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Drug-based therapies for vascular disease in Marfan syndrome: from mouse models to human patients.

Abstract
Marfan 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.
AuthorsJason R Cook, Harikiran Nistala, Francesco Ramirez
JournalThe Mount Sinai journal of medicine, New York (Mt Sinai J Med) 2010 Jul-Aug Vol. 77 Issue 4 Pg. 366-73 ISSN: 1931-7581 [Electronic] United States
PMID20687182 (Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't, Review)
Copyright2010 Mount Sinai School of Medicine.
Chemical References
  • Angiotensin II Type 1 Receptor Blockers
  • Antihypertensive Agents
  • Fibrillins
  • Microfilament Proteins
  • Transforming Growth Factor beta
  • Losartan
Topics
  • Angiotensin II Type 1 Receptor Blockers (therapeutic use)
  • Animals
  • Antihypertensive Agents (pharmacology, therapeutic use)
  • Aorta (pathology)
  • Aortic Aneurysm, Thoracic (drug therapy, genetics, pathology)
  • Connective Tissue (drug effects, pathology)
  • Disease Models, Animal
  • Fibrillins
  • Humans
  • Losartan (pharmacology, therapeutic use)
  • Marfan Syndrome (drug therapy, genetics, pathology)
  • Mice
  • Microfilament Proteins (drug effects)
  • Renin-Angiotensin System (drug effects)
  • Signal Transduction (drug effects)
  • Transforming Growth Factor beta (drug effects)

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