Sickle Cell Anemia: Stem Cell Fix?

Stem cells may treat sickle cell anemia, new research

Sickle cell anemia is the most common inherited blood disorder in
the U.S. It causes red blood cells to become misshapen. Those cells tend to
pile up in blood vessels, making it hard for the blood to carry oxygen around
the body.

Scientists today announced that they have used stem cells to curb sickle
cell anemia
symptoms in mice.

The stem cells "rescued" the mice from sickle cell anemia symptoms , write the researchers, who warn that more work
is needed to prevent possible dangerous side effects from stem cell

Here's a quick look at how the study worked.

First, the researchers took skin cells from mice that had sickle cell
anemia. Next, they used retroviruses to carry genes -- including a cancer gene -- into the skin
cells. The inserted genes caused the skin cells to act like embryonic stem

Then, the scientists coaxed the fledgling stem cells into precursors of
cells that make red blood cells. The researchers fixed the sickle cell gene
glitch in those cells and removed the cancer gene.

When the resulting cells were injected into the mice, the mice's sickle cell
symptoms eased so much that the sickle cell mice resembled mice without sickle
cell anemia.

But using retroviruses and cancer genes to transform skin cells into
embryonic-like stem cells may have long-term risks, and it will take more work
to learn how to limit those risks, write the researchers.

They included Jacob Hanna, MD, PhD, of the Whitehead Institute for
Biomedical Research in Cambridge, Mass.

The study appears in today's advance online edition of Science.

By Miranda Hitti
Reviewed by Brunilda Nazario
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