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Edited by

Marcello Maresca

AstraZeneca, BioPharmaceuticals R&D

Mölndal, Sweden

Sumit Deswal

AstraZeneca, BioPharmaceuticals R&D

Mölndal, Sweden

This edition first published 2022. © 2022 John Wiley & Sons, Inc.

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Library of Congress Cataloging‐in‐Publication Data

Names: Maresca, Marcello, editor. | Deswal, Sumit, editor.

Title: Genome editing in drug discovery / Marcello Maresca, AstraZeneca, BioPharmaceuticals R&D, Mölndal, Sweden, Sumit Deswal, AstraZeneca, BioPharmaceuticals R&D, Mö¨lndal, Sweden.

Description: First edition. | Hoboken, NJ : Wiley, 2022. | Includes index.

Identifiers: LCCN 2021025952 (print) | LCCN 2021025953 (ebook) | ISBN 9781119671343 (hardback) | ISBN 9781119671381 (adobe pdf) | ISBN 9781119671398 (epub)

Subjects: LCSH: Drug development. | Genetic engineering.

Classification: LCC RM301.25 .G46 2022 (print) | LCC RM301.25 (ebook) | DDC 615.1/9–dc23

LC record available at https://lccn.loc.gov/2021025952LC ebook record available at https://lccn.loc.gov/2021025953

Cover image: © Yurchanka Siarhei/Shutterstock

Cover design by Wiley

The development of CRISPR‐Cas9 for genome engineering has revolutionized the field of genome editing. Many of the cell types and animal models previously very challenging to genetic engineering, can now be engineered with high efficiency and precision using CRISPR‐Cas9‐derived tools. This has led to the development of many novel disease models helping scientists to better understand disease biology as well as providing opportunity to test novel therapeutics. By performing large‐scale functional genomics screens with CRISPR‐Cas9, it is now possible to identify and validate drug targets at a much faster rate and better precision. By assessing gene function comprehensively at large scale and in relevant cell type in early stages, candidate attrition rate is reduced. Genome editing is now part of almost every step in the early part of drug discovery pipeline, from target identification and its validation to mechanistic studies in relevant disease models. In addition, genome editing is used as a promising platform for gene therapy and molecular diagnostics.

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