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Cystic fibrosis risk variants confer protection against inflammatory bowel disease

Yu, M.; Zhang, Q.; Yuan, K.; Sazonovs, A.; Stevens, C.; Fachal, L.; the International Inflammatory Bowel Disease Genetics Consortium, ; Anderson, C.; Daly, M. J.; Huang, H.

2024-12-05 genetic and genomic medicine
10.1101/2024.12.02.24318364 medRxiv
Show abstract

Genetic mutations that yield defective cystic fibrosis transmembrane regulator (CFTR) protein cause cystic fibrosis, a life-limiting autosomal recessive Mendelian disorder. A protective role of CFTR loss-of-function mutations in inflammatory bowel disease (IBD) has been suggested, but its evidence has been inconclusive and contradictory. Here, leveraging the largest IBD exome sequencing dataset to date, comprising 38,558 cases and 66,945 controls in the discovery stage, and 35,797 cases and 179,942 controls in the replication stage, we established a protective role of CF-risk variants against IBD based on evidence from the association test of CFTR delF508 (p-value=8.96E-11) and the gene-based burden test of CF-risk variants (p-value=3.9E-07). Furthermore, we assessed variant prioritization methods, including AlphaMissense, using clinically annotated CF-risk variants as the gold standard. Our findings highlight the critical and unmet need for effective variant prioritization in gene-based burden tests.

Published in Cell Genomics (predicted rank #21) · training set

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