Using a recently developed technique for turning skin
cells into stem cells, scientists have cured mice of
sickle cell anemia — the first direct proof that the
easily obtained cells can reverse an inherited,
potentially fatal disease.
Researchers said the work, published in yesterday’s
online edition of the journal Science, points to a
promising future for the novel cells. Known as iPS cells,
they have been touted by President Bush and some
scientists as a possible substitute for embryonic stem
cells, which have been mired for years in political
controversy.
But researchers also cautioned that aspects of the new
approach will have to be changed before it can be tried in
human patients. Most important, the technique depends on
the use of gene-altered viruses that have the potential to
trigger tumor growth.
“The big issue is how to replace these viruses,”
said Rudolf Jaenisch of the Whitehead Institute for
Biomedical Research in Cambridge, Mass., who led the new
work with co-worker Jacob Hanna and Tim M. Townes of the
University of Alabama Schools of Medicine and Dentistry in
Birmingham.
“Induced pluripotent stem,” or iPS, cells, are
virtually identical to embryonic stem cells. They can
morph into all of the more than 200 cell types in the body
but are derived from skin, not from embryos. Mouse iPS
cells were first derived earlier this year, and scientists
reported last month to great fanfare that they had created
similar cells from human skin.
The new experiment started with the removal of a few
skin cells from the tail tips of mice sick with sickle
cell anemia, which can cause painful circulatory problems,
kidney failure and strokes.
The researchers converted those skin cells into iPS
cells by infecting them with viruses engineered to change
the cells’ gene activity so they would resemble
embryonic cells.
Using DNA splicing techniques in those cells, the
researchers then snipped out the small mutated stretches
of DNA that cause sickle cell disease and filled those
gaps with bits of DNA bearing the proper genetic code.
Next, the researchers treated the corrected iPS cells
with another kind of virus — this time one designed to
induce a genetic change that encouraged the cells to
mature into bone marrow cells.