How Patient-Derived Stem Cells are Changing the Trajectory of Congenital Heart Disease Research
Small blood samples –– and the patient-specific, induced pluripotent stem cells (iPSCs) they enable –– turn into valuable research material for understanding congenital cardiovascular disorders, especially when united with modern genome sequencing and editing, animal models and three dimensional tissue growth technologies. Written by Katie Brind’Amour, PhD
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atient-derived induced pluripotent stem cells (iPSCs) have a significant downside for studying most forms of adult heart disease: they resemble fetal heart cells in form and function. But this limitation, which keeps the cells from being easily applicable for the study of many adult conditions, is actually ideal for studying congenital heart problems. Congenital heart defects or disease (CHD) affect about 1% of all live births in the United States and rank among
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PediatricsNationwide.org | Fall/Winter 2021
the leading causes of birth defect-related deaths. These defects can range from mild to very severe, and many require ongoing monitoring and multiple surgeries. While operations and diagnostics have improved survival for many CHD patients — extending life into adulthood for many children who previously would have died in infancy or early childhood — much remains to be done to advance treatment options and maintenance therapeutics.
