Genome Editing in Drug Discovery

ISBN-13: 9781119671343 / Angielski / Twarda / 2022 / 352 str.

Marcello Maresca; Sumit Deswal

Spis treści

Preface viiList of Abbreviations viiiList of Contributors ixPart 1 Introduction to Drug Discovery and Genome Editing Methods 11 Genome Editing in Drug Discovery 3Steve Rees2 Historical Overview of Genome Editing from Bacteria to Higher Eukaryotes 9Marcello Maresca3 CRISPR Cas: From Bacterial Adaptive Immunity to the Swiss Army Knife of Drug Discovery 19Sasa Svikovic4 Commercially Available Reagents and Contract Research Services for CRISPR-Based Studies 47Klio Maratou, Aaron T. Cheng, Fiona M. Behan, Ning Sun, and Quinn Lu5 Computational Tools for Target Design and Analysis 61Gue-Ho Hwang and Sangsu BaePart 2 Genome Editing in Disease Modeling 736 Genome Editing in Cellular Disease Models 75Pierre Theurey, Kader Thiam, Yacine Chérifi, Alexandre Fraichard, and Amélie Rezza7 Utilizing CRISPR/Cas9 Technologies for in vivo Disease Modeling and Therapy 93Lukas Badertscher and Michelle J. PorrittPart 3 Genome Editing in Target Identification and Validation 1118 Pooled CRISPR KO Screens for Target Identification 113Antje Grotz and Sumit Deswal9 Functional Genomics: Arrayed CRISPR KO Screens 127Davide Gianni and Leire Escudero-Ibarz10 Applications of CRISPRi and CRISPRa in Drug Discovery 139Luke A. Gilbert11 Sequence Diversification Screens with CRISPR-Cas9-Guided Base Editors 151Jenna Persson and Bernhard Schmierer12 Single-Cell Transcriptomics and Epigenomics for CRISPR-Mediated Perturbation Studies 165Saumyaa Saumyaa, Ramy Elgendy, and Alessandro BonettiPart 4 Therapeutic Genome Editing 17513 DNA Repair Pathways in the Context of Therapeutic Genome Editing 177Sandra Wimberger, Nina Akrap, and Amir Taheri-Ghahfarokhi14 DNA Base Editing Strategies for Genome Editing 193Matthew Coelho, Songyuan Li, and Benjamin JM Taylor15 RNA Base Editing Technologies for Gene Therapy 203Sumit Deswal16 Genome Editing Applications in Cancer T Cell Therapy 213William A. Nyberg and Justin Eyquem17 Genome-Editing Applications in Stem Cell Engineering and Regenerative Medicine 231Pragya Gupta, Shashank Jaitly, Priya Thakur, Sangam Giri Goswami, Nupur Bhargava, and Sivaprakash Ramalingam18 Delivery and Formulation Methods for Therapeutic Genome Editing 261Erik Oude Blenke and Venkata R. Krishnamurthy19 Safety Aspects of Genome Editing: Immunogenicity 281Rakesh Kantilal Chandode and Roberto Nitsch20 Specificity of CRISPR-Cas9 Gene Editing 289Niklas Selfjord, Alexandra Madsen, and Pinar AkcakayaPart 5 Intellectual Property Aspects and Future Prospects 31321 Key Socio-Economic and (Bio)Ethical Challenges in the CRISPR-Cas9 Patent Landscape 315Franc Mali22 Emerging Technologies for Genome Editing 329Martin PeterkaIndex 337

O autorze

Marcello Maresca, PhD, is a Senior Director in the Discovery Sciences Department at AstraZeneca. He obtained his doctorate in Molecular Biology from the Max Planck Institute of Molecular Cell Biology and Genetics in Dresden, Germany.Sumit Deswal, PhD, is an Associate Principal Scientist with the Genome Engineering Team at AstraZeneca R&D in Sweden. He obtained his doctorate in Immunology from the Max Planck Institute of Immunobiology and Epigenetics in Freiburg, Germany.