Researchers use modified RNA transfection to correct genetic dysfunction in heart stem cells derived from Barth syndrome patients. The series of images show how inserting modified RNA into diseased cells causes the cells to produce functioning versions of the TAZ protein (first image: in green) that correctly localize in the mitochondria (second image: in red). When the images are merged to demonstrate this localization, green overlaps with red, giving the third image a yellow color. (Credit: Gang Wang and William Pu/Boston Children’s Hospital)
Harvard scientists have merged stem cell and ‘organ-on-a-chip’ technologies to grow, for the first time, functioning human heart tissue carrying an inherited cardiovascular disease. The research appears to be a big step forward for personalized medicine, as it is working proof that a chunk of tissue containing a patient’s specific genetic disorder can be replicated in the laboratory.
The work, published in Nature Medicine, is the result of a collaborative effort bringing together scientists from the Harvard Stem Cell Institute, the Wyss Institute for Biologically Inspired Engineering, Boston Children’s Hospital, the Harvard School of Engineering and Applied Sciences, and Harvard Medical School. It combines the ‘organs-on-chips’ expertise of Kevin Kit Parker, PhD, and stem cell and clinical insights by William Pu, MD.