These models have shown limited utility for developing therapies targeting MSLN: while preclinical studies targeting MSLN in pancreatic cancer cell lines and patient-derived xenograft mouse models have resulted in inhibition of cancer growth8,9, early-stage clinical trials have shown stabilized disease or increase in survival for only a few patients10,11, with partial and overall responses being mostly low or absent12,13. The gene discussed is MSLN; the disease is familial pancreatic carcinoma.