Nonviral Vectors for Gene Therapy

Lipid- and Polymer-based Gene Transfer

1st Edition - November 12, 2014

Editors: Leaf Huang, Dexi Liu, Ernst Wagner

Hardback ISBN:

9 7 8 - 0 - 1 2 - 8 0 0 1 4 8 - 6

eBook ISBN:

9 7 8 - 0 - 1 2 - 8 0 0 3 6 7 - 1

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… Read more

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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.

Advances in Genetics, Volume 88
Dedication
Chapter One. Nonviral Vectors: We Have Come a Long Way
- 1. Introduction
- 2. Chemical Methods
- 3. Physical Methods
- 4. Perspectives
Chapter Two. Lipid Nanoparticles for Gene Delivery
- 1. Introduction
- 2. Rational Designs to Overcome Extracellular and Intracellular Barriers
- 3. Current Lipidic Vectors for Gene Delivery
- 4. Gene Therapy Applications
- 5. Pharmacokinetics, Biodistribution and Toxicity of LNPs
- 6. Clinical Trials
- 7. Conclusions
Chapter Three. Nanotechnology for In vivo Targeted siRNA Delivery
- 1. RNA Potently Modifies Gene Expression
- 2. Targeting Strategies
- 3. Targeting the Liver
- 4. Targeting Primary Tumors and Metastasis
- 5. Endothelial Cell Targeting
- 6. Future Perspectives
Chapter Four. Lipid Nanoparticles for Short Interfering RNA Delivery
- 1. Introduction
- 2. Challenges and Strategies for Delivery of siRNA
- 3. Lipid-Based Delivery Systems
- 4. LNP-siRNA Formulations in Clinical Trials
- 5. Future Prospects
Chapter Five. Composite Nanoparticles for Gene Delivery
- 1. Nanomedicine and Gene Therapy
- 2. Composite Nanoparticles
- 3. Fabrication Methods of Composite Nanoparticles
- 4. Composite Nanoparticles for Targeted Gene Delivery
- 5. Conclusion
Chapter Six. Multifunctional Enveloped Nanodevices (MENDs)
- 1. Introduction
- 2. R8-MEND
- 3. KALA-MEND
- 4. MITO-Porter
- 5. YSK-MEND
- 6. SS-Cleavable Proton-Activated Lipid-like Material (ssPALM)
- 7. Perspectives
Chapter Seven. Lipid-Coated Calcium Phosphate Nanoparticles for Nonviral Gene Therapy
- 1. Recent Progress of Nonviral Gene Therapy and In vitro/In vivo Delivery Systems
- 2. LCP Nanoparticles as a Multifunctional Platform for Gene Delivery
- 3. Potential Therapeutic Applications of LCP: Cancer, Chronic Liver Disease, and Gene Therapy
- 4. LCP Nanoparticles: Conclusions
Chapter Eight. Polymers for Nucleic Acid Transfer—An Overview
- 1. Five Decades in Polyplexes: Challenges and Breakthroughs
- 2. Optimizing the Core: Biodegradable and Biocompatible Polymers
- 3. Optimizing the Shell: Bioinspired Smart Polyplexes
- 4. Next Steps: Multifunctional and Sequence-Defined Polymers
- 5. Perspectives
Chapter Nine. Recent Developments in Nucleic Acid Delivery with Polyethylenimines
- 1. Introduction
- 2. Mechanism of PEI-Mediated DNA Transfection
- 3. Proton Sponge: Myth or Reality?
- 4. PEI for Gene Delivery
- 5. PEI Derivatives for the Delivery of Exon Skipping Oligonucleotides
- 6. PEIs for siRNA Delivery
- 7. Conclusion
Chapter Ten. Bioresponsive Polymer-Based Nucleic Acid Carriers
- 1. Introduction
- 2. Polymer Self-Assemblies for Nucleic Acid Delivery
- 3. Reductive Environment-Responsive Nucleic Acid Delivery
- 4. Acidic pH-Responsive Nucleic Acid Delivery
- 5. Other Stimuli-Responsive Nucleic Acid Delivery
- 6. Conclusion and Future Perspectives
Chapter Eleven. Chitosan-Based Nanoparticles for Mucosal Delivery of RNAi Therapeutics
- 1. Introduction
- 2. RNA Interference
- 3. The Mucosal Barrier
- 4. Mucoadhesive Biomaterials
- 5. Chitosan
- 6. Chitosan/siRNA Nanoparticles
- 7. Chitosan/siRNA Nanoparticles for Delivery across Mucosal Barriers
- 8. Considerations and Future Perspectives
Chapter Twelve. Polycation-Mediated Integrated Cell Death Processes
- 1. Introduction: Cell Death Definitions, Guidelines, and Processes
- 2. Lysosomal-Related Cell Death
- 3. ER-Related Cell Death
- 4. Mitochondrion-Related Cell Death on Polycation Treatment
- 5. Cell Death-Assay Design, Considerations, and Interpretations
- 6. Safer Design of Polycationic Systems
- 7. Conclusions
Index
Color Plates