Genome Editing in Drug Discovery. Группа авторов

      

      Table of Contents

      1  Cover

      2  Title Page

      3  Copyright Page

      4  Preface

      5  List of Abbreviations

      6  List of Contributors

      7  Part 1: Introduction to Drug Discovery and Genome Editing Methods 1 Genome Editing in Drug Discovery 1.1 Introduction 1.2 Genome Engineering 1.3 CRISPR/Cas9 1.4 Applications of CRISPR Cas9 in Drug Discovery 1.5 Concluding Comments References 2 Historical Overview of Genome Editing from Bacteria to Higher Eukaryotes 2.1 Introduction 2.2 Bacterial DNA Engineering (Recombineering) 2.3 BAC Recombineering 2.4 Metabolic Engineering 2.5 Genetic Engineering in Higher Eukaryotes 2.6 Targeted Endonucleases 2.7 Novel Genome Editing Technologies 2.8 Conclusions References 3 CRISPR Cas 3.1 Introduction 3.2 CRISPR Biology in a Nutshell 3.3 The Diversity of CRISPR Systems 3.4 CRISPR Systems as the Basis for New Tools in Drug Discovery 3.5 Concluding Remarks References 4 Commercially Available Reagents and Contract Research Services for CRISPR‐Based Studies 4.1 Introduction 4.2 CRISPR Resources and Reagents for Bespoke Editing and Genetic Screening 4.3 In vivo CRISPR Screening 4.4 Working with Service Providers for Outsourcing CRISPR Studies 4.5 Considerations on Experimental Design and Controls Required when Outsourcing 4.6 Summary Acknowledgments References 5 Computational Tools for Target Design and Analysis 5.1 Introduction 5.2 Various Types of CRISPR Effectors 5.3 Computational Tools for Target Design 5.4 Summary Funding References

      8  Part 2: Genome Editing in Disease Modeling 6 Genome Editing in Cellular Disease Models 6.1 Gene Editing and Disease Models in Drug Discovery 6.2 Variety of Cellular Disease Models and Their Improvement with Gene Editing 6.3 Choosing and Designing a Relevant Genetically Engineered Cellular Disease Model 6.4 Technical Considerations of Gene Editing in Cells 6.5 Conclusion References 7 Utilizing CRISPR/Cas9 Technologies for in vivo Disease Modeling and Therapy 7.1 Introduction to CRISPR/Cas9 and in vivo Modeling 7.2 CRISPR Editing to Alter Gene Expression 7.3 Choice of Cas9 Species and/or Variant and Ortholog 7.4 Tissue‐Specific CRISPR/Cas9 Gene Editing 7.5 Advantages/Disadvantages of Cas9 Expressing Systems 7.6 Limiting and Detecting Off‐Target Editing in vivo 7.7 Animal Species 7.8 Delivery Systems of CRISPR/Cas9 Components in vivo 7.9 Concluding Remarks References

      9  Part 3: Genome Editing in Target Identification and Validation 8 Pooled CRISPR KO Screens for Target Identification 8.1 Introduction