"Gene Therapy: Tools and Potential Applications" ed. by Francisco Martin Molina

This book brings together a comprehensive collection of gene therapy tools and their therapeutic applications. This book allows the reader to come across with the opinions of different experts in the gene therapy field.

The first part of the book covers different gene therapy vectors focusing on their advantages and disadvantages. The second part of the book gets into gene therapy applications, from the latest successes on clinical trials to the new gene therapy targets that are still under development.

Contents
Section 1 Introduction
1 Non-Viral Delivery Systems in Gene Therapy
2 Plasmid Transgene Expression in vivo: Promoter and Tissue Variables
3 Silencing of Transgene Expression: A Gene Therapy Perspective
Section 2 Gene Therapy Tools: Synthetic
4 Cellular Uptake Mechanism of Non-Viral Gene Delivery and Means for Improving Transfection Efficiency
5 Polylipid Nanoparticle, a Novel Lipid-Based Vector for Liver Gene Transfer
6 DNA Electrotransfer: An Effective Tool for Gene Therapy
7 siRNAand Gene Formulation for Efficient Gene Therapy
Section 3 Gene Therapy Tools: Biological
8 Mesenchymal Stem Cells as Gene Delivery Vehicles
9 Cancer Gene Therapy - Key Biological Concepts in the Design of Multifunctional Non-Viral Delivery Systems
10 Gene Therapy Based on Fragment C of Tetanus Toxin in ALS: A Promising Neuroprotective Strategy for the Bench to the Bedside Approach
11 Transposons for Non-Viral Gene Transfer
12 Lentiviral Gene Therapy Vectors: Challenges and Future Directions
13 Lentiviral Vectors in Immunotherapy
14 Targeted Lentiviral Vectors: Current Applications and Future Potential
15 Vectors for Highly Efficient and Neuron-Specific Retrograde Gene Transfer for Gene Therapy of Neurological Diseases
16 Retroviral Genotoxicity
17 Efficient AAV Vector Production System: Towards Gene Therapy For Duchenne Muscular Dystrophy
Section 4 Applications: Inhereted Diseases
18 Gene Therapy for Primary Immunodeficiencies
19 Gene Therapy for Diabetic Retinopathy - Targeting the Renin-Angiotensin System
20 Gene Therapy for Retinitis Pigmentosa
21 Gene Therapy for Erythroid Metabolic Inherited Diseases
22 Targeting the Lung: Challenges in Gene Therapy for Cystic Fibrosis
23 Gene Therapy for the COL7A1 Gene
24 Molecular Therapy for Lysosomal Storage Diseases
Section 5 Applications: Others
25 Gene Therapy Perspectives Against Diseases of the Respiratory System
26 Gene Therapy in Critical Care Medicine
27 Clinical and Translational Challenges in Gene Therapy of Cardiovascular Diseases
28 Gene Therapy for Chronic Pain Management
29 Insulin Trafficking in a Glucose Responsive Engineered Human Liver Cell Line is Regulated by the Interaction of ATP-Sensitive Potassium Channels and Voltage-Gated Calcium Channels
30 Feasibility of Gene Therapy for Tooth Regeneration by Stimulation of a Third Dentition