http://www.loc.gov/mods/v3 http://www.loc.gov/standards/mods/v3/mods-3-2.xsd
Reprogramming the Genome: CRISPR-Cas-based Human Disease Therapy, presents the collation of chapters written by eminent scientists worldwide. CRISPR-Cas9 is a key technology for targeted genome editing and regulation in a number of organisms including mammalian cells. It is a rapid, simple, and cost-effective solution. CRISPR-Cas system has recently gained much scientific and public attention. This volume covers CRISPR-Cas9 based mammalian genome editing, creating disease models, cancer therapy, neurological, heredity, blood disorders, defective gene correction, stem cells therapy, epigenetic modifications, patents, ethics, biosafety and regulatory issues challenges and opportunities. This book is a key source of information on mammalian genome editing available in a single volume. This book will be useful for beginners in mammalian genome editing and also students, researchers, scientists, policymakers, clinicians and stakeholders interested in genome editing in several areas. Offers basic understanding and a clear picture of mammalian genome editing through CRISPR-Cas systems Discusses how to create mammalian disease models, stem cell modification, epigenetic modifications, correction of defective gene in blood disorders, heredity, neurological disorders and many more Discusses the application of CRISPR-Cas9 systems in basic sciences, biomedicine, molecular biology, translational sciences, neurobiology, neurology, cancer, stem cells, and many more.
Intro -- Reprogramming the Genome: CRISPR-Cas-based Human Disease Therapy -- Copyright -- Contents -- Contributors -- Preface -- Chapter One: An introduction to CRISPR-Cas systems for reprogramming the genome of mammalian cells -- 1. Introduction -- 2. Development of genome engineering technology -- 2.1. Zinc finger nucleases -- 2.2. Transcription activator-like effector nucleases (TALENs) -- 2.3. CRISPR-Cas9 system -- 3. Progress and applications of CRISPR-Cas9 system -- 3.1. Duchenne muscular dystrophy -- 3.2. CRISPR-Cas9 system for cancer therapy -- 3.3. CRISPR-Cas9 system for blood disorders -- 3.4. CRISPR-Cas9 system in epigenetics -- 4. Conclusion and future remarks -- Acknowledgments -- References -- Chapter Two: CRISPR-Cas systems for genome editing of mammalian cells -- 1. Introduction -- 2. CRISPR-Cas9 system for genome editing of mammalian cells -- 3. CRISPR-Cas9 for creating cell and animal models -- 4. CRISPR-Cas9 system for improving mammalian health -- 5. Base editing in mammalian cells -- 6. Conclusion and future remarks -- Acknowledgment -- References -- Chapter Three: Genome-wide detection and analysis of CRISPR-Cas off-targets -- 1. Introduction -- 1.1. CRISPR-Cas off-target detection methodologies -- 1.2. Off-target detection in vitro with GUIDE-Seq -- 2. GS-preprocess: A GUIDE-seq data preprocessing tool -- 2.1. Implementation and functionality -- 2.2. Resource usage and per-sample cost -- 2.3. Utility of GS-process and GUIDEseq -- 3. Vignette -- 3.1. Sequencing and preparation -- 3.2. Sample demultiplexing and input generation -- 4. Conclusion -- References -- Chapter Four: CRISPR-based diagnostics for detection of pathogens -- 1. Introduction -- 2. Progress and development of CRISPR-Cas systems -- 3. CRISPR-Cas9 mediated diagnostics -- 4. CRISPR-Cas12 mediated diagnostics -- 5. CRISPR-Cas13 mediated diagnostics.
6. Conclusion and future remarks -- Acknowledgments -- References -- Chapter Five: Creating cell lines for mimicking diseases -- 1. Introduction: Concepts and characteristics of cell lines -- 2. Genome editing of cell lines: The basics -- 2.1. The CRISPR-Cas9 system -- 2.2. Transfecting cells with the CRISPR/Cas9 machinery -- 2.3. Selection of modified cells and clonal expansion -- 2.4. How efficient is the process? -- 2.5. Validation of cell models: Confirming genotype and phenotype -- 3. Edited cell lines can mimic diseases in vitro: From diabetes to lysosomal diseases -- 3.1. Cell lines for monogenic diseases -- 3.1.1. Inborn errors of metabolism -- 3.1.2. Cystic fibrosis -- 3.1.3. Huntington´s disease -- 3.1.4. X-linked sideroblastic anemia -- 3.2. Cell lines for complex/multifactorial diseases -- 3.2.1. Kidney disease -- 3.2.2. Type 2 diabetes -- 3.2.3. Cancer -- 4. Conclusions and future remarks -- References -- Chapter Six: Modulating Cas9 activity for precision gene editing -- 1. Introduction -- 2. Targeted gene editing -- 2.1. CRISPR/Cas9 -- 3. A major hurdle: The off-targeting issue in gene editing -- 3.1. gRNA sequence is not solely responsible for off-targeting -- 3.2. Different detection methods for off-targeting mutations -- 3.3. gRNA modifications for increased specificity -- 4. Increasing specificity and on-target activity of Cas9 -- 4.1. Cas9n -- 4.2. fCas9 -- 4.3. spCas9-HF and eSpCas9 -- 4.4. HeFSpCas9s -- 4.5. HypaCas9 -- 4.6. evoCas9 -- 4.7. Sniper Cas9 -- 4.8. HiFi Cas9 -- 4.9. Cas9 variants as base editors and prime editors -- 5. Engineering of Cas9 PAM preferences -- 6. Modulating Cas9 availability -- 6.1. Modulating Cas9 activity and delivery systems -- 6.2. Split-Cas9 systems -- 6.3. Light-inducible Cas9 system -- 6.4. Modulating Cas9 activity with natural protein based inhibitors -- 6.5. Self-limiting Cas9.
Acknowledgments -- References -- Chapter Nine: CRISPR-Cas9 based genome editing for defective gene correction in humans and other mammals -- 1. Introduction -- 2. Gene correction and related strategies -- 2.1. Point mutation -- 2.2. Gene deletion -- 2.3. Genome deletion -- 2.4. Chromosomal rearrangements: Translocation and inversion -- 3. Strategies to improve efficiency of homology-mediated gene editing -- 3.1. Donor DNA-mediated enhancement of HDR efficiency -- 3.2. Small molecules that enhance HDR efficiency -- 4. Delivery methods of the CRISPR/Cas9 gene editing system -- 4.1. Viral delivery methods -- 4.2. Non-viral delivery methods -- 5. Application of CRISPR to the correction of defective genes in humans and other mammals -- 5.1. Correction of monogenic and multifactorial inherited disorders -- 5.1.1. Neurological diseases -- 5.1.2. Hematological diseases -- 5.1.3. Cardiovascular and musculoskeletal diseases -- 5.1.4. Retinal disorders -- 5.1.5. Other diseases -- 6. Conclusions and future perspectives -- Acknowledgments -- Author´s contribution -- Conflict of interests -- References -- Chapter Ten: Recent advances in stem cells and gene editing: Drug discovery and therapeutics -- 1. Introduction -- 2. Biomedical characteristics of stem cells -- 3. Advances in gene editing technologies -- 4. Type II CRISPR system -- 4.1. CRISPR/Cas9-base editors -- 4.2. Prime editors -- 4.3. Type V CRISPR system -- 4.4. Mad7 nuclease -- 4.5. Type VI CRISPR system -- 4.6. REPAIR tool -- 4.7. RESCUE tool -- 5. Biomedical importance of CRISPR/CAS-induced stem cells -- 5.1. Drug discovery -- 5.2. Animal model -- 5.3. Stem cell model -- 6. Therapeutic stem cells generated using CRISPR/Cas platforms -- 6.1. Gene therapy -- 6.1.1. Correction of point mutations -- 6.1.2. Knockin strategy -- 6.1.3. Knockout strategy -- 6.2. Immune response strategy -- 7. Conclusion.
Acknowledgment -- References -- Chapter Eleven: Genome editing of hPSCs: Recent progress in hPSC-based disease modeling for understanding disease mechanisms -- 1. Introduction -- 1.1. Disease modeling using human induced pluripotent stem cells (hiPSCs) -- 1.2. Development of the CRISPR/Cas9 system as a versatile genome-editing tool -- 1.3. Development of genome editing in human pluripotent stem cells (hPSCs) -- 2. Applications of genome-edited stem cells in disease modeling -- 2.1. Neurodegenerative diseases -- 2.2. Cardiovascular diseases -- 2.3. Rare diseases -- 3. Concluding remarks -- Acknowledgments -- References -- Chapter Twelve: Genome editing in cardiovascular diseases -- 1. Introduction -- 2. Use of CRISPR-Cas9 system in the cardiovascular diseases -- 3. Hypertrophic cardiomyopathy -- 4. Dilated cardiomyopathy -- 5. Arrhythmogenic cardiomyopathy -- 6. Conclusion and future perspectives -- References -- Chapter Thirteen: CRISPR/Cas9 in epigenetics studies of health and disease -- 1. Introduction -- 2. Why epigenetic modification is required? -- 3. History of epigenetics -- 4. Types of Epigenetic modification -- 4.1. Histone modification -- 4.1.1. Histone acetylation -- 4.1.2. Histone methylation -- 4.1.3. Histone phosphorylation -- 4.1.4. Histone ubiquitination -- 4.1.5. Histone sumoylation -- 4.1.6. ADP-ribosylation -- 4.2. Non-coding RNA interactions -- 4.3. DNA methylation -- 5. How the role of epigenetics is important about the different physiological process? -- 5.1. Cancer epigenetics -- 5.1.1. Differential epigenetic patterns -- 5.1.2. Activation of oncogenes -- 5.1.3. Suppression of tumor suppressor genes -- 5.1.4. Genome instability -- 5.2. Autoimmune disorder -- 5.3. Neurological disorder -- 5.4. Epigenetics in infectious diseases -- 5.5. Diabetes: Metabolic disorder -- 6. Clinical use of epigenetics studies.
Progress in molecular biology and translational science ; v. 181