Regenerative Medicine: Making Blood Stem Cells in the Lab

Endothelial cells becoming hematopoietic stem cells

Caption: Arrow in first panel points to an endothelial cell induced to become hematopoietic stem cell (HSC). Second and third panels show the expansion of HSCs over time.
Credit: Raphael Lis, Weill Cornell Medicine, New York, NY

Bone marrow transplants offer a way to cure leukemia, sickle cell disease, and a variety of other life-threatening blood disorders.There are two major problems, however: One is many patients don’t have a well-matched donor to provide the marrow needed to reconstitute their blood with healthy cells. Another is even with a well-matched donor, rejection or graft versus host disease can occur, and lifelong immunosuppression may be needed.

A much more powerful option would be to develop a means for every patient to serve as their own bone marrow donor. To address this challenge, researchers have been trying to develop reliable, lab-based methods for making the vital, blood-producing component of bone marrow: hematopoietic stem cells (HSCs).

Two new studies by NIH-funded research teams bring us closer to achieving this feat. In the first study, researchers developed a biochemical “recipe” to produce HSC-like cells from human induced pluripotent stem cells (iPSCs), which were derived from mature skin cells. In the second, researchers employed another approach to convert mature mouse endothelial cells, which line the inside of blood vessels, directly into self-renewing HSCs. When these HSCs were transplanted into mice, they fully reconstituted the animals’ blood systems with healthy red and white blood cells.

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Bright Lights, Cool Science

Photo of the developing brain of a live zebrafish

Photo Credit: Jennifer Peters, Ph.D., and Michael Taylor, Ph.D. at the Cell and Tissue Imaging Center at St. Jude Children’s Research Hospital.

This may look like a tangle of holiday lights, but it’s actually a peek inside  the developing brain of a live zebrafish. NIH-supported researchers created this award-winning picture by labeling the brain’s endothelial cells with fluorescent proteins and then using a confocal microscope to snap 3-D images as the cells assembled into the blood-brain barrier. So why are these guys taking photos of fish brains? They want to gain a better understanding of how the blood-brain barrier develops. And that’s  important because a major hurdle in the treatment of many brain disorders is figuring out better ways to move drugs and other therapies from the bloodstream and into the brain.