Background: Mutations in the tumor suppressor gene Adenomatous Polyposis Coli () are found in 80% of sporadic colorectal cancer (CRC) tumors and are also responsible for the inherited form of CRC, Familial adenomatous polyposis (FAP).
Methods: To identify novel therapeutic strategies for the treatment of mutated CRC, we generated a drug screening platform that incorporates a human cellular model of mutant CRC using CRISPR-cas9 gene editing and performed an FDA-approved drug screen targeting over 1000 compounds.
Results: We have identified the group of HMG-CoA Reductase (HMGCR) inhibitors known as statins, which cause a significantly greater loss in cell viability in the mutated cell lines and in mutated patient derived xenograft (PDX) models, compared to wild-type cells.
Kawasaki disease (KD) is a paediatric vasculitis associated with coronary artery aneurysms (CAA). Genetic variants influencing susceptibility to KD have been previously identified, but no risk alleles have been validated that influence CAA formation. We conducted a genome-wide association study (GWAS) for CAA in KD patients of European descent with 200 cases and 276 controls.
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