A practical guide for researchers and professionals applying genome editing techniques to drug discovery
In Genome Editing in Drug Discovery, a team of distinguished biologists delivers a comprehensive exploration of genome editing in the drug discovery process, with coverage of the technology’s history, current issues and techniques, and future perspectives and research directions. The book discusses techniques for disease modeling, target identification with CRISPR, safety studies, therapeutic editing, and intellectual property issues.
The safety and efficacy of drugs and new target discovery, as well as next-generation therapeutics are also presented.
Offering practical suggestions for practitioners and academicians involved in drug discovery, Genome Editing in Drug Discovery is a fulsome treatment of a technology that has become part of nearly every early step in the drug discovery pipeline. Selected contributions also include: - A thorough introduction to the applications of CRISPRi and CRISPRa in drug discovery - Comprehensive explorations of genome-editing applications in stem cell engineering and regenerative medicine - Practical discussions of the safety aspects of genome editing with respect to immunogenicity and the specificity of CRISPR-Cas9 gene editing - In-depth examinations of critical socio-economic and bioethical challenges in the CRISPR-Cas9 patent landscape
Perfect for academic researchers and professionals in the biotech and pharmaceutical industries, Genome Editing in Drug Discovery will also earn a place in the libraries of medicinal chemists, biochemists, and molecular biologists.
Table of Contents
Preface vii
List of Abbreviations viii
List of Contributors ix
Part 1 Introduction to Drug Discovery and Genome Editing Methods 1
1 Genome Editing in Drug Discovery 3
Steve Rees
2 Historical Overview of Genome Editing from Bacteria to Higher Eukaryotes 9
Marcello Maresca
3 CRISPR Cas: From Bacterial Adaptive Immunity to the Swiss Army Knife of Drug Discovery 19
Saša Šviković
4 Commercially Available Reagents and Contract Research Services for CRISPR-Based Studies 47
Klio Maratou, Aaron T. Cheng, Fiona M. Behan, Ning Sun, and Quinn Lu
5 Computational Tools for Target Design and Analysis 61
Gue-Ho Hwang and Sangsu Bae
Part 2 Genome Editing in Disease Modeling 73
6 Genome Editing in Cellular Disease Models 75
Pierre Theurey, Kader Thiam, Yacine Chérifi, Alexandre Fraichard, and Amélie Rezza
7 Utilizing CRISPR/Cas9 Technologies for in vivo Disease Modeling and Therapy 93
Lukas Badertscher and Michelle J. Porritt
Part 3 Genome Editing in Target Identification and Validation 111
8 Pooled CRISPR KO Screens for Target Identification 113
Antje Grotz and Sumit Deswal
9 Functional Genomics: Arrayed CRISPR KO Screens 127
Davide Gianni and Leire Escudero-Ibarz
10 Applications of CRISPRi and CRISPRa in Drug Discovery 139
Luke A. Gilbert
11 Sequence Diversification Screens with CRISPR-Cas9-Guided Base Editors 151
Jenna Persson and Bernhard Schmierer
12 Single-Cell Transcriptomics and Epigenomics for CRISPR-Mediated Perturbation Studies 165
Saumyaa Saumyaa, Ramy Elgendy, and Alessandro Bonetti
Part 4 Therapeutic Genome Editing 175
13 DNA Repair Pathways in the Context of Therapeutic Genome Editing 177
Sandra Wimberger, Nina Akrap, and Amir Taheri-Ghahfarokhi
14 DNA Base Editing Strategies for Genome Editing 193
Matthew Coelho, Songyuan Li, and Benjamin JM Taylor
15 RNA Base Editing Technologies for Gene Therapy 203
Sumit Deswal
16 Genome Editing Applications in Cancer T Cell Therapy 213
William A. Nyberg and Justin Eyquem
17 Genome-Editing Applications in Stem Cell Engineering and Regenerative Medicine 231
Pragya Gupta, Shashank Jaitly, Priya Thakur, Sangam Giri Goswami, Nupur Bhargava, and Sivaprakash Ramalingam
18 Delivery and Formulation Methods for Therapeutic Genome Editing 261
Erik Oude Blenke and Venkata R. Krishnamurthy
19 Safety Aspects of Genome Editing: Immunogenicity 281
Rakesh Kantilal Chandode and Roberto Nitsch
20 Specificity of CRISPR-Cas9 Gene Editing 289
Niklas Selfjord, Alexandra Madsen, and Pinar Akcakaya
Part 5 Intellectual Property Aspects and Future Prospects 313
21 Key Socio-Economic and (Bio)Ethical Challenges in the CRISPR-Cas9 Patent Landscape 315
Franc Mali
22 Emerging Technologies for Genome Editing 329
Martin Peterka
Index 337
