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Review
Drug-based therapies for vascular disease in Marfan syndrome: from mouse models to human patients.
- Jason R Cook, Harikiran Nistala, and Francesco Ramirez.
- Mount Sinai School of Medicine, New York, NY, USA.
- Mt. Sinai J. Med. 2010 Jul 1;77(4):366-73.
AbstractMarfan syndrome is a congenital disorder of the connective tissue with a long history of clinical and basic science breakthroughs that have forged our understanding of vascular-disease pathogenesis. The biomedical importance of Marfan syndrome was recently underscored by the discovery that the underlying genetic lesion impairs both tissue integrity and transforming growth factor-beta regulation of cell behavior. This discovery has led to the successful implementation of the first pharmacological intervention in a connective-tissue disorder otherwise incurable by either gene-based or stem cell-based therapeutic strategies. More generally, information gathered from the study of Marfan syndrome pathogenesis has the potential to improve the clinical management of common acquired disorders of connective-tissue degeneration.2010 Mount Sinai School of Medicine.
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