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Rescuing the BMPR2 Pathway, Where Can We Intervene?

Marlene Rabinovitch


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Conference: 2012 International PHA Conference and Scientific Sessions

Release Date: 01.15.2013

Presentation Type: Scientific Sessions

Learning Objectives:

  • Discuss the role of endothelial and smooth muscle cell survival and proliferation in the pathology of pulmonary arterial hypertension
  • Evaluate the implications of changes to the BMPR2 pathway in pulmonary artery endothelial cell and smooth muscle function and survival
  • Identify potential therapeutic targets within and around the BMPR2 signaling cascade

Mutations causing dysfunction of the BMPR2 gene is associated with idiopathic forms of pulmonary hypertension that affect families. Even in the absence of a mutation, many studies have shown reduced function of this gene because of other factors. We and other groups have worked hard to understand what this gene does to protect thecells of blood vessels against damage that leads to pulmonary hypertension. Our goal is to use this information toreverse the factors causing dysfunction of BMPR2. One option is to mimic the effects of BMPR2 by supplying thecell with the same things that BMPR2 supplies. We now have discovered three different treatment strategies tosupply the cells with factors that are normally made in response to activation of the BMPR2 gene. The next goal isto figure out a way to better activate the dysfunctional receptor. We have found a known pharmaceutical agent nowin the clinic for other purposes that appear to do just that.