T<scp>ranslational</scp> A<scp>dvances</scp> <scp>in</scp> <scp>the</scp> F<scp>ield</scp> <scp>of</scp> P<scp>ulmonary</scp> H<scp>ypertension</scp> Molecular Medicine of Pulmonary Arterial Hypertension. From Population Genetics to Precision Medicine and Gene Editing

The majority of the remarkable progress in understanding the molecular pathogenesis of Pulmonary Arterial Hypertension has been achieved within the past 2 decades. But despite important advances in treatment, PAH remains a disease with poor survival and no curative therapy. A rapidly growing molecular knowledge base provides hope for the possibility to develop specific therapies targeted at the origins of disease, as well as factors which modify therapeutic response. In this synopsis, we summarize highlights of the genetic basis of PAH, and the evolving fields of prediction of disease risk, its course, and response to specific treatments in individual PAH patients. Further, we summarize the major advances of gene editing approaches which promise a new era of intervention, which is now in its infancy but likely to advance rapidly.