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Retinoblastoma 1 (RB1) is infrequently implicated in melanoma pathogenesis. A single patient with a 9-year history of chronic lymphocytic leukemia and a cutaneous collision tumor harbored a constitutional t(12;17)(p13;p13) disrupting RB1, suggesting a possible germline predisposition to melanoma ([PMID:29371889]). In a clonal architecture study of 124 metastatic melanomas, truncating RB1 mutations were identified in multiple patients, indicating recurrent somatic loss of function in melanoma genomes ([PMID:25393105]). Functional assays demonstrate that RB1 represses cell cycle progression and transcriptional activation of oncogenic promoters such as IL6, consistent with a tumor suppressor mechanism in diverse cell types ([PMID:1652755]).
Integration & Clinical Utility: The current evidence supports a Limited clinical validity for RB1 in melanoma, based on one germline case and several somatic studies without segregation data. Genetic evidence is Limited: somatic truncating mutations are observed but familial segregation is unproven. Functional evidence is Moderate: RB1’s role in cell-cycle control and promoter repression is well established. Further family-based investigations and segregation analyses are required to define RB1 as a melanoma predisposition gene. Key Take-home: RB1 functions as a somatic tumor suppressor in melanoma with moderate mechanistic support but limited germline evidence for clinical genetic testing.
Gene–Disease AssociationLimitedOne germline case of constitutional RB1 disruption in a CLL/MM collision tumour (PMID:29371889) and recurrent somatic truncating mutations in metastatic melanomas (PMID:25393105) Genetic EvidenceLimitedSomatic RB1 truncating mutations observed in multiple melanoma genomes without germline segregation data Functional EvidenceModerateRB1 represses cell-cycle genes and oncogenic promoters such as IL6, supporting a tumor suppressor mechanism (PMID:1652755) |