Genome Editing in Drug Discovery

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.

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 a Senior 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.

Prelim pages

Contents

Preface ((to follow))

1. Genome editing in drug discovery

2. Historical overview of Genome Editing from bacteria to higher eukaryotes

3. CRISPR Cas: from bacterial adaptive immunity to the swiss army knife of drug discovery

4. Commercial reagents and services for genome editing

5. Computational tools for target design and analysis

6. Genome editing in cellular disease models

7. Utilizing CRISPR/Cas9 technologies for in vivo disease modeling and therapy

8. Pooled CRISPR KO screens for target identification

9. Functional genomics: arrayed CRISPR KO screens

10. Applications of CRISPRi and CRISPRa in drug discovery

11. High throughput targeted mutagenesis by CRISPR-Cas9 base editing

12. Single-cell transcriptomics and epigenomics for CRISPR-mediated perturbation studies

13. DNA repair pathways in the context of therapeutic genome editing

14. DNA base editing strategies for genome editing

15. RNA base editing technologies for gene therapy

16. Genome Editing applications in cancer T Cell Therapy

17. Genome-editing applications in stem cell engineering and regenerative medicine

18. Delivery and Formulation Methods for Therapeutic Genome Editing

19. Safety aspects of genome editing: Immunogenicity

20. Specificity of CRISPR-Cas9 gene editing

21. Key socio-economic and (bio)ethical challenges in the crispr-cas9 patent landscape

22. Future prospects and emerging technologies for genome editing

Subject Index

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