CRISPRing future medicines

ABSTRACT

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.

KEYWORDS:

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

Article highlights

• CRISPR technology has rapidly become the preferred way for genome engineering.

• The CRISPR toolbox is continuously expanding and has the potential to accelerate drug discovery and reduce attrition rates.

• CRISPR-based screening is an additional tool in the field of functional genomics and provides a powerful way to drug target discovery.

• CRISPR technology facilitates the target validation process by providing an easy genome engineering tool to interrogate the target-disease and drug–target relationship and to generate predictive cellular and animal models of disease.

• CRISPR technology is currently being investigated in preclinical and clinical phase for use in cell gene therapy approaches.

This box summarizes key points contained in the article.

Acknowledgments

The authors thank Shushant Jain, Research Leader at Charles River, Leiden for their critical reading of the manuscript. Figures 2 and 3 were created using BioRender’s web-based software (BioRender.com).

Declaration of interest

The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This review was written by employees of and supported by Charles River Laboratories, Leiden, The Netherlands.