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

Jason R. Cook, Harikiran Nistala, Francesco Ramirez

Research output: Contribution to journalReview articlepeer-review

10 Scopus citations


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-β 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.

Original languageEnglish (US)
Pages (from-to)366-373
Number of pages8
JournalMount Sinai Journal of Medicine
Issue number4
StatePublished - Jul 2010
Externally publishedYes


  • Aortic aneurysm
  • Connective tissue
  • Fibrillin
  • Losartan
  • Marfan syndrome
  • Pharmacology
  • TGFβ
  • Vascular therapy

ASJC Scopus subject areas

  • General Medicine


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