Biotechnologies for Gene Therapy : RNA, CRISPR, Nanobots, and Preclinical Applications

The purpose of this book is to highlight some of latest developments and applications of CRISPR, RNA, and DNA to treat diseases ranging from cancers to cardiovascular and degenerative disorders. It also features innovations of the delivery methods for nucleic acids ranging from nanodevices made from DNA and pseudo amino acids to viral vectors. This is an ideal book for academics, clinicians, and students interested in gene therapy. Preface Contents About the Editors Therapeutic Potential of MicroRNAs and Their Nanoparticle-based Delivery in the Treatment of Liver Fibrosis 1 Introduction 2 The Role of Hepatic Stellate Cells in Liver Fibrosis 3 The Roles of MicroRNAs in Liver Fibrosis 3.1 Potential Antifibrotic miRNAs 3.2 Potential Profibrotic miRNAs 4 Nanoparticle-based Delivery of MiRNA for Liver Fibrosis Therapy 5 Conclusions References MicroRNA as a Versatile Regulator of Wnt the Signaling Pathway in Colorectal Cancer 1 Introduction 2 General Pathogenesis of Colorectal Cancer 3 Overview of Wnt Signaling Pathways 3.1 Canonical Pathway 3.2 Noncanonical Pathway 3.3 Dysregulation of Wnt Signaling Pathway in CRC 4 MicroRNA as an Important Regulator of Wnt Signaling Pathway in CRC Pathogenesis 4.1 MiRNAs Regulating Oncogenic Potential in CRC Via Wnt Signaling 4.2 MiRNAs Regulating Drug Resistance of CRC Via Wnt Signaling 4.3 MiRNAs Regulated by the Wnt/Myc Axis and their Role in CRC 5 Conclusion and Future Direction References Small Activating RNA Therapy for Angiogenesis 1 Introduction 2 Small Noncoding RNAs 2.1 Small Activating RNA Classes 2.2 Small Activating ncRNA Functions in the Cells 3 Small Activating RNAs in the Regulation of Angiogenesis 4 Nucleic Acid-based Therapy 5 Future Vision References Muscular Dystrophy Therapy Using Viral Vector-based CRISPR/Cas 1 Introduction 2 CRISPR/Cas: From Immune Systems to Genome Editing Tool 3 Strategies for CRISPR-mediated DMD Mutation Correction 4 Adeno-Associated Viral Vector-based Delivery of CRISPR/Cas 5 Adenoviral Vector-based Delivery of CRIPSR/Cas 6 Lentiviral Vector-based Delivery of CRISPR/Cas 7 Challenges 8 Conclusions References CRISPR-Cas-Based Gene Therapy to Target Viral Infections 1 Introduction 2 Discovery and Description of the CRISPR-Cas Mechanism 3 CRISPR-Cas as a Gene-Editing Tool 4 CRISPR-Cas as a Diagnostic Tool 5 CRISPR Editing To Control Pathogenic Viruses 6 RNA Viruses 6.1 Hepatitis C Virus 6.1.1 Targeting Viral RNA 6.1.2 Targeting Host Factors 6.2 Human Immunodeficiency Virus 6.2.1 Targeting Viral RNA 6.2.2 Targeting Viral DNA 6.2.3 Targeting Host Factors 6.3 Dengue Virus 6.3.1 Targeting Viral RNA 6.3.2 Targeting Host Factors 6.4 Influenza A Virus 6.4.1 Targeting the Viral Genome 6.4.2 Targeting Host Factors 6.5 Human Coronaviruses 6.5.1 Targeting Viral RNA 6.5.2 Targeting Host Factors 6.6 Lymphocytic Choriomeningitis Virus 6.7 Vesicular Stomatitis Virus 7 DNA Viruses 7.1 Herpesviruses 7.1.1 Targeting Viral DNA 7.1.2 Viral Genome Modification: Knock-out and Knock-in Strategies 7.2 Human Papilloma Virus 7.2.1 Targeting Viral DNA 7.2.2 Activation of Viral Restriction Factors 7.3 Hepatitis B Virus 7.3.1 Targeting Viral DNA 7.3.2 Targeting Host Factors 8 Challenges and Limitations of CRISPR-Cas Therapeutics 9 Conclusions References CRISPR Targeting the Integrated HTLV-1 Virus 1 Genome Organization 2 Geographical Distribution and Viral Transmission 3 Viral Lifecycle 4 HTLV-1-Associated Diseases 5 In Vitro and In Vivo Models of HTLV-1 Disease 6 CRISPR/Cas9 Targeting of HTLV-1 7 Concluding Remarks References Retroviral Vectors for Gene Therapy of Monogenic Diseases 1 Introduction 2 Retroviral Vectors Based on Gammaretrovirus MLV 3 Lentiviral Vectors Derived from HIV-1 4 Retroviral Vectors Based on Alpharetrovirus ASLV and Spumavirus PFV 5 Conclusions References DNA Origami Nanodevices for Therapeutic Delivery Applications 1 Introduction 2 Design and Fabrication 2.1 Computer-Aided Design Approaches 2.2 Fabrication and Scaling Up Production 2.3 Purification and Storage Methods 3 Characterization Methods 3.1 Gel Electrophoresis 3.2 Microscopy 4 Nontherapeutic Uses for DNA Origami 4.1 Biosensing 4.2 Templating Nanofabrication and Plasmonics 4.3 DNA Computation 4.4 Molecular Machines 5 DNA Origami for Drug Delivery Applications 5.1 DNA Origami Stability in Physiological and High-Temperature Conditions 5.2 Loading and Release Methods 5.3 Targeting DNA Origami Nanostructures 5.4 Cellular Activation 5.5 Vaccine Development 5.6 Therapeutic Oligonucleotide Delivery Including siRNA, Antisense, CRISPR, and Gene Delivery 6 Conclusions References Index