transfer"

3. Future Perspectives

References

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

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

7.1 Cholesterol-Conjugated siRNA-Loaded BLs

7.2 siRNA-Loaded BLs Using Electrostatic Interaction between Cationic Lipid and siRNA

8. MicroRNA-Loaded BLs

9. Endosomal Escape Enhanced by BLs

10. Conclusions

Acknowledgments

Electroporation-Mediated Gene Delivery

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

Acknowledgments.

References

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

Sustained Expression from DNA Vectors

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

Noncoding Oligonucleotides: The Belle of the Ball in Gene Therapy

2. Noncoding Oligonucleotides-Based Therapeutics in Gene Therapy

2.1 RNA Interference (siRNA and shRNA)

2.2 Antisense Oligonucleotides

2.3 Aptamers-Chemical Antibodies

2.4 Ribozymes-Catalytic RNAs

2.5 RNA Decoys

2.6 Bacteriophage phi 29 RNA

3. Challenges for Successful Gene Therapy Using Noncoding Oligonucleotides

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Self-Amplifying mRNA Vaccines

2. History of Nucleic Acid Vaccines

3. Introduction to mRNA Vaccines.

4. Self-Amplifying mRNA

5. Delivery of Self-Amplifying mRNA Vaccines

5.1 Viral Delivery Systems

5.2 Nonviral Delivery Systems

5.2.1 Naked

5.2.2 Particle-Mediated Epidermal Delivery or "Gene Gun"

5.2.3 Electroporation (EP)

5.2.4 Lipid Nanoparticles

5.2.4.1 LNP Delivery of a Rabies Self-Amplifying mRNA

5.2.4.2 LNP Delivery of a Human Cytomegalovirus Self-Amplifying mRNA

5.2.5 Cationic Nanoemulsions

5.2.5.1 CNE Delivery of a HCV Self-Amplifying mRNA

5.2.6 Polymer PRINT™ Particles

6. Production and Purification of Self-Amplifying mRNA

6.1 Cell-Free Synthesis Using an In vitro Transcription Reaction

6.2 Purification

7. Stability of mRNA

8. Future Prospects for Nonviral Delivery of Self-Amplifying mRNA Vaccines

Gene Electrotransfer Clinical Trials

2. GET in Clinical Trials

2.1 Overview

2.2 Cancer

2.2.1 Cancer Immunotherapy

2.2.2 Cancer Vaccines

2.2.3 Antiangiogenesis

2.3 DNA Vaccines for Infectious Agents

2.3.1 Human Immunodeficiency Virus

2.3.2 Hepatitis Viruses

2.3.3 Human Papillomavirus 16/18

2.3.4 Hantaviruses

2.4 Safety and Tolerability

3. Summary and Conclusions

Index

Color Plates.

Notes:

Bibliographic Level Mode of Issuance: Monograph

Includes bibliographical references at the end of each chapters and index.

Description based on online resource; title from PDF title page (ebrary, viewed February 5, 2015).

ISBN:

0-12-802470-4

0-12-802272-8