A novel TSC1 variant associated with tuberous sclerosis and sacrococcygeal teratoma.
Saba AhmadLuis ManonGifty BhatJerry MachadoAlice ZalanNikolas Mata-MachadoSteven GarzonAkira YoshiiPublished in: Human genome variation (2020)
Tuberous sclerosis complex (TSC) is an autosomal dominant disease associated with tumors and malformed tissues in the brain and other vital organs. We report a novel de novo frameshift variant of the TSC1 gene (c.434dup;p. Ser146Valfs*8) in a child with TSC who initially presented with a sacral teratoma. This previously unreported association between TSC and teratoma has broad implications for the pathophysiology of embryonic tumors and mechanisms underlying cellular differentiation.