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RESEARCH MCEWEN STEM CELL INSTITUTE

Stem Cells

to Liver

Cells

Scientists create liver cells with

vital blood-clotting protein

Researchers have found a way to generate the
cells that may hold the key to new treatments
for hemophilia A, a genetic disorder in which the
blood does not clot normally.

The cells that were produced are known as liver
sinusoidal endothelial cells and are found in
specific regions of the liver. The research team
made the cells from human pluripotent stem cells,
which can give rise to most cells in the human
body.
Dr. Keller concludes, “This finding moves us
“Individuals with hemophilia A lack a clotting closer to understanding the role of these cells in
factor known as Factor VIII, which is produced liver function and to developing new therapies to
by these endothelial cells. We found a way to make treat a number of conditions, such as hemophilia A
large numbers of the precursors of these cells in and chronic liver diseases.”
the laboratory, and we were able to validate that
they can make the missing clotting factor,” says Dr. Gage BK, et al. Cell Stem Cell. 2020 July 7. doi:10.1016/j.
Blair Gage, a postdoctoral fellow in Dr. Gordon stem.2020.06.007. Supported by the University of Toronto’s
Keller’s laboratory and first author of the study. Medicine by Design Initiative, which receives funding from
the Canada First Research Excellence Fund; the Canadian
Institutes of Health Research; the McEwen Stem Cell Institute;
The team first used human pluripotent stem cells and the UHN Foundation.
to generate venous angioblasts—precursors to
the endothelial cells that line the veins. Next, by (Top left) Immunofluorescence image showing human
transplanting these precursors into experimental pluripotent stem cell-derived liver endothelial cells. The cells
models, the researchers were able to coax them to are stained red and green with white nuclei. A blood factor
related to Factor VIII, known as Von Willebrand Factor,
mature into sinusoidal endothelial cells that were is stained in blue. (Top right) Dr. Blair Gage. (Right) Dr.
capable of producing Factor VIII. Alexander Mikryukov.

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