Mingxia Gu, MD, PhD

Almost all children born with structural heart/lung disease have only one therapy option — surgery. My group works to give these families more options by developing new therapies for early intervention, before the baby is even born. As an instructor in Pediatric Cardiology since 2016, my overarching research goal is to develop a novel therapy to boost the regeneration of the heart, lung and vasculature in patients with a congenital cardiac and/or pulmonary defect.

Our group conducts leading interdisciplinary research using patient-specific induced pluripotent stem cells (iPSCs), derived cardiovascular cells, vessel and lung organoids, animal models, and single cell RNA/ATAC sequencing for disease modeling and high-throughput drug screening. With an established translational research study set-up, we have access to a large patient cohort that provides human tissue for the generation of iPSCs, organoids and single-cell transcriptomic profiling.

We are also interested in exploring the role of vascular insufficiency in causing cardiopulmonary diseases such as congenital heart defects, pulmonary atresia and pulmonary arterial hypertension. We hope to understand the genetic and epigenetic underpinnings of the variation in disease phenotypes and drug response in a personalized manner. Our team works closely with developmental and stem cell biologists, clinicians, bioengineers, translational scientists and entrepreneurs with the common goal of accelerating discovery and facilitating bench-to-bedside translational science.

I decided to pursue research as a medical student, when I realized that there are too many unanswered questions in our field. For example, why do patients have different clinical expressivity? Why do patients respond to drugs differently? Why does part of the heart fail to grow during development? And if the cardiomyocytes stop proliferating soon after birth, can we find a way to reset the clock and help the heart regenerate?

The drive to answer these questions led me to complete my PhD training in stem cells and regenerative medicine, and I have been highly engaged in clinically driven, basic biomedical research since then. I’m also enthusiastic about the possibility of bringing our benchtop discoveries to clinical trials.

My team and I have made several notable discoveries. To understand more about the application of stem cells, we evaluated the therapeutic efficacy of iPSC-derived vascular and cardiac cells using a variety of animal models (mouse, rat, porcine) with cardiomyopathies and vasculopathies. We further elucidated the molecular mechanism using single-cell transcriptomic profiling (Gu et al., Circulation Research, 2012; Gu et al., European Heart Journal, 2015).

To understand patient-specific phenotypes, we utilized iPSC-derived endothelial cells and integrative omics to identify the modifiers that protected unaffected BMPR2 mutation carriers from developing pulmonary arterial hypertension (PAH) (Gu et al., Cell Stem Cell). Using a similar stem cell platform and single cell transcriptomic analysis, we revealed the endocardial and endothelial abnormalities that lead to the valvular and ventricular hypoplasia in single ventricle congenital heart disease.

I’m proud to have received a number of honors and awards during my career. I was a finalist for the 2019 Stanford Cardiovascular Institute Travel Award, and I received the 8th Annual Stanford Art of Science Competition in 2019. I have also been awarded:

• ISSCR 2019 Abstract Merit Award
• ISSCR 2019 Annual Conference Travel Award
• 2019 Eureka International Certificate Program in Translational Medicine Awards
• 2018 Stanford Maternal and Child Health Research Institute Instructor K Award
• 2018 Stanford Cardiovascular Institute Seed Grant Best Oral Abstract
• 2017 Stanford Annual Heart Center Research Day
• 2017 Stanford Cardiovascular Institute Seed Grant
• 2017-2022 Stanford Cardiovascular Institute Travel Award
• 2017 NIH/NHLBI K99/R00 Pathway to Independence Career Award
• 2016 Stanford Cardiovascular Institute Best Manuscript Award
• 2016 American Heart Association Junior Investigator Travel Award
• 2016 Outstanding speaker, Seventh Annual Pediatrics Research Retreat
• 2014-2016 American Heart Association Postdoctoral Research Fellowship
• 2013 American Heart Association Best Manuscript Award

In addition to my research, I enjoy teaching and mentoring. My diverse experience includes mentoring high school students, undergraduates, postdoc fellows and clinical fellows for their research in the lab. I also enjoy traveling nationally and internationally to present my work, build up new collaborations and learn about different cultures all over the world.

I am very excited to continue mentoring and advising young scientists. As a woman in science, it is one of my goals to promote diversity in research by reaching out to underrepresented groups and developing opportunities in which they can thrive.