Stem Cell Program

Stem Cell Program | Heart Disease

Congenital heart disease is the most frequent type of severe congenital malformation, and the leading cause of morbidity and mortality in infancy. The Boston Children’s Hospital Benderson Family Heart Center is a leading national and international center for the treatment of congenital heart disease. The Basic and Translational Research Laboratories of the Department of Cardiology study the mechanisms underlying congenital and acquired heart disease, and pioneer new treatment approaches based on the discoveries made.

William Pu, MD, director Department of Cardiology, Basic and Translational Research, has integrated stem cell biology with bioengineering, molecular biology, and animal models to develop innovative models of inherited heart disease. Using these models, Pu’s lab is developing novel therapies for inherited heart disease, including gene therapy for inherited disease of heart muscle and heart rhythm.

Da-Zhi Wang, PhD, studies RNA biology in heart and muscle development, disease, and regeneration. Among the model systems used in the Wang lab are human induced pluripotent stem cell-derived cardiomyocytes. The Wang lab discovered a protein, CIP, that is important for muscle cell nuclear positioning and that modulates the severity of muscle disease caused by dystrophin mutation. The lab has also identified a set of long non-coding RNAs that are implicated in cardiac disease pathogenesis.

The integration of form and function in the heart makes bioengineering integral to developing tissue models of human heart disease. The Disease Biophysics Group of Kevin Kit Parker, PhD, professor of Engineering and Applied Sciences at Harvard University, has established a branch at Boston Children’s. In collaboration with Pu and other investigators in the Basic and Translational Research Labs, Parker is developing novel heart disease models. Pu and Parker co-direct the Center for Accelerating Therapeutic Discovery (CAT-D), funded by the Massachusetts Life Sciences Center, to use microphysiological systems to accelerate translation of new therapies to patients.

Caroline Burns, PhD, associate professor of pediatrics, and Geoff Burns, PhD, assistant professor of pediatrics, use zebrafish models to study congenital heart disease. Recent work has identified cardiac progenitor populations that contribute to the formation of the heart and great vessels, and identified genetic pathways linked to hypoplastic left heart syndrome, among the most severe forms of congenital heart disease. The Burns lab also studies heart regeneration, and recently showed that cardiomyocyte ploidy is closely linked to regenerative potential.

Vassilios Bezzerides, MD, PhD, assistant professor of Pediatrics, is an electrophysiologist uses stem cell and mouse models to study the pathogenesis of inherited rhythm disorders. Using these model systems, he has developed a novel gene therapy approach for CPVT, a malignant inherited arrhythmia, and is working to translate this discovery into a first-in-man clinical trial.

The commitment and compassion with which we care for all children and families is matched only by the pioneering spirit of discovery and innovation that drives us to think differently, to find answers, and to build a better tomorrow for children everywhere.

Kevin B. Churchwell, President and CEO

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