دانلود کتاب Gene Delivery: Nanotechnology and Therapeutic Applications

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Cover Half Title Series Page Title Page Copyright Page Dedication Table of Contents Preface Editor List of Contributors Chapter 1: Challenges in Delivering Gene Therapy 1.1 Introduction 1.2 Experimental Concepts of Gene Therapy 1.3 Aims of Gene Therapy 1.4 Delivery Systems of Gene Therapy 1.5 Vectors of Gene Therapy 1.6 Retroviral Vectors 1.7 Lentiviral Vectors 1.8 Adenoviral Vectors 1.9 Nonviral Vectors 1.10 Challenges in Delivery Systems 1.11 Challenges in Gene Delivery 1.12 Immune Response Challenges in Gene Delivery 1.13 Future Perspectives 1.14 Conclusion References Chapter 2: Advances in Genome Editing: The Technology of Choice for Precise and Efficient Disease Treatment, with Special Focus on Nano Delivery Systems 2.1 Introduction 2.2 Three Major Genome Editing Methods 2.2.1 Zinc-Finger Nucleases (ZFNs) 2.2.2 Transcription Activator-Like Effector Nucleases (TALEN) 2.3 Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR/CAS) 2.3.1 Applications of Genome Editing in Therapy 2.3.2 Nano Delivery Approaches in Genome Editing for Disease Treatment 2.3.2.1 Lipid Nanoparticles 2.3.2.2 Polymeric Nanoparticles 2.3.2.3 Extra-Cellular Vesicles 2.3.2.4 Miscellaneous Nanoparticles 2.4 Future Perspectives Abbreviations References Chapter 3: Extracellular Vesicles for Nucleic Acid Delivery: Progress and Prospects for Safe RNA-Based Gene Therapy 3.1 Introduction 3.2 Extracellular Vesicles (EVs) 3.2.1 Origin and Biogenesis 3.2.2 RNA-Sorting Mechanisms 3.2.3 Composition of Extracellular Vesicles (EVs) 3.2.3.1 Proteins and Lipids 3.2.3.2 RNAs 3.2.4 Inherent Capacity of Extracellular Vesicles in Crossing Physical Barriers 3.3 Role of EVs in Nucleic Acid Transfer and Communication 3.3.1 EVs as Nucleic Acid Delivery Tool 3.3.2 Loading After EV Isolation 3.3.2.1 Simple Incubation 3.3.2.2 Electroporation 3.3.2.3 Sonication 3.3.3 Loading before EV isolation 3.4 Functionalized EVs for Targeted Delivery 3.5 Therapeutic Applications of EVs in Nucleic Acid Delivery 3.6 Conclusion References Chapter 4: Green Synthesis of Nanoparticles in Oligonucleotide Drug Delivery System 4.1 Introduction 4.2 Types of Oligonucleotides 4.2.1 Antisense oligonucleotides (ASO) 4.2.1.1 RNAse H-dependent oligonucleotide 4.2.2 Steric-blocker oligonucleotides 4.2.3 Small Interfering RNA or Silencing RNA (siRNA) 4.2.4 Micro RNA (miRNA) 4.2.5 Aptamer 4.2.6 CpG Oligonucleotides 4.3 Therapeutic Importance of Oligonucleotides 4.4 Drug Delivery Approaches for Oligonucleotides 4.5 Green Synthesis of Nanoparticles 4.6 Approaches Involved in the Green Synthesis of Nanoparticles 4.6.1 Green Synthesis of Nanoparticles 4.6.2 Bacteria-Mediated Nanoparticle Generation 4.6.3 Silver Nanoparticles 4.6.4 Gold Nanoparticle 4.6.5 Magnetite Nanoparticles 4.6.6 Palladium and Platinum Nanoparticles 4.6.7 Selenium and Tellurium Nanoparticles 4.6.8 Fungi-Mediated Nanoparticle Generation 4.6.9 Actinomycetes-Mediated Nanoparticle Generation References Chapter 5: Development of m-RNA Vaccines in Covid-19 Pandemic Scenario 5.1 Historical Landmarks Outlining the Development of mRNA Vaccines 5.2 Challenges Towards Rationale Design of mRNA Vaccines 5.3 Clinical Trials Evaluating the Safety and Immunogenicity of mRNA Vaccines 5.4 mRNA Vaccines in Adolescents and Older Adults 5.5 mRNA Technology: A Promising Alternative for Future Implications and Supporting Data Review 5.6 Safety and Efficacy of mRNA-1273 and BNT162b2 Vaccines (Phase III Clinical Trial) 5.7 Conclusion References Chapter 6: Gene Therapy for Cardiovascular Diseases: Clinical Evidences 6.1 Introduction 6.2 Targets for Cardiovascular Gene Therapy 6.2.1 Ischemic Heart Diseases 6.2.2 Atherogenesis and Thrombosis 6.2.3 Restenosis, In-Stent Restenosis, Graft Failure 6.2.4 Systemic Hypertension 6.2.5 Pulmonary Hypertension 6.2.6 Heart Failure 6.2.6.1 Ca 2+ Protein Cycling as a Target 6.2.6.2 Targeting of Beta-Adrenergic System 6.3 Clinical Studies on Gene Therapy for CVDs 6.4 Conclusion and Future Perspective References Chapter 7: Current Application of CRISPR/Cas9 Gene-Editing Technique to Eradication of HIV/AIDS 7.1 Introduction 7.2 Overview of CRISPR/Cas9 Technology 7.3 Application of CRISPR/Cas9 System to HIV/AIDS Prevention and Treatment 7.3.1 Inactivation and Elimination of HIV-1 Provirus by CRISPR/Cas9 Technology 7.3.2 Disruption of Co-receptors CCR5 and CXCR4 by CRISPR/Cas9 Technology 7.3.3 Reactivation of Latent HIV-1 Virus by CRISPR/Cas9 Technology 7.3.4 Reactivation of Host Restriction Factors During HIV-1 Infection 7.3.5 CRISPR/CAS9 System Delivery Approach 7.4 Conclusion References Chapter 8: siRNA Delivery for Therapeutic Applications Using Nanoparticles 8.1 Introduction 8.2 Mechanism of Gene Silencing 8.3 Nanoparticles in siRNA Delivery 8.4 siRNA Conjugation with Peptides or Polymers (Less Than 10 nm in Size) 8.5 Polyethylene Amine and Cationic Based Peptides and Proteins (100 to 300 nm in Size) 8.6 Cationic Based Lipid Nanoparticles (100 to 300 nm in Size) 8.7 Neutral Liposomes (<200 nm in Size) 8.8 Liposomes and Lipoplexes 8.9 Other Nanoparticles 8.10 Polyplexes 8.11 Nanomicelles 8.12 Carbon-Based Nanomaterials 8.13 Dendrimers 8.14 Metal Based Nanoparticles 8.15 Mesoporous Silica and Silicon-Based Nanoparticles 8.16 Hybrid Nanoparticles 8.17 Gold Nanoparticles 8.18 Iron Oxide Nanoparticles References Chapter 9: Clinical Perspectives on Gene Therapy for Retinal and Eye Diseases 9.1 Introduction 9.1.1 Age Related Macular Degeneration (AMD or ARMD) 9.1.2 Retinitis Pigmentosa (RP) 9.1.3 Leber’s congenital amaurosis (LCA) 9.2 Categories of Gene Therapy 9.3 Eye as a Lucrative Target for Gene Therapy 9.3.1 Ocular Gene Delivery Vectors 9.3.2 Ocular Routes of Administration 9.3.3 Outcome Measures in Clinical Trials for Study of Effectivity of Gene Therapy in Eye Diseases 9.4 Current Therapies for Retinal Diseases Under Investigation 9.4.1 Leber’s Congenital Amaurosis (LCA) 9.4.2 Achromatopsia 9.4.3 Retinitis Pigmentosa (RP) 9.4.4 Leber’s Hereditary Optic Neuropathy (LHON) 9.4.5 AMD 9.4.6 Retinoblastoma 9.4.7 Other Retinal Diseases 9.5 Gene Therapy in Other Eye Diseases 9.6 Future Directions 9.7 Conclusion Acknowledgement References Chapter 10: Herpesvirus microRNAs for Use in Gene Therapy Immune-Evasion Strategies 10.1 Introduction 10.1.1 What is Gene Therapy? 10.1.1.1 Importance of Gene Therapy in Transplantation Biology 10.1.1.2 Transplantation and Graft Rejection 10.2 What are miRNAs 10.3 What are Herpes Viruses 10.4 Epstein Barr Virus and associated microRNAs 10.5 Human Cytomegalovirus and Associated microRNAs 10.6 Kaposi’s Sarcoma Associated Herpesvirus and Associated microRNAs 10.7 Herpes Simplex Virus and Associated microRNAs References Chapter 11: Gene Therapy and Small Molecules Used in the Treatment of Cystic Fibrosis 11.1 Introduction 11.2 History of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene 11.3 Pathophysiology of CFTR Gene 11.4 Ionic content and fluid on epithelial surfaces 11.5 Role of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) 11.6 Cystic fibrosis related lung disease 11.7 Signs and Symptoms of Cystic Fibrosis 11.7.1 Respiratory Symptoms 11.7.2 Gastro-enterologic Symptoms 11.7.3 Miscellaneous Symptoms 11.7.4 Symptoms that may Indicate the Presence of CFRD (Cystic Fibrosis Related Diabetes) 11.8 Diagnosis 11.8.1 Sweat Testing 11.8.1.1 Interpretation of Sweat Test 11.8.2 Genotyping 11.8.3 Semen Analysis 11.8.4 Sinus Radiographs 11.8.5 Tests of Exocrine Pancreatic Function 11.8.6 Nasal Potential Difference Measurements 11.8.7 Bronchoalveolar Lavage 11.8.8 Respiratory Tract Microbiology 11.8.9 Newborn Screening 11.9 National Registries: The Crucial Provision to Monitor Progress 11.10 Different Class Mutations of Cystic Fibrosis 11.11 Treatment options 11.11.1 Gene Therapy 11.11.1.1 Importance of Gene Therapy 11.11.1.2 Challenges for Gene Therapy 11.11.1.3 Gene Editing 11.11.1.4 Gene Transfer Involving Both Viral and Non-viral Gene Therapy 11.11.1.5 In-utero Gene Therapy for Cystic Fibrosis 11.11.2 Small Molecules Used in the Treatment of Cystic Fibrosis 11.11.2.1 Potentiator 11.11.2.2 Stabilizer 11.11.2.3 Corrector 11.11.2.4 Amplifier 11.11.3 Managing a Good Nutritional State in Older Children 11.11.4 Gastrointestinal Therapies 11.11.5 Pulmonary Therapies 11.11.6 Organ Transplantations 11.12 Common Issues Complicating Cystic Fibrosis and Its Treatment 11.13 Conclusion References Chapter 12: Non-Viral Delivery of Genome-Editing Nucleases for Gene Therapy 12.1 Introduction 12.2 Methods of Delivery and Nonviral Approaches for Gene Editing Nucleases 12.2.1 Electroporation 12.2.1.1 Selections of Cargoes for Gene Therapy 12.2.1.1.1 Plasmid DNA 12.2.1.1.2 mRNA 12.2.1.1.3 Proteins 12.3 Hydrodynamic Delivery 12.4 Lipid Nanoparticles 12.5 Polymer Based Nanoparticles 12.6 Cell Penetrating Peptides 12.7 DNA Origami References Chapter 13: CRISPER Gene Therapy Recent Trends and Clinical Applications 13.1 Introduction 13.2 CRISPR–Cas9 System 13.2.1 Structure of CRISPR Loci 13.3 The Mechanism Involved in the CRISPR–Cas System 13.3.1 Adaptation of CRISPR–Cas Spacer Sequences 13.3.2 Expression and Maturation of CRISPR–Cas System 13.3.3 Interference of CRISPR–Cas System 13.4 Biology of Type II CRISPR–Cas9 13.5 Recent Trends in CRISPER Gene Therapy 13.5.1 CRISPR System Accurately Cut Target DNA 13.5.2 Non-Homologous End Joining (NHEJ) and (Homology Directed Repair) HDR 13.5.3 On-Target Activity and Off-Target Activity 13.5.4 CRISPR Tools 13.5.5 CRISPR in Gene Editing 13.6 Clinical Application of CRISPER Gene Therapy 13.6.1 Gene Disruption 13.6.2 In vivo CRISPR Gene Therapy 13.6.3 CRISPR Editing in Human Embryos and Ethical Considerations 13.7 Conclusion References Chapter 14: Clinical Applications of Gene Therapy for Immuno-Deficiencies 14.1 Primary Immunodeficiencies (PIDS) 14.1.1 Severe Combined Immunodeficiencies 14.1.1.1 Adenosine Deaminase-Deficient Severe Combined Immunodeficiency (ADA-SCID) 14.1.1.2 X-linked Severe Combined Immunodeficiency (X-SCID) 14.1.1.3 Artemis SCID and Recombinase-activating Gene 1 (RAG1) Deficiency 14.1.2 Combined Immunodeficiencies 14.1.2.1 Wiskott–Aldrich Syndrome (WAS) 14.1.3 Disorders of Phagocyte Number and Function 14.1.3.1 Chronic Granulomatous Disease (CGD) 14.1.3.2 Leucocyte Adhesion Defect Type 1 (LAD-1) 14.1.4 Diseases of Immune Dysregulation 14.1.4.1 Familial Hemophagocytic Lymphohistiocytosis (FLH) 14.1.4.2 X-Linked Lymphoproliferative Disease 1 (XLP1) 14.1.4.3 Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked (IPEX) Syndrome 14.2 Acquired Immunodeficiencies (AIDS) 14.3 Conclusion References Chapter 15: Regulatory Challenges for Gene Delivery 15.1 Introduction 15.1.1 Gene Delivery 15.1.1.1 Somatic Gene Editing 15.1.1.2 Genome Editing Technologies: Viral Vectors and Nonviral Vectors 15.1.2 Clinical Trials in Gene Therapy 15.1.2.1 Clinical Trials in Liver 15.1.2.2 Clinical Trials in Rare Diseases 15.1.2.3 Clinical Trials in Cancer 15.1.3 Ethical Considerations for Gene Therapy 15.1.3.1 Regulations for Somatic Gene Editing in United States 15.1.3.2 Regulations for Somatic Gene Editing in United Kingdom 15.1.3.3 Current Regulatory Framework: Is It Fit for Purpose? 15.2 The Legal and Regulatory Landscape in Gene Therapy 15.2.1 EU Guidelines on Quality, Non-Clinical, and Clinical Aspects of Medicinal Products Containing Genetically Modified Cells 15.2.2 EU (Draft) Guideline on Quality, Non-Clinical, and Clinical Requirements for Investigational Gene Therapy in Clinical Trials 15.2.3 EU (Draft) Guideline on Safety and Efficacy Follow-Up and Risk Management of Gene Therapy Products 15.2.4 Gene Editing Regulations 15.2.4.1 Human Gene Editing Regulations 15.2.4.2 Animal Gene Editing Regulations 15.2.4.3 Plant Gene Editing Regulations 15.3 Conclusion and Future Trends References Index