16.1 Introduction
16.2 CAR T Cells
16.3 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
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
