PT  - JOURNAL ARTICLE
AU  - Rui Wang
AU  - Sean Graham
AU  - Ning Sun
AU  - Donna McCarthy
AU  - Ruoqi Peng
AU  - Jamie Erickson
AU  - Xiaochun Zhu
AU  - Marc Wurbel
AU  - Robert Dunstan
AU  - Namjin Chung
AU  - Edda Fiebiger
AU  - Tariq Ghayur
AU  - Jijie Gu
TI  - Validation of a CRISPR/cas9-based technology platform for examining specific immune gene functions in an experimental murine model of IBD
AID  - 10.1101/619791
DP  - 2019 Jan 01
TA  - bioRxiv
PG  - 619791
4099  - http://biorxiv.org/content/early/2019/04/26/619791.short
4100  - http://biorxiv.org/content/early/2019/04/26/619791.full
AB  - Inflammatory bowel diseases (IBD) are complex, multifactorial disorders characterized by chronic relapsing intestinal inflammation. Association studies have identified hundreds of genes that are linked to IBD and potentially regulate its pathology. The further dissection of the genetic network underlining IBD pathogenesis and pathophysiology is hindered by the limited capacity to investigate the role of each GWAS association through functional studies, including the generation of knockout animal models for each of the associated genes. The CRISPR/Cas9 system represents a cutting edge technology which has the potential to transform the field of IBD research by facilitating the introduction of genetic alterations in an efficient and effective manner. Using the CD40-mediated-colitis model, our results demonstrate the validity of a CRISPR/Cas9-based platform as a tool for the validation of target genes or interference strategies in experimental IBD. The utilization of this discovery strategy will allow for the timely in vivo validation of therapeutic targets as the rapidly emerge from current genetic and genomics efforts with human disease tissue. As such, the CRISPR/Cas9-based platform can significantly shorten the time span between target identification and generation of proof of principle experiments for drug discovery.