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Letter

Nature Genetics 33, 407–411 (1 March 2003) | doi:10.1038/ng1116

Dysregulation of TGF-|[beta]| activation contributes to pathogenesis in Marfan syndrome

Enid R. Neptune , Pamela A. Frischmeyer , Dan E. Arking , Loretha Myers , Tracie E. Bunton , Barbara Gayraud , Francesco Ramirez , Lynn Y. Sakai & Harry C. Dietz

Marfan syndrome is an autosomal dominant disorder of connective tissue caused by mutations in fibrillin-1 (encoded by FBN1 in humans and Fbn1 in mice), a matrix component of extracellular microfibrils. A distinct subgroup of individuals with Marfan syndrome have distal airspace enlargement, historically described as emphysema, which frequently results in spontaneous lung rupture (pneumothorax; refs.