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There are many ways in which human Stem Cells can be used in basic
research and in clinical research. However, there are many technical
hurdles between the promise of stem cells and the realization of these
uses, which will only be overcome by continued intensive stem cell
research.
Studies of Human Embryonic Stem Cells may yield information about the
complex events that occur during human development. A primary goal of
this work is to identify how Undifferentiated stem cells become
differentiated. Scientists know that turning genes on and off is
central to this process. Some of the most serious medical conditions,
such as cancer and birth defects, are due to abnormal Cell Division and
Differentiation. A better understanding of the genetic and molecular
controls of these processes may yield information about how such
diseases arise and suggest new strategies for therapy. A significant
hurdle to this use and most uses of stem cells is that scientists do
not yet fully understand the Signals that turn specific genes on and
off to influence the differentiation of the stem cell.
Human stem cells could also be used to test new drugs. For example, new
medications could be tested for safety on differentiated cells
generated from human Pluripotent cell lines. Other kinds of cell lines
are already used in this way. Cancer cell lines, for example, are used
to screen potential anti-tumor drugs. But, the availability of
pluripotent stem cells would allow drug testing in a wider range of
cell types. However, to screen drugs effectively, the conditions must
be identical when comparing different drugs. Therefore, scientists will
have to be able to precisely control the differentiation of stem cells
into the specific cell type on which drugs will be tested. Current
knowledge of the signals controlling differentiation fall well short of
being able to mimic these conditions precisely to consistently have
identical differentiated cells for each drug being tested.
Perhaps the most important potential application of human stem cells is
the generation of cells and tissues that could be used for cell-based
therapies. Today, donated organs and tissues are often used to replace
ailing or destroyed tissue, but the need for transplantable tissues and
organs far outweighs the available supply. Stem cells, directed to
differentiate into specific cell types, offer the possibility of a
renewable source of replacement cells and tissues to treat diseases
including Parkinson's and Alzheimer's diseases, spinal cord injury,
stroke, burns, heart disease, diabetes, osteoarthritis, and rheumatoid
arthritis.
For example, it may become possible to generate healthy heart muscle
cells in the laboratory and then transplant those cells into patients
with chronic heart disease. Preliminary research in mice and other
animals indicates that bone marrow stem cells, transplanted into a
damaged heart, can generate heart muscle cells and successfully
repopulate the heart tissue. Other recent studies in Cell Culture
systems indicate that it may be possible to direct the differentiation
of Embryonic Stem Cells or adult bone marrow cells into heart muscle
cells (Figure 4).
In people who suffer from type I diabetes, the cells of the pancreas
that normally produce insulin are destroyed by the patient's own immune
system. New studies indicate that it may be possible to direct the
differentiation of human embryonic stem cells in cell culture to form
insulin-producing cells that eventually could be used in
transplantation therapy for diabetics.
To realize the promise of novel Cell-based Therapies for such pervasive
and debilitating diseases, scientists must be able to easily and
reproducibly manipulate stem cells so that they possess the necessary
characteristics for successful differentiation, transplantation and
engraftment. The following is a list of steps in successful cell-based
treatments that scientists will have to learn to precisely control to
bring such treatments to the clinic. To be useful for transplant
purposes, stem cells must be reproducibly made to:
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Proliferate extensively and generate sufficient quantities of tissue.
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Differentiate into the desired cell type(s).
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Survive in the recipient after transplant.
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Integrate into the surrounding tissue after transplant.
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Function appropriately for the duration of the recipient's life.
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Avoid harming the recipient in any way.
Also, to avoid the problem of immune rejection, scientists are
experimenting with different research strategies to generate tissues
that will not be rejected.
To summarize, the promise of stem cell therapies is an exciting one,
but significant technical hurdles remain that will only be overcome
through years of intensive research.
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