Article

Tacrolimus rescues endothelial ALK1 loss-of-function signaling and improves HHT vascular pathology

2017-05-13

Abstract excerpt

Hereditary hemorrhagic telangiectasia (HHT) is a genetic vascular disorder arising from endothelial cell (EC) proliferation and hypervascularization, for which no cure exists. Because HHT is caused by loss-of-function mutations in BMP9-ALK1-Smad1/5/8 signaling, interventions aimed at activating this pathway are of therapeutic value. By screening FDA-approved drug libraries, we identified tacrolimus (FK-506) as a p...

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Literature Corpus work: a8b14e6f-a914-5911-8aca-934bc161a61a
DOI: 10.1101/137737

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