Gene Delivery: Nanotechnology and Therapeutic Applications

Description

Gene delivery is a transport of genes of therapeutic values into the chromosomes of the cells or tissues which can be targeted to replace the faulty genes. In last two decades lot of research efforts are dedicated to gene delivery for therapeutic applications. Today gene therapy is promising approach in treatment of genetic diseases including mitochondrial related diseases like blindness, muscular dystrophy, cystic fibrosis, and some cancers. Gene Delivery Systems: Nano Delivery Technologies observes the exploration of nanotechnology for gene therapy and gene delivery. Written by prominent authors in the field, this book covers various aspects of gene delivery including challenges in delivering gene therapy, advances in genome editing, RNA-based gene therapy, Green nanoparticles for oligonucleotide delivery. Additional features include" Provides the most up to date information on the development of gene therapy, from the technology involved to gene correction and genome editing. Includes knowledge of the current application of CRISPR/Cas9 gene-editing technique; an approach that has recently been given the Noble Prize. Examines the development of mRNA vaccines for Covid -19 in challenging pandemic scenario Discusses siRNA, mRNA, and DNA plasmids. 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 (