4.5 Review

CRISPRing future medicines

Journal

EXPERT OPINION ON DRUG DISCOVERY
Volume 16, Issue 4, Pages 463-473

Publisher

TAYLOR & FRANCIS LTD
DOI: 10.1080/17460441.2021.1850687

Keywords

CRISPR; CRISPR-engineered animal and cellular models; cell and gene therapy; drug discovery; functional genomic screening; genome engineering; target discovery; target validation

Funding

  1. Charles River Laboratories, Leiden, The Netherlands

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CRISPR technology is rapidly revolutionizing drug discovery by providing a simple way for genome engineering in the early stages of drug development, and it has attracted attention for application in cell and gene therapy. The technology is becoming the preferred tool for genome engineering and is expected to play a key role in supporting genome engineering requirements in drug discovery as well as in cell and gene therapy development in the future.
Introduction: The ability to engineer mammalian genomes in a quick and cost-effective way has led to rapid adaptation of CRISPR technology in biomedical research. CRISPR-based engineering has the potential to accelerate drug discovery, to support the reduction of high attrition rate in drug development and to enhance development of cell and gene-based therapies. Areas covered: How CRISPR technology is transforming drug discovery is discussed in this review. From target identification to target validation in both in vitro and in vivo models, CRISPR technology is positively impacting the early stages of drug development by providing a straightforward way to genome engineering. This property also attracted attention for CRISPR application in the cell and gene therapy area. Expert opinion: CRISPR technology is rapidly becoming the preferred tool for genome engineering and nowadays it is hard to imagine the drug discovery pipeline without this technology. With the years to come, CRISPR technology will undoubtedly be further refined and will flourish into a mature technology that will play a key role in supporting genome engineering requirements in the drug discovery pipeline as well as in cell and gene therapy development.

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