Using CRISPR/Cas to study gene function and model disease in vivo

Abstract

The recent discovery of the CRISPR/Cas system and repurposing of this technology to edit a variety of different genomes have revolutionized an array of scientific fields, from genetics and translational research, to agriculture and bioproduction. In particular, the prospect of rapid and precise genome editing in laboratory animals by CRISPR/Cas has generated an immense interest in the scientific community. Here we review current in vivo applications of CRISPR/Cas and how this technology can improve our knowledge of gene function and our understanding of biological processes in animal models.

Creating cell and animal models of human disease by genome editing using CRISPR/Cas9

“…Subsequently, some efforts were made to combine the aforementioned technology with somatic cell nuclear transfer, aiming to increase its efficiency, all of which unfortunately ended in failure . Recently, the world of modern medicine has been revolutionized to a great degree with the discovery of novel genome‐editing‐based gene therapy methods . Because experimentation on humans is unethical and impractical with respect to identifying the basic mechanisms causing disease, biomedical researchers use animal or cellular models to mimic human diseases; as an example, mice have a set of genes similar to humans and hence are qualified for experimentation .…”

“…9 Recently, the world of modern medicine has been revolutionized to a great degree with the discovery of novel genome-editing-based gene therapy methods. 118 Because experimentation on humans is unethical and impractical with respect to identifying the basic mechanisms causing disease, biomedical researchers use animal or cellular models to mimic human diseases; as an example, mice have a set of genes similar to humans and hence are qualified for experimentation. 119 As a result of the unlimited resources and the simplicity of genetic manipulation in animal models, it has been possible to conduct animal studies with the aim of achieving therapeutic goals.…”

50 years of The FEBS Journal: looking back as well as ahead

“…The first CRISPR papers appeared in 2014 , with a fantastic review published at the end of 2014 that brought many new potential authors and readers to the journal and resulted in a feed‐forward loop of wonderful papers and reviews . The interest in the topic was so great that, in 2016, John Doench coordinated a Special Issue devoted to CRISPR/Cas9‐mediated gene editing .…”

Cancer diagnosis and immunotherapy in the age of CRISPR

“…Some of the earliest applications took advantage of the ability of the CRISPR‐Cas9 system to generate true gene knockouts as an effective alternative to RNAi for genetic screening purposes, and the use of CRISPR screens in cancer has been previously summarized . The use of CRISPR for the creation of new and more accurate tumor models is also well established and has been reviewed elsewhere . However, CRISPR has the potential to be much more than a research tool and CRISPR technologies are increasingly moving away from the bench and toward the clinic.…”

“…[17][18][19] The use of CRISPR for the creation of new and more accurate tumor models is also well established and has been reviewed elsewhere. [20][21][22] However, CRISPR has the potential to be much more than a research tool and CRISPR technologies are increasingly moving away from the bench and toward the clinic. Recent developments have highlighted the ability of CRISPR-based genome editing to improve cancer immunotherapies based on adoptive cell transfer, and have introduced novel, CRISPR-based, detection tools for DNA and RNA, with implications for tumor diagnosis and genotyping.…”