Bridging the Translational Gap: A hiPSC Model of Marfan Syndrome for Drug Discovery
Sanjay Sinha,
,
University of Cambridge
Marfan syndrome is a connective tissue disorder, caused by mutations in
FBN1, with pleiotropic manifestations including aortic aneurysms and
dissection. Although the angiotensin II receptor blocker, losartan, was
effective in treating the disease in mouse models, clinical trials have
been much less successful. In this presentation, I will discuss the use
of a hiPSC model of aortic disease in Marfan syndrome to understand the
molecular basis of this disease and why the mouse studies may not have
translated well to patients. I will also describe how this complex in
vitro system can be used for novel drug screening for this devastating
clinical disorder.
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