Contents

Preface

Chapter 1

Advances in Research and Applications of CRISPR

Abstract

Introduction

Gene Therapy

Cancer Treatment

Genetic Disease Treatment

Sickle Cell Disease (SCD) and β-Thalassemia

Type B Hemophilia

Muscular Dystrophy

Virus Infection Treatment

Human Immunodeficiency Virus (HIV)

Drug Target Identification and Validation

Genetics and Breeding

Conclusion

Acknowledgments

References

Chapter 2

CRISPR/Cas Genome Editing: Targeting Hepatic Diseases

Abstract

Introduction

Systemic Metabolic Disorders

Haemophilia B

PCSK9 & Familial Hypercholesterolemia

Hereditary Tyrosinemia Type I (HT-I)

Virus Infection

Hepatocarcinoma

Concerns in the Cas9 Delivery Systems

Transduction Efficiency

Specificity

Safety-Immunogenicity

Safety-Genotoxicity

Conclusion

References

Chapter 3

Applications and Advances of CRISPR/Cas9, a Versatile Tool for Genome Editing in Animal Models

Abstract

A List of Non-Standard Abbreviations

CRISPR and CRISPR/Cas9 System

Applications of CRISPR Technology on Animal Model Generation

Monkey

Rat

Mouse

Pig

Sheep

Zebrafish

Drosophila Melanogaster

Caenorhabditis Elegans

Conclusion and Perspective

Acknowledgments

References

Chapter 4

Gene Targeting on Cyp2c Locus in Rats Using the CRISPR/Cas9 System

Abstract

A List of Non-Standard Abbreviations

Introduction

Methods

Materials and Chemicals

Animals

Cyp2c6, Cyp2c11 and Cyp2c12 KO Target Site Selection

DNA Constructs and In Vitro Transcription

Co-Microinjection of sgRNA and Cas9 mRNA into Zygotes

Genotyping of Founders and Progenies

Results

Cyp2c6, Cyp2c11 and Cyp2c12 KO Target Site Selection

Genotyping of F0 Founders

Discussion

Acknowledgments

References

Chapter 5

Applications of CRISPR/Cas9 for Therapy in Human Genetic Diseases

Abstract

Introduction

Applications of CRISPR/Cas9 Genome Editing in Diseases

CRISPR/Cas9 Gene-Editing in Point Mutation-Induced Diseases

CRISPR/Cas9 Gene-Editing in Gene or Genome Deletion-Related Diseases

CRISPR/Cas9 Gene-Editing in Chromosomal Inversion- Related Diseases

Specificity and Safety

Conclusion

References

Chapter 6

Utilization of CRISPR in Gene Function and Drug Target Validation

Abstract

Introduction

Characteristics of CRISPR/Cas9 System

RNAi versus CRISPR/Cas9

Different CRISPR/Cas9 Systems

Applications of CRISPR/Cas

Mechanistic Toxicology

Disease Models

Drug Target Identification

Drug Resistance-Associated Gene Identification

Conclusion

References

Chapter 7

Applications of CRISPR/Cas9 in Plant Genome Editing

Abstract

Introduction

Cases About CRISPR/Cas9 in Plant Gene Editing

Arabidopsis Thaliana

Brassica Oleracea

Citrus Sinensis

Cucumis Sativus

Glycine Max

Hordeum Vulgare

Marchantia Polymorpha

Medicago Truncatula

Nicotiana Benthamiana

Nicotiana Tabacum

Oryza Sativa

Petunia Hybrid

Populus Tomentosa

Solanum Lycopersicum

Solanum Tuberosum

Triticum Aestivum

Vitis Vinifera

Zea Mays

Discussion

Medicinal Plant Gene Function Study

The Generation of New Better Crop Varieties

The Difficulties Needed to Be Overcome in Plant Genome Editing

Conclusion

References

Chapter 8

Genome Editing on Human Embryos by CRISPR is Attractive but Controversial

Abstract

Introduction

Gene Editing on Embryos Using CRISPR

Conclusion

References

About the Editor

Index

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