Stem Cells: Regenerative Medicine
Stem Cells 3.0: The Holy Grail of Regenerative Medicine
Imagine being able to craft a new cardiac cell to replace those damaged by a heart attack, or new retinal cells to restore sight. This is the vision of researcher and cardiologist Young-sup Yoon, MD, PhD, associate professor in cardiology, who is doing groundbreaking research in stem cell technology.
"The methods of generating stem cells that currently exist using gene transfer are inefficient or expensive and take repeated treatment," says Yoon. "We believe our method is safer and more cost effective."
Stem cell medicine has been identified as a treatment for conditions including heart disease, diabetes, spinal cord injury, stroke, and cancer. The use of embryonic stem cells, however, has been stymied by political and ethical issues, and difficulties in obtaining embryonic tissues and isolating relatively rare cell types have limited the large-scale production of populations of pure stem cells.
Also, the use of viral vectors and foreign genes to make induced pluripotent stem cells sometimes have unintended consequences in the individuals receiving the new cells, including the chance of causing cancer. "The best case scenario is not to use genes at all," Yoon says.
The development of chemically induced pluripotent stem cells provides a patient with a copious, immune-matched supply of pluripotent cells, which can differentiate into whatever cell types are needed. Yoon and collaborators at Emory and in Rome use a "unique cocktail of small molecules" that alter gene expression and generate pluripotent cells.
"This is the Holy Grail for regenerative medicine in that it allows you to take cells from the patient and modify them without the use of viral vectors or exogenous DNA, easy to harvest, much easier to grow, and can be used both for regenerative medicine and research tools," says Just Burns, OTT licensing associate. The OTT helped Yoon and collaborators to file a PCT patent application on the molecules themselves, the use of the molecules, and the system for reprogramming cells. Additionally, his work with chemically induced pluripotent stem cells received the Innovation of the Year award for 2013 at Emory’s Office of Technology Transfer annual banquet.
Yoon's research is part of a National Institutes of Health grant to study stem cells.
"At this point, it's a small homerun," Yoon says. "It's a matter of getting the resources to scale up. As a physician scientist, I know there are a lot of heart patients waiting to die unless they get a matched donor. This technology is really promising for patients who right now don't have other options."
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Figure 1: CiPSCs were able to differentiate into all three embryonic germ layers by forming embryoid bodies (EBs) in vitro. EB formation of CiPSC was induced by suspension culture for 9 days and whole EBs were immunostained with antibodies to show the expression markers for ectoderm (βIII-tubulin, red, left panel), mesoderm (smooth muscle actin, green, middle panel), and endoderm (alpha-fetoprotein, green, right panel). Nuclei of cells were stained with DAPI (blue).
Figure 2: CiPSCs were able to differentiate into all three embryonic germ layers by forming embryoid bodies (EBs) in vitro. EB formation of CiPSC was induced by suspension culture for 9 days and whole EBs were immunostained with antibodies to show the expression markers for ectoderm (βIII-tubulin, red, top panel), mesoderm (smooth muscle actin, green, middle panel), and endoderm (alpha-fetoprotein, green, bottom panel). Left panels show cell nuclei stained with DAPI (blue) and right panels are merged images for nuclei and immunostained cells in whole EBs.