Document Detail


Drug-based therapies for vascular disease in Marfan syndrome: from mouse models to human patients.
MedLine Citation:
PMID:  20687182     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
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.
Authors:
Jason R Cook; Harikiran Nistala; Francesco Ramirez
Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Review    
Journal Detail:
Title:  The Mount Sinai journal of medicine, New York     Volume:  77     ISSN:  1931-7581     ISO Abbreviation:  Mt. Sinai J. Med.     Publication Date:    2010 Jul-Aug
Date Detail:
Created Date:  2010-08-05     Completed Date:  2011-06-02     Revised Date:  2011-08-01    
Medline Journal Info:
Nlm Unique ID:  0241032     Medline TA:  Mt Sinai J Med     Country:  United States    
Other Details:
Languages:  eng     Pagination:  366-73     Citation Subset:  IM    
Copyright Information:
2010 Mount Sinai School of Medicine.
Affiliation:
Mount Sinai School of Medicine, New York, NY, USA.
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MeSH Terms
Descriptor/Qualifier:
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
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
Grant Support
ID/Acronym/Agency:
AR-049 698/AR/NIAMS NIH HHS; P01 AR049698-050001/AR/NIAMS NIH HHS; T32GM062754/GM/NIGMS NIH HHS
Chemical
Reg. No./Substance:
0/Angiotensin II Type 1 Receptor Blockers; 0/Antihypertensive Agents; 0/Microfilament Proteins; 0/Transforming Growth Factor beta; 0/fibrillin; 114798-26-4/Losartan
Comments/Corrections

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