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Germline inactivation of KLLN has been investigated as a genetic etiology in PTEN‐negative Cowden syndrome (CS) patients. In a cohort of 136 CS/CS‐like individuals, a single patient harbored a germline [NM_001126049.2]c.357C>T (p.Leu119=) variant in KLLN, corresponding to a frequency of <1% (PMID:23446638). No additional segregation data were reported, limiting case‐level evidence for sequence variants in CS.
Functional and epigenetic analyses provide concordant evidence that KLLN acts as a tumor suppressor in CS. Germline hypermethylation of the KLLN promoter occurs in ~37% of PTEN mutation-negative CS patients and associates with elevated risks of breast and renal cancers (PMID:23446638; PMID:25669429). In vitro studies demonstrate that KLLN loss impairs p53-mediated G2 arrest and G1 checkpoint via downregulation of p21, confirming a loss-of-function mechanism (PMID:23446638; PMID:34878965).
Gene–Disease AssociationLimitedSingle CS/CSL proband with germline KLLN variant; no segregation; supportive epigenetic and functional data Functional EvidenceModerateKLLN promoter hypermethylation in CS cohorts and in vitro studies show loss-of-function disrupts G2/M and G1 checkpoints (PMID:23446638; PMID:25669429; PMID:34878965) |