Genome Editing in Drug Discovery. Группа авторов

Gene Editing in T Cells 16.4 Gene Editing in T Cell Therapy 16.5 Conclusion References 17 Genome‐Editing Applications in Stem Cell Engineering and Regenerative Medicine 17.1 Introduction 17.2 Hematological Disorders 17.3 Neurodegenerative Diseases 17.4 Duchenne Muscular Dystrophy 17.5 Ocular Diseases 17.6 Inborn Errors of Metabolism 17.7 Lysosomal Storage Disorders (LSDs) 17.8 Hereditary Tyrosinemia Type I (HT‐1) 17.9 Ornithine Transcarbamylase Deficiency (OTCD) 17.10 Primary Immune Deficiencies 17.11 Current Challenges in Therapeutic Genome Editing 17.12 Conclusions Acknowledgements References 18 Delivery and Formulation Methods for Therapeutic Genome Editing 18.1 Introduction 18.2 Payloads and Modalities 18.3 Delivery Technologies 18.4 In vivo Delivery Strategies 18.5 Conclusion References 19 Safety Aspects of Genome Editing 19.1 Introduction 19.2 Immunogenicity 19.3 Delivery‐Dependent Immunogenicity 19.4 Methods to Study Cas9 Immunogenicity 19.5 Mitigation Strategies 19.6 Outlook References 20 Specificity of CRISPR‐Cas9 Gene Editing 20.1 Introduction 20.2 Detecting Genome‐wide Off‐target Effects 20.3 Reducing Genome‐wide Off‐target Effects 20.4 Other Unwanted Effects: Translocations and Large Deletions 20.5 Clinical Implications and Future Directions References

      11  Part 5: Intellectual Property Aspects and Future Prospects 21 Key Socio‐Economic and (Bio)Ethical Challenges in the CRISPR‐Cas9 Patent Landscape 21.1 Introduction 21.2 CRISPR‐Cas9 is Attracting Great Interest from Both the Business‐enterprise and Academic Sectors 21.3 The Business‐enterprise Sector is Ever More Interested in Using the New and Less Restrictive Forms of IPR for CRISPR‐Cas9 21.4 The Academic Research Sector has Created an Extremely Competitive CRISPR‐Cas9 Patent Landscape 21.5 Certain Socioeconomic and (Bio)ethical Concerns Connected with the CRISPR‐Cas9 Patent Landscape 21.6 Conclusion References 22 Emerging Technologies for Genome Editing 22.1 Introduction 22.2 Improving and Expanding the Cas9 Toolbox 22.3 Prime Editing 22.4 Targeted Transposition and Beyond References

      12  Index

      13  End User License Agreement

      List of Tables

      1 Chapter 4Table 4.1 Major providers of CRISPR reagents.Table 4.2 Genome editing service providers.

      2 Chapter 5Table 5.1 Type of CRISPRs and properties.Table 5.2 Web‐based tools for target design with potential off‐target sites...Table 5.3 Editing activity prediction tools.Table 5.4 Editing outcome prediction tools.Table 5.5 Web tools for analysis.

      3 Chapter 6Table 6.1 Variety of in vitro systems available for cellular disease modeli...

      4 Chapter 7Table 7.1 Comparison of Cas9 proteins.

      5 Chapter 8Table 8.1 Some of the available academic and commercial human and mouse poo...

      6 Chapter 9Table 9.1 CRISPR reagent delivery methods. General advantages and limitatio...

      7 Chapter 11Table 11.1 Arrayed versus pooled CRISPR screens.

      8 Chapter 12Table 12.1 Summary of pooled genetic screens that combine CRISPR perturbati...Table 12.2 Epigenomic technologies