19 February 2018 by Jun Yang

Stem cell based drug screening for pulmonary arterial hypertension


Deficiency of bone morphogenetic protein type II receptor (BMPRII) signaling has been implicated in the pathology of pulmonary arterial hypertension (PAH). Targeting BMPRII signaling with small molecular compound to selectively improve the function of the vascular endothelium could be a desirable therapeutic intervention for PAH.


To apply stem cell technology in drug discovery and identify BMPRII signalling regulators for the treatment of PAH.

Methods and Results

Because BMP2 has been reported to enhance endothelial BMPRII signaling, we screened 8 160 small molecular compounds for their potential as BMP2 upregulators. And we performed an assay on the BMPRII downstream gene Inhibitor of DNA binding 1 (Id1) using a dual reporter driven specifically in endothelial cells differentiated from human embryonic stem cells (hESCs-ECs). The identified leading compound restored BMPRII downstream signalling in human pulmonary arterial endothelium cells (hPAECs) and endothelial cells differentiated from PAH patients derived induced pluripotent stem cell (iPSCs-ECs).


Using stem cell technology, we gained a clearer understanding of the genetic cause of PAH and provided a potential drug screening method for cardiopulmonary disease.

Key Contributors

Yanjiang Xing1*, Shuang Zhao1*, Qingxia Wei1*, Fang Zhou1, Shiqiang Gong1, Xin Zhao1, Rafia AI-Lamki, Daniel Ortmann, Mingxia Du1, Roger Pedersen, Guangdong Shang, Shuyi Si, Nicholas W Morrell, Jun Yang1 *These authors contribute equally to this paper. 1 State Key Laboratory of Medical Molecular Biology, Department of Cell Biology, Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences and School of Basic Medicine, Peking Union Medical College, Beijing 100005, China.

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