Nonviral Vectors for Gene Therapy

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