Patient stems cells, genetic tools, and heart-on-chip technology provide new insight into possible treatment strategies for a genetic heart condition, according to a new study published in Nature Medicine.
Stem cells can be derived from patient skin biopsies and cultured into most cell types, including cardiac cells. Using stem cells from patients with Barth syndrome, researchers were able to show that a genetic mutation was the cause of functional abnormalities of the patients’ cardiac cells.
This non-animal technique advances both knowledge of Barth syndrome and stem cell-based in vitro technology for studying heart conditions.
- Wang G, McCain ML, Yang L, et. al. Modeling the mitochondrial cardiomyopathy of Barth syndrome with induced pluripotent stem cell and heart-on-chip technologies. Nat Med. Published ahead of print May 11, 2014.