Article

Development of novel therapies for Marfan syndrome using a human iPSC-disease model

2019-01-01

Abstract excerpt

Marfan syndrome (MFS) is a connective tissue disorder caused by mutations in fibrillin-1, a matrix component encoded by the gene FBN1, with pleiotropic manifestations including severe cardiovascular complications, such as aortic aneurysms and dissection. Current treatments focus on surgically removing the aneurysm or on minimising aortic wall stress by controlling haemodynamics, but neither of these strategies tac...

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Literature Corpus work: 9a560654-593c-5c58-8834-197f30aa556e
DOI: 10.17863/cam.38053

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