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| Stem cell
research and EB – what does it all mean?
Everyone at DebRA knows that
finding a cure for EB is the ultimate aim of all research. Everyone also knows that this
isn’t easy, but it’s true to say that considerable progress is being made.
Although definitive “cures” may not be on the horizon yet, new strategies to
develop better treatments are emerging, and DebRA currently funds several international
projects that are focused on developing new therapies for people with EB.
These new treatments involve
gene, protein and cell therapies, all of which are designed to restore or correct the
primary faults that cause EB.
Of course, researchers, and everyone whose lives are directly or indirectly affected by
EB, are always on the look out for better and more effective ways to speed up progress. |
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With that in mind, many people will have been aware of the recent media interest in
“embryonic stem cells” and will be wondering whether or not this might be
relevant to EB research?
At present, the gene therapy research that DebRA funds does involve work on stem cells,
but this mainly involves skin stem cells – small groups of cells (keratinocytes) from
within the epidermis or hair follicles that are capable of surviving and making
generations of other skin cells that can go on to make new skin.
By targeting gene therapy to these stem cells, it is hoped that the benefits will last for
a long time rather than just a week or two if any non-stem cell keratinocyte receives the
new gene therapy. Other EB researchers are also studying stem cells in the dermis and in
the bone marrow but, in essence, these strategies all involve attempts to manipulate
“adult” stem cells.
The other type of stem cell, however, that researchers are also now focusing on, are the
embryonic stem cells.
These human cells, which were first identified in 1998, represent part of an early embryo,
known as the inner cell mass.
Their importance to modern biology and medicine derives from two unique characteristics
that distinguish them from all other organ-specific stem cells identified to date. First,
they can be maintained and expanded as pure populations of undifferentiated cells for
extended periods of time, possibly indefinitely, in culture. Secondly, they are
pluripotent, which means that they possess the capacity to generate every cell type in the
body.
This implies that, given the right laboratory conditions, it will be possible to generate
new tissues, including skin. At present, there is considerable research activity looking
at the various chemicals and signals that can make the embryonic stem cells develop in
different ways, such as making new blood cells, muscle, heart or skin.
Much of this work is currently confined to mouse embryonic skin cells, but the principles
and lessons learnt will be directly relevant to human cells. Some of the emerging pathways
of making different organs from embryonic stem cells are shown in the figure below (ES =
embryonic stem cells)
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So far,
generating blood cells and heart muscle has been studied in some detail, but basic
research that explores how to make skin is also being tested.
Research on embryonic stem cells,
therefore, may have enormous future implications for “cell replacement”
therapies relevant to EB. For now, the topic is a research one, and one that is subject to
stringent regulations and ethical debate.
Everyone at DebRA, however, will
be keeping up-to-date with the latest information on both adult and embryonic stem cells,
especially with how this research technology may help people living with EB.
31 May 2005.
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