Stem cells used to make “heart disease on a chip”
Harvard scientists have for the first time grown in a lab human heart tissue carrying heart disease. Experts are calling this a big step forward in the evolution of personalized medicine.
The study involved stem cell and ‘organ-on-a-chip’ technologies. Skin cells were taken from two people with Barth syndrome—a rare heart disorder caused by a mutation of a gene called Tafazzin, or TAZ. The researchers then manipulated the skin cells to become stem cells carrying the same genetic mutation. The stem cells were then grown in the lab to become diseased tissue.
The second method involved something called genome editing, in which researchers mutated TAZ in normal cells. By delivering the TAZ gene product to the diseased tissue, the researchers were essentially able to correct the engineered defect and were thereby able to create the first tissue-based model of correction of a genetic heart disease.
The study is significant in that researchers were able to demonstrate the ability to restore function in diseased human heart tissue. They now hope to reproduce their findings in animal models of Barth syndrome to see if there are implications for humans with cardiovascular disease.