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Stem Cell Technology: (Module in Cell Molecular Biology)

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STEM CELL

TECHNOLOGY
(MODULE IN CELL
MOLECULAR BIOLOGY)

BY; ShulamitAspiras
To; prof. Elizabeth olarte
Introduction

The life sciences segment for clinical applications has witnessed some developments in
the past few years with the emergence of human stem cell research. Stem cells are forerunners
of various cells present in the body which are unspecialized, undifferentiated cells, capable of
multiplying on their own. An immature stem cell, which is also its simplest form, can transform
itself into any possible mature cell. Depending upon its source, stem cells can be categorized
into three different categories viz. embryonic stem cells, adult stem cells and cord blood stem
cells.The use of stem cells in animals and humans for clinical research is an emerging science.
The use of ex-vivo expanded stem cells has been identified as the new use of drugs as per FDA,
USA, i.e. investigational new drugs.The Government of India has started promoting Stem Cell
Research in the country through its various science agencies. The key areas for research have
now been identified through discussions on basic and applied researches for various diseases.
Various programs have also been conducted supporting embryonic and adult stem cells. Some
of the major programs include establishments of hESC lines, use of limbal stem cells to repair
corneal surface disorder caused by limbal stem cell deficiency; isolation, purification and
characterization of haematopoitic, mesenchymal and liver stem cells; differentiation of stem
cells into neural, cardiac and cell lineages, etc. There are some technologies which are
frequently used in stem cell research and they are stem cell culture, separation and
identification techniques. At present, fluorescence activated cell sorting (FACS) and magnetic
activated cell sorting (MACS) are the most popularly used stem cell separation methods.
Further to that, FACS is the most common one amongst the two. This was the method used by
researchers to obtain the cancer stem cells from breast cancer, brain gliomas, prostate cancer,
gastric cancer, lung cancer, liver cancer and pancreatic cancer. Apart from stem cell separation
method, cell labeling technique has also been useful in stem cell research. By labeling specific
gene, functions of the stem cell could be detected and identified. Lastly, with the advances in
microscopy, confocal microscopy techniques have been developed and used to observe the
stem cells and their surroundings. Processes like monitoring multiple events, cell trafficking and
targeting would be noninvasive. The optical molecular area has witnessed some new algorithms
in the recent years such as multilevel adaptive finite element method, Bayesian approach and
graph cuts. New molecular imaging systems have been developed with advancements in
hardware and machine technique like spectrum CT of Cold Spring Biotech Corporation with
three-dimensional imaging function. Additionally, in order to capture the signals derived from
F-FDG probe Cerenkov Luminescence imaging (CLI) using Cerenkov theory has been developed.
Compared to the traditional imaging method, all these imaging technologies have shown their
advantage in tumor mechanism and antitumor evaluation research and have been applied in
the stem cell research.
Table of Contents
WHAT IS A STEM CELL?

Ultimately, every cell in the human body can be traced back to a fertilized egg that came
into existence from the union of egg and sperm. But the body is made up of over 200 different
types of cells, not just one. All of these cell types come from a pool of stem cells in the early
embryo. During early development, as well as later in life, various types of stem cells give rise to
the specialized or differentiated cells that carry out the specific functions of the body, such as
skin, blood, muscle, and nerve cells. Over the past two decades, scientists have been gradually
deciphering the processes by which unspecialized stem cells become the many specialized cell
types in the body. Stem cells can regenerate themselves or produce specialized cell types. This
property makes stem cells appealing for scientists seeking to create medical treatments that
replace lost or damaged cells.
TYPES OF STEM CELLS

Stem cells are found in all of us, from the early stages of human development to the end
of life. All stem cells may prove useful for medical research, but each of the different types has
both promise and limitations. Embryonic stem cells, which can be derived from a very early
stage in human development, have the potential to produce all of the body’s cell types. Adult
stem cells, which are found in certain tissues in fully developed humans, from babies to adults,
may be limited to producing only certain types of specialized cells. Recently, scientists have also
identified stem cells in umbilical cord blood and the placenta that can give rise to the various
types of blood cells.

Embryonic Stem Cells A blastocyst (BLAST-oh-sist), is a pre-implantation embryo that


develops 5 days after the fertilization of an egg by a sperm. It contains all the material
necessary for the development of a complete human being. The blastocyst is a mostly hollow
sphere of cells that is smaller than the period at the end of this sentence. In its interior is the
inner cell mass, which is composed of 30-34 cells that are referred to by scientists as
pluripotent because they can differentiate into all of the cell types of the body. In comon usage,
“embryo” can refer to all stages of development from fertilization until a somewhat ill-defined
stage when it is called a fetus. Scientists use terms such as “morula” and “blastocyst” to refer to
precise, specific stages of pre-implantation development. In order to be as precise as possible,
this booklet uses the scientific terms when describing scientific concepts but uses the term
“embryo” where more precision seemed likely to confuse rather than clarify. Embryonic stem
cells are derived from the inner cell mass of the blastocyst. In culture, they can self-replicate or
produce specialized cell types. In normal development, the blastocyst would implant in the wall
of the uterus to become the embryo and continue developing into a mature organism. Its outer
cells would begin to form the placenta and the inner cell mass would begin to differentiate into
the progressively more specialized cell types of the body. When the blastocyst is used for stem
cell research, scientists remove the inner cell mass and place these cells in a culture dish with a
nutrient-rich liquid where they give rise to embryonic stem cells. Embryonic stem cells seem to
be more flexible than stem cells found in adults, because they have the potential to produce
every cell type in the human body. They are also generally easier to collect, purify and maintain
in the laboratory than adult stem cells. Scientists can induce embryonic stem cells to replicate
themselves in an undifferentiated state for very long periods of time before stimulating them to
create specialized cells. This means that just a few embryonic stem cells can build a large bank
of stem cells to be used in experiments. However, such undifferentiated stem cells could not be
used directly for tissue transplants because they can cause a type of tumor called a teratoma.
To be used for therapies, embryonic stem cells would first need to be differentiated into
specialized cell types. Some find embryonic stem cell research to be morally objectionable,
because when scientists remove the inner cell mass, the blastocyst no longer has the potential
to become a fully developed human being.

Sources of Embryonic Stem Cells (In Vitro Fertilization)

The largest potential source of blastocysts for stem cell research is from in vitro
fertilization (IVF) clinics. The process of IVF requires the retrieval of a woman’s eggs via a
surgical procedure after undergoing an intensive regimen of “fertility drugs,” which stimulate
her ovaries to produce multiple mature eggs. When IVF is used for reproductive purposes,
doctors typically fertilize all of the donated eggs in order to maximize their chance of producing
a viable blastocyst that can be implanted in the womb. Because not all the fertilized eggs are
implanted, this has resulted in a large bank of “excess” blastocysts that are currently stored in
freezers around the country. The blastocysts stored in IVF clinics could prove to be a major
source of embryonA human blastocyst, which is produced about 5 days after fertilization, is
smaller than the period at the end of this sentence. NIH/Mr. J. Conaghan. ic stem cells for use in
medical research. However, because most of these blastocysts were created before the advent
of stem cell research, most donors were not asked for their permission to use these left-over
blastocysts for research. The in vitro fertilization (IVF) technique could potentially also be used
to produce blastocysts specifically for research purposes. This would facilitate the isolation of
stem cells with specific genetic traits necessary for the study of particular diseases. For
example, it may be possible to study the origins of an inherited disease like cystic fibrosis using
stem cells made from egg and sperm donors who have this disease. The creation of stem cells
specifically for research using IVF is, however, ethically problematic for some people because it
involves intentionally creating a blastocyst that will never develop into a human being.

Nuclear Transfer: The process called nuclear transfer offers another potential way to
produce embryonic stem cells. In animals, nuclear transfer has been accomplished by inserting
the nucleus of an already differentiated adult cell—for example, a skin cell—into a donated egg
that has had its nucleus removed. This egg, which now contains the genetic material of the skin
cell, is then stimulated to form a blastocyst from which embryonic stem cells can be derived.
The stem cells that are created in this way are therefore copies or “clones” of the original adult
cell because their nuclear DNA matches that of the adult cell. As of the summer of 2006,
nuclear transfer has not been successful in the production of human embryonic stem cells,1 but
progress in animal research suggests that scientists may be able to use this technique to
develop human stem cells in the future. Through nuclear transfer, scientists could produce a
blastocyst by inserting the nucleus from an adult cell (for example, a skin cell) into an egg
without a nucleus. All the stem cells derived from this blastocyst are genetically matched to the
adult cell. Claims by Korean scientists of successful derivation of human embryonic stem cells
using nuclear transfer have been found to be invalid and were retracted. Scientists believe that
if they are able to use nuclear transfer to derive human stem cells, it could allow them to study
the development and progression of specific diseases by creating stem cells containing the
genes responsible for certain disorders. In the future, scientists may also be able to create
“personalized” stem cells that contain only the DNA of a specific patient. The embryonic stem
cells created by nuclear transfer would be genetically matched to a person needing a
transplant, making it far less likely that the patient’s body would reject the new cells than it
would be with traditional tissue transplant procedures. Although using nuclear transfer to
produce stem cells is not the same as reproductive cloning, some are concerned about the
potential misapplication of the technique for reproductive cloning purposes. Other ethical
considerations include egg donation, which requires informed consent, and the possible
destruction of blastocysts.

Adult Stem Cells

Adult stem cells are hidden deep within organs, surrounded by millions of ordinary cells,
and may help replenish some of the body’s cells when needed. In fact, some adult stem cells
are currently being used in therapies. They have been found in several

Producing Embryonic Stem Cells .Using Nuclear Transfer Is Not the Same as
Reproductive Cloning

The use of nuclear transfer to develop disease-specific stem cells can be called research
cloning, and the use of this technique for personalized tissue transplants is sometimes called
therapeutic cloning. These terms must be carefully distinguished from reproductive cloning, in
which the intent is to implant a cloned embryo in a female’s womb and allow it to develop fully
into an individual. This was the technique by which Dolly the sheep was made and is now
widely used for reproductive cloning in animals. In humans, however, reproductive cloning has
been actively discouraged by most in the scientific community. The National Academies
concluded ,“Human reproductive cloning should not now be practiced. It is dangerous and likely
to fail” in the 2002 report Scientific and Medical Aspects of Human Reproductive Cloning.

Some of the known sources of adult stem cells. Organs that need a constant supply of
cells, such as the blood, skin, and lining of the gut, and have also been found in surprising places
like the brain, which is not known to readily replenish its cells. Unlike embryonic stem cells,
adult stem cells are already somewhat specialized. For example, blood stem cells normally only
give rise to the many types of blood cells, and nerve stem cells can only make the various types
of brain cells. Recent research however, suggests that some adult stem cells might be more
flexible than previously thought, and may be made to produce a wider variety of cell types. For
example, some experiments have suggested that blood stem cells isolated from adult mice may
also be able to produce liver, muscle, and skin cells, but these results are not yet proven and
have not been demonstrated with human cells. Nevertheless, scientists are working on finding
a way to stimulate adult stem cells, or even other types of adult cells, to be more versatile. If
they succeed, it could provide another source of unspecialized stem cells.

COMPARISON OF THE DIFFERENT SOURCES OF STEM CELLS

EMBRYONIC STEM CELLS ADULT STEM CELLS


ATTRIBUTES In Vitro Fertilization Nuclear transfer Adult Tissues
 Can produce all cell types  Can produce all cell  Demonstrated
 Relatively easy to types success in some
identify, isolate ,maintain  Relatively easy to treatments
and grow in the identify, isolate  Stem cells may
laboratory ,maintain and grow be genetically
 Large source of ‘’excess’’ in the laboratory matched to
blastocysts from IVF  Stem cells may be patient
clinics genetically matched
to patient
LIMITATIONS  Limited number of cell  Not yet achieved  Produce limited
lines available for with human cells number of cell
federally funded  Risk of creating types
research teratomas (tumors)  Not found in all
 Risk of creating from implanting tissues
teratomas (tumors) from undifferentiated  Difficult to
implanting stem cells identify , isolate
undifferentiated stem ,maintain , and
cells grow in the
laboratory
ETHICAL  Destruction of human  Destruction of  No major
CONCERNS blastocysts human blastocysts ethical
 Donation f blastocysts  Donation of eggs concerns have
requires informed requires informed been raised
consent consent
 Concern about
misapplication for
reproductive cloning
WORKING WITH STEM CELLS

The day-to-day work that goes on in the laboratories across the country studying stem cells
begins with developing ways to identify stem cells, culture cell lines, and stimulate stem cells to
differentiate. Once these first steps have been achieved, work on animals plays an important role in
furthering basic research and developing medical applications. This work is necessary to form the
foundation of knowledge that will point the way to medical advances.

Identifying Stem Cells As early as 1961, scientists knew that adult bone marrow contained cells
that could make all of the blood cell types. But it wasn’t until 1988 that those stem cells were isolated as
pure populations. Why did it take so long? The techniques for identifying stem cells have only recently
been developed. Partly, this is because adult stem cells are, by their very nature, inconspicuous in shape,
size, and function. They also tend to hide deep in tissues and are present only in very low numbers,
making their identification and isolation like finding a needle in a haystack.

How do scientists know when they have found a stem cell? Every cell displays an array of
proteins on its surface; different cell types have different proteins. Scientists can use these surface
proteins as “markers” that characterize individual cell types—a type of “molecular ID.” For example,
using molecules that recognize and attach to specific surface proteins and that can fluoresce under
certain wavelengths of light, scientists can visually tell the difference between a blood stem cell and a
mature white blood cell. Unfortunately, not all stem cells can now be identified in this manner because
scientists have not yet identified markers for all stem cell types. Scientists also identify stem cells by
observing their behavior in the laboratory: stem cells must be able to remain unspecialized and self-
renew for long periods of time. Scientists believe that there might be more types of adult stem cells than
the handful that have already been identified, but finding them is a difficult process.

Culturing Cell Lines and Stimulating Stem Cells to Differentiate Cell culture is a term that refers
to the growth and maintenance of cells in a controlled environment outside of an organism. A successful
stem cell culture is one that keeps the cells healthy, dividing, and unspecialized. The culturing of stem
cells is the first step in establishing a stem cell line—a propagating collection of genetically identical
cells. Cell lines are important because they provide a long-term supply of multiplying cells that can be
shared among scientists for research and therapy development. The National Academies report Stem
Cells and the Future of Regenerative Medicine (2001) described some of the challenges of maintaining
cell lines: “Over time, all cell lines…change, typically accumulating harmful genetic mutations. There is
no reason to expect stem cell lines to behave differently. While there is much that can be learned using
existing stem cell lines…such concerns necessitate continued monitoring of these cells as well as the
development of new stem cell lines in the future.” Once they have established a stable stem cell line,
scientists start the process of causing the stem cells to differentiate into specialized cell types. The
cellular environment in which stem cells naturally reside provides scientists with clues about how to
make them differentiate in a culture dish. For example, in the bone marrow, where blood stem cells
reside, bone cells send physical and chemical signals that tell the blood stem cells when to differentiate.
Scientists are just beginning to understand these signals and have developed ways to mimic the natural
processes in cell cultures. Usually, the technology involves adding certain proteins to the cell culture
and, in some cases, introducing specific genes into the stem cells. It will be essential that scientists are
sure that stem cells have fully differentiated before they can use them for medical applications. If
completely undifferentiated stem cells (such as embryonic stem cells) are implanted directly into an
organism, they can cause a type of tumor called a teratoma, which scientists have observed in
experiments using mice. Semi-specialized adult stem cells and differentiated cells derived from
embryonic stem cells are unlikely to cause teratomas.

The Role of Animals in Stem Cell Research For medical research, as well as for research that
explores the basic processes in the development of organisms and diseases, scientists often rely on
animals. Implanting human cells into animals such as mice has long been common practice in order to
test the safety and effectiveness of new drugs, procedures, and medical devices before clinical testing in
human volunteers. For stem cell research, scientists use animals to make sure the stem cells are able to
incorporate into the tissue, do not cause any harmful consequences, and function in concert with the
rest of the body. For example, before using stem cells to replace the pancreatic cells that are destroyed
by type I diabetes in humans, scientists will transplant human stem cells into a mouse to see whether
the stem cells yield healthy, insulin-producing cells. If their methods prove successful in mice, scientists
may eventually apply the technology to developing treatments for diabetes in humans. Animal studies
can also reveal how human cells differentiate during normal development. For example, scientists may
implant human stem cells into a developing mouse to observe the processes involved in building and
organizing the different tissue types that make up the human body. Scientists can also trace the
development and progression of certain diseases within an animal. By implanting human stem cells that
lead to a particular disease into a mouse blastocyst, scientists can observe when and how the afflicted
cells begin to show signs of disease and can test drugs that might prevent that process. Organisms that
contain cells or tissues from another individual of the same or a different species are called chimeras. A
common example of a chimera is a mouse that has been injected with some human cells so that it can
be used for studying a human disease or testing a new drug. A person who has had a blood transfusion
or a person who has received a heart valve transplant from a pig is technically a chimera, as well. The
making of chimeras for research has unique ethical implications that have been the topic of discussions
among scientists, ethicists and the public, especially when the chimeras contain both human and animal
cells.

Alternatives to Using Embryos in Stem Cell Research To address ethical concerns about the
destruction of blastocysts, scientists are trying to find new ways of obtaining stem cells that behave like
embryonic stem cells but that don’t require harming a blastocyst. As the science progresses, ethical
issues surrounding these alternatives may also arise. Some possible alternatives include:

• Cells collected from the morula (MOR-yoo-la), the developmental stage prior to the blastocyst.
The morula, a solid ball of about 16–30 cells, seems able to sustain the loss of a few cells without
developmental damage so that the remaining cells can continue to develop. Cell extraction from the
morula is already being used in some clinics to screen for genetic disorders in embryos produced by in
vitro fertilization. Researchers have recently shown that cells isolated from a mouse morula can give rise
to embryonic stem cells while the remaining morula cells develop into a healthy mouse. However, this
process may still be morally objectionable to some because of the chance of harm to the morula, and
because the long-term effects of removing cells from a morula are not yet known.

• The creation of embryonic stem cells through a process called altered nuclear transfer (ANT).
In this variation of the nuclear transfer technique, scientists create a blastocyst whose genetic material
has been changed so that further development and implantation into the uterus is not possible. It aims
to create embryo-like entities that are not truly embryos but that can be a source of pluripotent stem
cells. ANT, so far only tested with mouse blastocysts, could allow the creation of embryonic stem cells
without destroying a viable human blastocyst. Some who object to embryonic stem cell research
support ANT because the resulting blastocyst could never develop into a full human being and therefore
would not have the moral status of a human embryo. However, this procedure is objectionable to some
because they believe that it involves the creation of an imperfect blastocyst that is designed to be
destroyed.

• Causing an adult cell to act like an embryonic stem cell. During development, as cells become
more and more specialized, they gradually lose the ability to turn on the genes that allow embryonic
stem cells to be so versatile. The silencing of these genes seems to be responsible for keeping
specialized cells specialized and limiting the differentiation capacities of adult stem cells. By
“reprogramming” adult stem cells so that they can turn on the genes that allow versatility, scientists
hope to cause them to revert to a more flexible state. It is even possible that scientists could one day
“reprogram” any cell, not only stem cells. However, research in this area is in the early stages and
scientists may be many years away from making an adult cell as versatile as an embryonic stem cell.

WHY STEM CELL RESEARCH IS BEING PURSUED Right now, only a few diseases are treatable
with stem cell therapies because scientists can only regenerate a few types of tissues. However, the
success of the most established stem cell-based therapies—blood and skin transplants—gives hope that
someday stem cells will allow scientists to develop therapies for a variety of diseases previously thought
to be incurable. Many major diseases are caused by the loss of a single type of cell or tissue. For
example, type I diabetes (juvenile-onset) is caused by the loss of the insulin-producing cells of the
pancreas, and its treatment is limited to merely alleviating the symptoms. Finding a cure for such
diseases would be much easier if scientists could simply re-grow the missing or damaged cells and
implant them into patients.

Blood Stem Cells After scraping a knee or donating blood, the body replenishes the blood cells
that are lost by drawing on a small number of semi-specialized hematopoietic (heem-AT-oh-poh-EH-tik)
stem cells contained in the blood and bone marrow. For decades, scientists have been using this type of
adult stem cell to treat patients with diseases such as leukemia, sickle cell anemia, bone marrow
damage, and some metabolic disorders and immunodeficiencies where the body has lost its ability to
replenish its own set of healthy blood cells. Hematopoietic stem cells give rise to all the blood cell types,
from infection-fighting white blood cells to blood-clotting platelets. Preliminary results have suggested
that they may also be able to produce other cell types not found in blood, but this is not yet proven.
WHY STEM CELL RESEARCH IS BEING PURSUED In the past, the only way to use hematopoietic
stem cells for therapies was through bone marrow transplants. Extracting bone marrow is an
uncomfortable and invasive procedure, and in order for a transplant to work, the donor and recipient
must be genetically similar. If they are too genetically different, the blood cells produced from the
transplanted marrow may recognize the patient’s body as foreign and fight against the patient’s own
cells and organs. Additionally, the patient’s immune system may reject the transplant, causing a
dangerous “war” within the patient’s body. More recently, scientists have developed ways to derive
hematopoietic stem cells from the blood contained in the umbilical cord and placenta at birth. The stem
cells isolated from a person’s own umbilical cord blood and placenta, if used for therapies later in life,
would be less likely to cause an “internal war” within the recipient’s body. They are also more accessible
than the stem cells in bone marrow because the extraction of this blood poses no risk to the mother or
infant.

The Changed Face of Skin Grafts For many years, scientists have been harnessing the
regenerative capabilities of human skin to treat victims of severe burns using skin transplants. Skin
transplants are possible because of the existence of stem cells located just under the top layer of skin.
Every day, thousands of new skin cells are produced to replace those that have been shed. When
someone suffers severe burns that destroy the source of these stem cells, their skin can no longer
regenerate on its own. Traditionally, doctors treated severe burns by transplanting sections of skin from
undamaged areas of the body onto the burned areas, but if doctors could not find enough unharmed
skin to cover the burned areas, the patient could die. Now, scientists can grow vast sheets of new skin
by culturing the stem cells from small pieces of healthy skin. This practice, which is a type of tissue
engineering, has become routine for treating burn victims over the past 20 years. Recently, scientists
have identified other types of stem cells in hair follicles and deeper layers of the skin. The inclusion of
these new stem cells into engineered skin should help create more natural-looking skin transplants in
the future.

Stem Cells Found in Umbilical Cord Blood In 2005, the National Academies issued a report,
Cord Blood: Establishing a National Hematopoietic Stem Cell Bank Program, which recommended that a
national cord blood “bank” be established to harness the medical potential of this source of stem cells.
Such a bank would not only benefit the people from whom the blood was collected but anyone in need
of blood transplants. As with blood banks for blood transfusions, scientists could screen the bank to find
the best match for each patient, providing a safer, more personalized living-cell therapy.

Possible Future Treatment for Parkinson’s Disease? When most people reach for a pen, their
body acts in one smooth and controlled movement. This is because the instant a person thinks of
grabbing the pen, a series of nerve cells fire in an orchestrated symphony from the brain to the muscles
responsible for that action. For the movement to be precise and smooth, all the nerve cells in the
“grabbing-the-pen network” must function properly, including cells that tell unneeded muscles to stay
still. In Parkinson’s disease, the brain cells responsible for keeping unneeded muscles from moving
degenerate and die. This results in progressively more dramatic and uncontrolled movements, tremors,
and spasms. To date, there is no cure for Parkinson’s disease because no one has figured out a way to
bring back the specialized nerve cells that have died.
Are the Promises of Stem Cell Therapies Realistic? The list of medical achievements stem cells
could offer seems to be expanding at an incredible pace. The role of stem cells in medicine is already
very real, but there is a danger of exaggerating the promise of new medical developments. What tend to
be “over-promised” are not only the potential outcomes of both embryonic and adult stem cell
research, but also the time scales that are involved. The basic research needed to develop viable
therapeutic options is a lengthy process that may extend over many years and decades. Even after
science has moved from basic research to developing medical applications, it still takes many years to
thoroughly test those applications and demonstrate that they are safe to prescribe for patients. This is
true for all medical treatments, including the development of new drugs, procedures, and medical
equipment, and is not specific to the living cell therapies made possible by stem cell research. There are
also many legal and social questions that must be addressed before stem cell-based therapies become
clinically available. Legal issues that will affect stem cell applications include how to address intellectual
property concerns and how to apply and enforce diverse and sometimes conflicting state and national
laws. Social issues include concerns about the destruction of embryos, the distribution of the benefits of
the research, and the protection of both physical and privacy interests of egg and sperm donors and
clinical research subjects.

1956 First successful bone marrow transplant

1981 Embryonic stem cells are isolated from mouse blastocysts 1988 Hematopoietic (blood)
stem cells from adult mice are purified and characterized

1992 Stem cells are identified in the adult human brain

1998 The first human embryonic stem cells are isolated Because Parkinson’s disease results
from the loss of one specific type of nerve cell, stem cells offer a very tangible possibility for treatment.
Researchers have recently learned how to differentiate embryonic stem cells into the specific type of
brain cell that is lost in Parkinson’s disease. They have also successfully transplanted adult nerve stem
cells into rat brains. When this technique is proven to be effective and safe, transplantation of stem cells
into the brains of patients may one day allow doctors to reverse the burden of Parkinson’s disease and
restore control of movement. Another strategy currently under study is the addition of chemicals or
growth factors that aim to induce the patient’s own stem cells to repair the damaged nerves without
needing to grow and transplant stem cells.

Possible Fix for Diabetes? In people who suffer from type I diabetes, the beta cells of the
pancreas that normally produce insulin are destroyed by the patient’s overactive immune system.
Without insulin, the cells of the body cannot take up glucose and they starve. Patients with type I
diabetes

STEM CELL TIMELINE require insulin injections several times a day for their entire lives. The only
current cure is a pancreatic transplant from a recently deceased donor, but the demand for transplants
far outweighs the supply. While adult stem cells have not yet been found in the pancreas, scientists have
made progress transforming embryonic stem cells into insulin-producing cells. Combining beta-cell
transplants with methods to “fix” the patient’s immune system—including chemotherapy to destroy
malfunctioning immunesystem cells and blood transplants to replenish healthy white blood cells—could
offer great hope for the many Americans suffering with type I diabetes.

Cancer: Getting to the Root of the Problem Why are some cancers so hard to eliminate, even
after many rounds of chemotherapy? The answer may lie in a few abnormal stem cells. Cancerous stem
cells were first identified in 1997 when a research group from the University of Toronto transferred a
few blood stem cells from human leukemia patients into mice and watched leukemia develop in the
mice. Stem celllike cells have also recently been found in breast and brain tumors. Like normal stem
cells, tumor stem cells exist in very low numbers, but they can replicate and give rise to a multitude of
cells. Unlike normal stem cells, however, cancerous stem cells lack the controls that tell them when to
stop dividing. Traditional chemotherapy kills off the majority of the tumor cells, but if any of the
cancerous stem cells survive the treatment, the cancer may return. Research into the differences in
gene expression between normal and tumor stem cells may lead to treatments where the root of the
problem—the cancer stem cell—is targeted.

Stem cell research continues to advance. Preliminary results from recent studies support the
promise of stem cells for conducting basic research that may eventually lead to medical achievements.
For example, in 2005, human embryonic stem cells were shown to differentiate into active functioning
nerve cells when placed in mouse brains. Scientists also made significant progess in deriving pancreatic
cells from adult stem cells. In 2006, scientists were able to derive embryonic stem cells from the morula
of a mouse, and embryonic stem cells were first grown without animal products in the culture. Results
of these and other recent experiments must be replicated and consistently demonstrated by other
researchers before they become generally accepted by the scientific community.

2001 Mouse embryonic stem cells are created by nuclear transfer

2002 Pancreatic cells derived from mouse embryonic stem cells cure diabetes in mice

2004 The type of nerve cell lost in Parkinson’s disease is produced from human embryonic stem
cells

17 Stem cells offer opportunities for scientific advances that go far beyond regenerative
medicine. They offer a window for addressing many of biology’s most fundamental questions. Watching
embryonic stem cells give rise to specialized cells is like peeking into the earliest development of the
many tissues and organs of the human body. Stem cell research may help clarify the role genes play in
human development and how genetic mutations affect normal processes. They can be used to study
how infectious agents invade and attack human cells, to investigate the genetic and environmental
factors that are involved in cancer and other diseases, and to decipher what happens during aging. Stem
cells may also revolutionize traditional chemical medicine. Because embryonic stem cells can continue
to divide for long periods of time and produce a variety of cell types, they could provide a valuable
source of human cells for testing drugs or measuring the effects of toxins on normal tissues without
risking the health of a single human volunteer. In the future, thousands of compounds could be quickly
tested on a wide assortment of cell types derived from stem cells, making drug discovery more efficient
and cost effective. Using nuclear transfer to produce stem cells could be particularly useful for testing
drugs for disorders that are of genetic origin. For example, it is difficult to study the progression of
Alzheimer’s and Parkinson’s diseases in the brains of live patients— but by using the cells of an
Alzheimer’s patient to create stem cell lines with nuclear transfer, scientists could trace the
development of the disease in a culture dish and test drugs that regenerate lost nerve cells with no
danger to the patient. Stem cells may also help scientists calculate the effects of toxic substances in
drugs, food, and the environment.

Is an Embryo a Person? The controversy over embryonic stem cell research touches on some of
the same fundamental questions that society has grappled with in the debates over contraception,
abortion, and in vitro fertilization. The questions at the center of the controversy concern the nature of
early human life and the legal and moral status of the human embryo. Embryonic stem cell research
often involves removing the inner cell mass from “excess” blastocysts that are unneeded by couples
who have completed their fertility treatment. This prevents those blastocysts from continuing to
develop. Although such blastocysts would likely be discarded (and thus destroyed) by the clinics in any
case, some believe that this does not make it morally acceptable

ETHICS, MORAL VALUES, AND U.S. LAW The National Academies published the Guidelines for
Human Embryonic Stem Cell Research in 2005. Here, members of the committee present at a report
briefing. to use them for research or therapeutic purposes. They believe that the life of a human being
begins at the moment of conception and that society undermines a commitment to human equality and
to the protection of vulnerable individuals if blastocysts are used for such purposes. Some cultures and
religious traditions oppose the use of human life as a means to some other end, no matter how noble
that end might be. Other traditions support embryonic stem cell research because they believe that the
embryo gains the moral status of a human being only after a few weeks or months of development.
Many traditions emphasize obligations to heal the sick and ease suffering—goals for which embryonic
stem cell research holds great potential—and favor embryonic stem cell research for this reason. Several
religious groups are currently involved in internal discussions about the status of the human embryo and
have not yet established official opinions on the matter. Public opinion polls suggest that the majority of
both religious and non-religious.

The National Academies’ Guidelines for Human Embryonic Stem Cell Research In order to
provide all scientists—those working in universities and private companies and with both public and
private funding—with a common set of scientific and ethical guidelines, the National Academies
published the Guidelines for Human Embryonic Stem Cell Research in 2005. The report outlines the need
for institutional oversight mechanisms for monitoring all human embryonic stem cell research and
provides specific guidance regarding the derivation of new stem cell lines. Under the guidelines, certain
activities, such as experimenting on human embryos by inserting stem cells into them, are not
permitted. The guidelines also require that all egg, sperm, and blastocyst donations follow appropriate
informed consent and confidentiality procedures. Because the ethical and technical questions
associated with human embryonic stem cell research are likely to change as science advances, in 2006,
the National Academies established a panel of experts to monitor and review scientific developments
and changing ethical, legal, and policy issues and to prepare periodic reports to update the guidelines as
needed. For more information on the guidelines, please visit www.nationalacademies.org/stemcells.
Americans support embryonic stem cell research, although public opinion seems divided about the
creation or use of human blastocysts solely for research. The Relationship of Stem Cell Research to
Reproductive Cloning Although cloning and stem cell research are often lumped together in the context
of ethical debates, the goals and results of the two are very different. The common factor between
current attempts at reproductive cloning and stem cell research is a laboratory technique called nuclear
transfer. Using nuclear transfer, scientists can create blastocysts containing stem cells that are “clones”
of a single adult cell by inserting the genetic material from an adult cell (for example, a skin cell) into an
egg whose nucleus has been removed (this process is described in more detail on page 6). Scientists
hope that they could derive stem cells from the cells inside such blastocysts and grow replacement
tissues that are genetically matched to specific patients, thus offering patients a safer alternative to
traditional tissue transplants. Reproductive cloning, such as the process that was used to create Dolly
the sheep, also uses the nuclear transfer technique. However, instead of removing the inner cell mass to
derive a stem cell line, the blastocyst is implanted into the uterus and allowed to develop fully. In 2002,
the National Academies issued the report Scientific and Medical Aspects of Human Reproductive
Cloning, which concluded “Human reproductive cloning should not now be practiced. It is dangerous
and likely to fail.” The Ethics of Human-Animal Chimeras Chimeras are organisms composed of cells or
tissues from more than one individual. Chimeras have been produced for research for many years, but
when human and animal cells are mixed in the laboratory, there is a clear need for heightened ethical
consideration. Cells from different organisms can be combined either in the early developmental stages
(for example, introducing human cells into a mouse blastocyst to observe certain developmental
processes) or after an individual is fully developed (for example, implanting 21 ETHICS, moral values,
AND U.S. LAW “Human reproductive cloning should not now be practiced. It is dangerous and likely to
fail.” —Scientific and Medical Aspects of Human Reproductive Cloning, National Academies Press, 2005
human stem cell-derived pancreatic cells into a mouse to test their ability to function in a living body).
Chimeras are considered essential for advancing stem cell research to viable therapies, since no therapy
can be tested in humans without research in animals first. Some people believe that the creation of
chimeras involving human cells for medical research is morally acceptable as long as the chimera has no
level of human consciousness. Therefore, research in which it is possible for human stem cells to
produce part of an animal’s brain should be conducted with great care. The National Academies’
guidelines prohibit the introduction of human cells into the blastocyst of a non-human primate, or the
introduction of any animal or human cells into a human blastocyst. The guidelines also prohibit the
breeding of human-animal chimeras in the unlikely event that any human genetic material would be
contained in their reproductive cells.

Advantages and Disadvantages of Stem Cell


Research
 Advantages and Disadvantages
Stem cell research is the use of either adult or embryonic cells to treat and cure
diseases. Stem cells are immature and not fully developed cells of the human body that
have the potential to be manipulated into different types of cells. There is a large debate
surrounding stem cell research, which is mostly a moral one. Let’s take a look at the
good and bad that could come from the use of stem cells in our society.

The Advantages of Stem Cell Research


1. Immense Medical Benefits
The main purpose of researching stem cells is to asses their use in the medical field. It
has been shown that they can be extremely beneficial in therapeutic cloning to treat
chronic illnesses. They have also been a wonderful stepping stone into the
advancement of regenerative medicines.

2. A Better Knowledge of Human Growth


By studying the very foundation of human growth, stem cells, in depth, scientists have
been able to gain a much better understanding of how our bodies work. This is vital for
advancing medicine, medical processes, and even cures for degenerative illnesses.

3. The Key To Reversing Aging


Stem cells are the key to regenerative processes, which could possibly be age reversal.
By reviving organs in our body that have become old or worn out, you could essentially
live forever or at least greatly prolong the lives of humans.

4. Cure Development Defects Before They Happen


Stem cell research doesn’t just benefit the people walking around the earth, but also the
ones that haven’t been born yet. The effects of these cells on embryos could change
the way that birth defects are treated. They could possibly be corrected before the child
is even born, greatly improving their quality of life and chance of survival.

The Disadvantages of Stem Cell Research


1. High Uncertainties
One of the largest issues that people have with stem cell research is just how risky and
unknown it truly is. Major advancements still need to be made and new technology must
be developed before it can be used in an efficient and safe way.

2. Unknown Side Effects


Since stem cells are still in their research phase, the true long term effects of their use is
not known or understood. Using them could cause new illnesses and disease to
develop, or even interfere with the natural functions of the body.

3. Playing God
The moral argument is another big one when it comes to this topic. Many people believe
that altering the basic structure of a human’s genes is putting hands somewhere they
should not be. The moral issue is the most frequently argued.

4. Perpetuates Cloning of Humans


Cloning is the process of making an exact genetic copy of a living organism. Stem cell
research also aids the research into human cloning. Human cloning has been widely
reviewed as a detriment to society with many negative benefits.

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Important Facts About Stem Cell Research


 Parkinson’s disease, spinal cord injuries, birth defects, damaged organs, and cancer
could all be possibly treated with the use of stem cells.
 Bone marrow transplantation is the only form of stem cell research that has been
proven to be effective and safe.
 Manipulating cells and their functions in a clinical setting or laboratory can hinder their
normal functions and render them dangerous for use.
 Stem cells can copy themselves for an unlimited period of time.
 The reason that they have gained so much attention, is because stem cells are
“blank” cells that can be turned into almost anything that the body needs.
 Therapeutic cloning is a type of stem cell research.

Benefits of Stem Cells


By: Ian Murnaghan BSc (hons), MSc - Updated: 19 Jan 2017 | *Discuss

With all the controversy surrounding stem cells you may have missed hearing about
many of the benefits for the health and medical fields. You may not even be aware that
stem cells already have many applications for treating disease. Their potential to treat
even more diseases in the future means that scientists are working hard to learn about
how stem cells function and how they can treat some of the more serious diseases
affecting the world.

Stem Cells and Human Development


Stem cells have enormous potential in health and medical research but to fully harness this
potential, scientists are studying how stem cells transform, or differentiate, into the diverse
range of specialised cells that make humans what they are today. Because diseases such as
cancer or conditions such as birth defects are thought to occur because of problems in the
differentiation process, an understanding of the development that happens in normal cells will
help scientists treat the developmental errors that can occur.
Stem Cells and Cell-Based Therapies
Another potential application of stem cells is to form cells and tissues for medical therapies.
Currently, it is donated organs and tissues that are substituted for damaged or dysfunctional
ones. Sadly, the number of people awaiting a transplant is much higher than the number of
available organs. Transplant waiting lists are enormous and many people die awaiting
transplants. Stem cells offer a viable source of replacement cells to treat diseases and can
potentially reduce the morbidity and mortality for those awaiting transplants. Some of the areas
that stem cells can benefit include:

 Parkinson's disease
 Type I diabetes
 Arthritis
 Burn victims
 Cardiovascular diseases

Potential to Reverse Diseases


By directing stem cells to differentiate into specialised cell types, there is the exciting possibility
to provide a renewable source of replacement cells for those suffering from diseases.

The potential to reverse diseases is also not a foreign one.

Heart Attack - For example, a patient who has suffered from a heart attack and
sustained heart damage could have the damaged tissue replaced by healthy new
muscle cells.

Parkinson's Disease - The destruction of brain cells in conditions such as


Parkinson's disease can hopefully be reversed with the replacement of new, healthy
and functioning brain cells.

Genetic Defects - Even more promising is the potential to address genetic defects
that are present from birth by restoring function and health with the introduction of
normal healthy cells that do not have these defects.

Burn Victims
Burn victims tend to endure an enormous amount of pain from their wounds as well as
frustration from the challenges of healing. Instead of donor tissues being donated, stem cells
could be used to produce new and healthy tissues. This is essentially similar to therapies
already being used, such as bone marrow transplants, where stem cells create different
specialised blood cells.

Scientists aim to locate and remove specific stem cells from a tissue and then trigger
them to differentiate outside of the body before transplanting them back into the patient
to replace damaged tissues. In burn victims, a very small piece of the skin can be
progressively grown, allowing doctors to cover a burn that is often much larger than the
original size of the skin piece.

Stem Cells and Drug Testing


Stem cells have an important benefit for the pharmaceutical field. New drugs can be tested on
stem cells to assess their safety before testing drugs on animal and human models. For
example, a cancer cell line could be created to test an anti-tumour drug. If the conditions can be
perfectly replicated, testing drugs could provide very accurate results.

The current benefits of stem cell usage are already well documented and it is expected
that continued research will pave the way for new treatments. For those suffering from
serious diseases, stem cells offer hope for effective treatment or perhaps even a
reversal of the disease. Time will confirm the full success of stem cell therapies and
continued research should teach us more about using stem cells to treat debilitating
medical conditions.

Check out the features on stem cell therapy on this site for more information.

Benefits of Stem Cells


By: Ian Murnaghan BSc (hons), MSc - Updated: 19 Jan 2017 | *Discuss

With all the controversy surrounding stem cells you may have missed hearing about
many of the benefits for the health and medical fields. You may not even be aware that
stem cells already have many applications for treating disease. Their potential to treat
even more diseases in the future means that scientists are working hard to learn about
how stem cells function and how they can treat some of the more serious diseases
affecting the world.
Stem Cells and Human Development
Stem cells have enormous potential in health and medical research but to fully harness this
potential, scientists are studying how stem cells transform, or differentiate, into the diverse
range of specialised cells that make humans what they are today. Because diseases such as
cancer or conditions such as birth defects are thought to occur because of problems in the
differentiation process, an understanding of the development that happens in normal cells will
help scientists treat the developmental errors that can occur.

Stem Cells and Cell-Based Therapies


Another potential application of stem cells is to form cells and tissues for medical therapies.
Currently, it is donated organs and tissues that are substituted for damaged or dysfunctional
ones. Sadly, the number of people awaiting a transplant is much higher than the number of
available organs. Transplant waiting lists are enormous and many people die awaiting
transplants. Stem cells offer a viable source of replacement cells to treat diseases and can
potentially reduce the morbidity and mortality for those awaiting transplants. Some of the areas
that stem cells can benefit include:

 Parkinson's disease
 Type I diabetes
 Arthritis
 Burn victims
 Cardiovascular diseases

Potential to Reverse Diseases


By directing stem cells to differentiate into specialised cell types, there is the exciting possibility
to provide a renewable source of replacement cells for those suffering from diseases.

The potential to reverse diseases is also not a foreign one.

Heart Attack - For example, a patient who has suffered from a heart attack and
sustained heart damage could have the damaged tissue replaced by healthy new
muscle cells.

Parkinson's Disease - The destruction of brain cells in conditions such as


Parkinson's disease can hopefully be reversed with the replacement of new, healthy
and functioning brain cells.

Genetic Defects - Even more promising is the potential to address genetic defects
that are present from birth by restoring function and health with the introduction of
normal healthy cells that do not have these defects.
Burn Victims
Burn victims tend to endure an enormous amount of pain from their wounds as well as
frustration from the challenges of healing. Instead of donor tissues being donated, stem cells
could be used to produce new and healthy tissues. This is essentially similar to therapies
already being used, such as bone marrow transplants, where stem cells create different
specialised blood cells.

Scientists aim to locate and remove specific stem cells from a tissue and then trigger
them to differentiate outside of the body before transplanting them back into the patient
to replace damaged tissues. In burn victims, a very small piece of the skin can be
progressively grown, allowing doctors to cover a burn that is often much larger than the
original size of the skin piece.

Stem Cells and Drug Testing


Stem cells have an important benefit for the pharmaceutical field. New drugs can be tested on
stem cells to assess their safety before testing drugs on animal and human models. For
example, a cancer cell line could be created to test an anti-tumour drug. If the conditions can be
perfectly replicated, testing drugs could provide very accurate results.

The current benefits of stem cell usage are already well documented and it is expected
that continued research will pave the way for new treatments. For those suffering from
serious diseases, stem cells offer hope for effective treatment or perhaps even a
reversal of the disease. Time will confirm the full success of stem cell therapies and
continued research should teach us more about using stem cells to treat debilitating
medical conditions.

Check out the features on stem cell therapy on this site for more information.

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