Genome Editing in Drug Discovery

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 that have been developed for single‐cell...

9 Chapter 13Table 13.1 Phase I and II clinical trials with applied designer nucleases a...Table 13.2 Phase I and II clinical trials with designer nucleases in immuno...

10 Chapter 15Table 15.1 A summary of the RNA editing technologies developed so far.

11 Chapter 16Table 16.1 A decade of gene‐targeting experiments in primary T cells.Table 16.2 Knock‐out experiments performed on primary T cells.

12 Chapter 17Table 17.1 List of clinical trials for various human diseases using genome‐...

13 Chapter 18Table 18.1 Overview of viral vectors used for gene therapy.

14 Chapter 20Table 20.1 Summary of in silico off‐target detection tools.Table 20.2 Summary of genome‐wide methods for detecting CRISPR‐Cas9 off‐tar...

1 Chapter 2 Figure 2.1 Graphical overview of genome engineering technologies (upper pane...

2 Chapter 3 Figure 3.1 Phases of CRISPR‐mediated immune response. ( a) The simplifi... Figure 3.2 Overview of class 1 and class 2 CRISPR systems. General compositi... Figure 3.3 crRNA biogenesis pathways. (a) depicts a canonical crRNA biogenes... Figure 3.4 Interference mechanism in class 1 systems. Panel ( a)depicts inte... Figure 3.5 Interference mechanisms in class 2 systems. Representative mechan... Figure 3.6 Adaptation phase(s) in CRISPR system. Depending on whether invadi... Figure 3.7 Applications of CRISPR systems beyond genome editing. Fusing cata...

3 Chapter 4 Figure 4.1 Process workflow for project externalization of genome editing to...

4 Chapter 6Figure 6.1 Critical steps for the generation of a relevant cellular disease ...

5 Chapter 7Figure 7.1 Overview of different Cas9 variants and depiction of selected del...

6 Chapter 8Figure 8.1 CRISPR screening approaches. To introduce CRISPR edits, a cell po...Figure 8.2 Synthetic lethality and lineage dependencies discovery using pool...Figure 8.3 Positive selection vs a negative selection pooled screen.Figure 8.4 Workflow of pooled CRISPR‐Cas screen analysis pipeline.Figure 8.5 A multitude of choices to be made for planning cancer‐immunothera...

7 Chapter 9Figure 9.1 Genome‐wide arrayed CRISPR screening platform.Overview of ...Figure 9.2 Arrayed lentiviral CRISPR screening platform.Overview of a stand...

8 Chapter 10Figure 10.1 dCas9 is a programmable RNA‐guided DNA‐binding platform that can...Figure 10.2 An approach for pooled CRISPRi/a screens in human cells. Custom ...

9 Chapter 11Figure 11.1 General principle of a pooled CRISPR screen.The example shown i...Figure 11.2 Top. Workflow of a pooled sequence diversification screen using...Figure 11.3 Applications of sequence diversification screens.(a) The major ...

10 Chapter 12Figure 12.1 Schematic representation of scRNA‐seq approaches for evaluating ...

11 Chapter 13Figure 13.1 Schematic representation of the major DNA double‐strand break (D...Figure 13.2 Transcript abundance of DSB sensor proteins across normal tissue...Figure 13.3 Genome editing strategies utilizing non‐homologous end joining (...

12 Chapter 14Figure 14.1 Cytosine and adenine base editing in DNA. (a) Architecture of cy...Figure 14.2 Decision flow chart for choosing base editing tools.

13 Chapter 15Figure 15.1 Brief history of developments in the RNA editing technologies.Figure 15.2 ADAR proteins structure and function. (a) Chemical reaction cata...Figure 15.3 Different ADAR‐based RNA editing systems. Top 3 (a–c) use endoge...

14 Chapter 16Figure 16.1 A schematic representation of the TCR complex and the three gene...

15 Chapter 17Figure 17.1 Timeline of genome‐editing nucleases and genome‐editing research...Figure 17.2 Ex vivo genome editing‐based autologous cell replacement therapy...Figure 17.3 In vivo genome‐editing approach for various human diseasesFigure 17.4 Genome‐editing approaches to enhance dystrophin levels.DM...

16 Chapter 18Figure 18.1 Three main pillars of delivery methods. Ex‐vivo manipulation of ...Figure 18.2 The different components required for therapeutic gene editing. ...Figure 18.3 Modified mRNA in a lipid nanoparticle.Modified mRNA or “mod‐RNA...Figure 18.4 Internal structure and components of a lipid nanoparticle.Nucle...Figure 18.5 Chemical structures of cationic ionizable lipids.Dlin‐MC3‐DMA o...Figure 18.6 Chemical structures of polymers for nucleic acid delivery.Diffe...Figure 18.7 Different applications require different delivery strategies.Zi...

17 Chapter 19Figure 19.1 Innate and adaptive immune responses to various CRISPR delivery....

18 Chapter 20Figure 20.1 Cell‐based off‐target detection methods. (a) IDLV capture. IDLV ...Figure 20.2 In situ off‐target detection methods. (a) BLESS. Following treat...Figure 20.3 In vitro off‐target detection methods. (a) Digenome‐/DIG‐seq. Pu...Figure 20.4 Overview of methods for reduction of genome‐wide off‐targets. (a...

19 Chapter 22Figure 22.1 Schematic representation of prime editing.

1 Cover Page

2 Genome Editing in Drug Discovery

3 Copyright Page

4 Preface

5 List of Abbreviations

6 List of Contributors

7 Table of Contents

8 Begin Reading

9 Index

10 WILEY END USER LICENSE AGREEMENT

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