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DR. ROOPAMJAIN
Professor & Head
STEM CELL
Introduction
Features of stem cells
3 Unique features of stem cells
Distinguishing features of progenetor/precursor cells
& Stem cells
classification of stem cell
Embryonic stem cell
Adult stem cell
Induced pluripotent stem cells
Regenerative medicine & cell therapy
Clinical applications
Introduction
• self-renewal
• differentiation.
• Self-renewal is the ability of cells to proliferate.
• blank cell / precursor cell
• building block of the human body
FeaturesofStemCells
unique cells.
amazing ability to developinto several distinct cell types
Stem Cells can be used as a repair system for the body.
divide without limit in order to replenish various types of cells.
Whena stem cell divides - remain a stem cell or become
another type of cell with a more specialized function
(i.e. a muscle cell, a red blood cell, a brain cell, etc.).
Threeuniquepropertiesofstemcells
Stem cells are capable of
dividing & renewing themselves
for long periods
Threeuniquepropertiesofstemcells
A stem cell is "uncommitted," until it receives a signal to
develop into a specialized cell
Threeuniquepropertiesofstemcells
Stem cells are unspecialized cells that develop into
specialized cells
(make up the different types of tissues in the
human body).
Distinguishing Features ofProgenitor/Precursor Cellsand StemCells.
A stem cell is an unspecializedcell that develops into a
variety of specialized cell types.
A stem cell divides and gives rise to one additional stem
cell and a specializedcell.
Example:
a hematopoietic stem cell produce a second generation
stem cell and a neuron.
Distinguishing Features ofProgenitor/Precursor Cellsand StemCells(Conti……..)
A progenitor cell is unspecialized that is capable of
undergoing cell division and yielding two specialized
cells.
Example: a myeloid progenitor/precursor cell undergoing
cell division to yield two specialized cells (a neutrophil
and a red blood cell).
classification
The potency of a stem cell is defined by the types of
more differentiated cells that the stem cell can make.
Stem cells can beeither:
• totipotent,
• pluripotent,
• multipotent, or
• unipotent.
Totipotent cells have the capability to produce all
cell types of the developing organism, including both
embryonic and extraembryonic (eg, placenta) tissues.
Totipotentstemcells
Pluripotentcells
Pluripotent cells can only make cells of the embryo
proper,
Can make all cells of the embryo (germ cells, cells from
any of the germ layers. Therefore, they can make any
cell of the body.
Multipotentcells
Multipotent cells can only make cells within a given
germ layer.
For example, multipotent stem cells from a mesodermal
tissue like the blood can make all the cells of the blood,
but cannot make cells of a different germ layer such as
neural cells (ectoderm) or liver cells (endoderm).
Unipotentcells
Unipotent cells make cells of a single cell type.
An example is a germ cell stem cell that makes the
cells that mature to become egg or sperm, but
not other cell types.
Embryonicstemcells
Embryonic stem cells are MOST UNDIFFIRENTIATED
Derived from the inner cell mass of a blastocyst.
LIMITLESS Cell renewal capacity
Can give rise to every cell in the body (Therefore said
TOTIPOTENT)
Induced to form specialized cells of all 3 germ cell layers
(neuron, cardiac muscle, liver cells, pancreatic islets cells
Embryonic stemcells
Human embryos reach the blastocyst stage 4–5 days post
fertilization, at which time they consist of 50–150 cells.
Human ES cells measure approximately 14 μm
This would provide a radical new treatment approach to a
wide variety of conditions where age, disease, or trauma
has led to tissue damage or dysfunction.
Advantagesof EmbryonicStem Cell
1. Flexible - appear to have the potential to
makeany cell.
2.Immortal - one embryonic stem cell line can
potentially provide an endless supply of cells
with definedcharacteristics.
3. Availability - embryos from in vitro
fertilization clinics.
Disadvantages of Embryonicstemcell
1. Difficult to differentiate uniformly and
homogeneously into a targettissue.
2. Immunogenic - embryonic stem cells from a
random embryo donor are likely to be rejected after
transplantation
3. Tumorigenic - capable of forming tumors or
promoting tumor formation.
4. Destruction of developing human life.
ADULT STEM CELLS
•Undifferentiated cell, found among differentiated cells
(Stem cell niches).
•The adult stem cell can renew itself and can differentiate to
yield some or all of the major specialized cell types of the
tissue or organ.
•The primaryroles of adult stem cells in a living organismare
to maintain and repair the tissue in which they are
found.
•Scientistsalso use the termsomaticstemcell instead of
adult stem cell.
ADULT STEM CELLS
Differentiationpathwaysofadult stemcells
Neural stem cells - in the brain give rise to its three
major cell types: nerve cells (neurons) and two categories
of non-neuronal cells —astrocytes and oligodendrocytes.
Epithelial stem cells - lining of the digestive tract occur in
deep crypts) - give rise to several cell types: absorptive
cells, goblet cells, Paneth cells, and enteroendocrine cells.
Skin stem cells - occur in the basal layer of the epidermis
and at the base of hair follicles.
The epidermal stem cells - give rise to keratinocytes
The follicular stem cells - can give rise to both the hair
follicle and to the epidermis
Adult stem cells from bone marrow and umbilical cords
appear to be as flexible asthe embryonic type
Somewhat specialized – inducement may be simpler.
Not immunogenic - recipients whoreceive the
products of their own stemcells will not experience
immunerejection.
Relative ease of procurement - some adult stem
cells are easy to harvest (skin, muscle,
marrow, fat)
Non-tumorigenic- tend not to form tumors.
Noharm done to the donor.
Advantages ofAdult StemCell
Disadvantages ofAdult stem cells
Limited quantity - can sometimes be difficult to
obtain in large numbers.
Finite - may not live as long as embryonic
stem cells in culture.
Less flexible - may be more difficult to reprogram
to form other tissue types
Whyare adult stemcells preferable to embryonic stem cells?
• Adult stem cells are naturally exist in our bodies,
and provide a natural repair mechanism for many
tissues.
• They belong in the microenvironment of an
adult body, while embryonic stem cells belong in
the microenvironment of the early embryo, where
they tend to cause tumors and immune system
reactions.
Induced pluripotent stem cells
• Induced pluripotent stem cells (iPSCs) are adult cells that
have been genetically reprogrammed to an embryonic stem
cell–like state.
• Mouse iPSCs were first reported in 2006
• human iPSCs were first reported in late 2007.
• Mouse iPSCs demonstrate important characteristics of
pluripotent stem cells, including expressing stem cell markers.
• Human iPSCs also express stem cell markers and are
capable of generating cells characteristic of all three germ
layers.
(1)Isolate and culture donor cells.
(2)Transduce stem cell-associated genes into the cells by viral
vectors. Red cells indicate the cells expressing the exogenous genes.
(3)Harvest and culture the cells according to ES cell culture.
(4)A small subset of the transfected cells become iPS cells and
generate ES-like colonies.
SourceofStemcells
from autologus,
• Stem cells may be derived
allogeneic or xenogenic sources.
• Histocompatability is prerequisite for transplantation
of allogeneic stem cells.
• Fatal tissue is the best current tissue source for
human neural stem cells, however ethical issues are a
majorconcern.
Potential sources of stem cells
Fetal tissue that becomes available after an abortion
Excess embryos from assisted reproductive technologies such
as commonly used in fertility clinics
Embryos created through in vitro fertilization specifically
for research purpose
Other sources of stem cells are those from umbilical cord blood,
& bone marrow.
In addition, neural stem cells, haematopoetic stem cells &
mesenchymal stem cells can be harvested from fetal blood and
fetal tissue.
Placenta a Source of Stem Cells
Placental stem cells, like
umbilical cord blood and bone
marrow stem cells, can be used to
cure chronic blood-related
disorders such as sickle cell
disease, Thalasemia, and
leukaemia.
Umbilical Cord Blood Stem Cell
Transplant
Umbilical cord blood stem
cell transplants are less prone to
rejection than either bone marrow
or peripheral blood stem cells.
This is probably because the cells
have not yet developed the features
that can be recognized and attacked
by the recipient's immune system.
Applications
• Stemcells can be used to studydevelopment
• Stem cells may help us understand how a complex organism develops from a
fertilised egg.
• In the laboratory, scientists can follow stem cells as they divide and become
increasingly specialized, making skin, bone, brain, and other cell types. Identifying
the signals and mechanisms that determine whether a stem cell chooses to carry on
replicating itself or differentiate into a specialized cell type, and into which cell type,
will help us understand what controls normal development.
• Some of the most serious medical conditions, such as cancer and birth defects, are
due to abnormal cell division anddifferentiation.
• 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. This is an important goal of stem cell research.
Stem cells have the ability to replace
damaged cells and treat disease
•treatment of extensive burns,
•to restore the blood system in patients with leukaemia and other
blooddisorders.
•Stem cells may also hold the key to replacing cells lost in many
other devastating diseases
•Stem cells, offer the possibility of a renewable source of
replacement cells and tissues to treat diseases including
Parkinson's, stroke, heart disease and diabetes.
Stem cells could provide a resource for
testing new medical treatments
•New medications could be tested for safety on
specialized cells.
•Other kinds of cell lines are already usedin this way.
•used to screen potential anti-tumourdrugs.
Celltherapy.
• Treatment of neural diseases such as Parkinson's
disease, Huntington’s disease andAlzheimer's
disease.
Stem cells could be used to repair or replace
damaged neurons.
Repair of damaged organs such as the liver and
pancreas.
Treatments forAIDS.
H
o
wDoesCellTherapyWork?
Stem cells can be used to
generate healthy and
functioning specialized
cells, which can then
replace diseased or
dysfunctional cells.
It is similar to the process of
organ transplantation only the
treatment consists of
transplanting cells instead of
organs.
How Does Cell TherapyWork?
Bone marrow transplants are an example of cell
therapy in which the stem cells in a donor's marrow are
used to replace the blood cells of the victims of
leukemia.
Cell therapy is also being used in experiments to graft
new skin cells to treat serious burn victims, and to grow
newcorneas for the sight-impaired.
In all of these uses, the goal is for the healthy cells
to become integrated into the body and begin to
function like the patient's owncells.
Stem cells & Regenerative Medicine

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Stem cells & Regenerative Medicine

  • 1. DR. ROOPAMJAIN Professor & Head STEM CELL
  • 2. Introduction Features of stem cells 3 Unique features of stem cells Distinguishing features of progenetor/precursor cells & Stem cells classification of stem cell Embryonic stem cell Adult stem cell Induced pluripotent stem cells Regenerative medicine & cell therapy Clinical applications
  • 3. Introduction • self-renewal • differentiation. • Self-renewal is the ability of cells to proliferate. • blank cell / precursor cell • building block of the human body
  • 4.
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  • 6.
  • 7. FeaturesofStemCells unique cells. amazing ability to developinto several distinct cell types Stem Cells can be used as a repair system for the body. divide without limit in order to replenish various types of cells. Whena stem cell divides - remain a stem cell or become another type of cell with a more specialized function (i.e. a muscle cell, a red blood cell, a brain cell, etc.).
  • 8. Threeuniquepropertiesofstemcells Stem cells are capable of dividing & renewing themselves for long periods
  • 9. Threeuniquepropertiesofstemcells A stem cell is "uncommitted," until it receives a signal to develop into a specialized cell
  • 10. Threeuniquepropertiesofstemcells Stem cells are unspecialized cells that develop into specialized cells (make up the different types of tissues in the human body).
  • 11.
  • 12. Distinguishing Features ofProgenitor/Precursor Cellsand StemCells. A stem cell is an unspecializedcell that develops into a variety of specialized cell types. A stem cell divides and gives rise to one additional stem cell and a specializedcell. Example: a hematopoietic stem cell produce a second generation stem cell and a neuron.
  • 13. Distinguishing Features ofProgenitor/Precursor Cellsand StemCells(Conti……..) A progenitor cell is unspecialized that is capable of undergoing cell division and yielding two specialized cells. Example: a myeloid progenitor/precursor cell undergoing cell division to yield two specialized cells (a neutrophil and a red blood cell).
  • 14.
  • 16. The potency of a stem cell is defined by the types of more differentiated cells that the stem cell can make. Stem cells can beeither: • totipotent, • pluripotent, • multipotent, or • unipotent.
  • 17. Totipotent cells have the capability to produce all cell types of the developing organism, including both embryonic and extraembryonic (eg, placenta) tissues. Totipotentstemcells
  • 18. Pluripotentcells Pluripotent cells can only make cells of the embryo proper, Can make all cells of the embryo (germ cells, cells from any of the germ layers. Therefore, they can make any cell of the body.
  • 19.
  • 20. Multipotentcells Multipotent cells can only make cells within a given germ layer. For example, multipotent stem cells from a mesodermal tissue like the blood can make all the cells of the blood, but cannot make cells of a different germ layer such as neural cells (ectoderm) or liver cells (endoderm).
  • 21.
  • 22. Unipotentcells Unipotent cells make cells of a single cell type. An example is a germ cell stem cell that makes the cells that mature to become egg or sperm, but not other cell types.
  • 23.
  • 24.
  • 25.
  • 26.
  • 27.
  • 28. Embryonicstemcells Embryonic stem cells are MOST UNDIFFIRENTIATED Derived from the inner cell mass of a blastocyst. LIMITLESS Cell renewal capacity Can give rise to every cell in the body (Therefore said TOTIPOTENT) Induced to form specialized cells of all 3 germ cell layers (neuron, cardiac muscle, liver cells, pancreatic islets cells
  • 29. Embryonic stemcells Human embryos reach the blastocyst stage 4–5 days post fertilization, at which time they consist of 50–150 cells. Human ES cells measure approximately 14 μm This would provide a radical new treatment approach to a wide variety of conditions where age, disease, or trauma has led to tissue damage or dysfunction.
  • 30.
  • 31.
  • 32.
  • 33.
  • 34.
  • 35.
  • 36. Advantagesof EmbryonicStem Cell 1. Flexible - appear to have the potential to makeany cell. 2.Immortal - one embryonic stem cell line can potentially provide an endless supply of cells with definedcharacteristics. 3. Availability - embryos from in vitro fertilization clinics.
  • 37. Disadvantages of Embryonicstemcell 1. Difficult to differentiate uniformly and homogeneously into a targettissue. 2. Immunogenic - embryonic stem cells from a random embryo donor are likely to be rejected after transplantation 3. Tumorigenic - capable of forming tumors or promoting tumor formation. 4. Destruction of developing human life.
  • 39. •Undifferentiated cell, found among differentiated cells (Stem cell niches). •The adult stem cell can renew itself and can differentiate to yield some or all of the major specialized cell types of the tissue or organ. •The primaryroles of adult stem cells in a living organismare to maintain and repair the tissue in which they are found. •Scientistsalso use the termsomaticstemcell instead of adult stem cell. ADULT STEM CELLS
  • 40. Differentiationpathwaysofadult stemcells Neural stem cells - in the brain give rise to its three major cell types: nerve cells (neurons) and two categories of non-neuronal cells —astrocytes and oligodendrocytes. Epithelial stem cells - lining of the digestive tract occur in deep crypts) - give rise to several cell types: absorptive cells, goblet cells, Paneth cells, and enteroendocrine cells. Skin stem cells - occur in the basal layer of the epidermis and at the base of hair follicles. The epidermal stem cells - give rise to keratinocytes The follicular stem cells - can give rise to both the hair follicle and to the epidermis
  • 41. Adult stem cells from bone marrow and umbilical cords appear to be as flexible asthe embryonic type Somewhat specialized – inducement may be simpler. Not immunogenic - recipients whoreceive the products of their own stemcells will not experience immunerejection. Relative ease of procurement - some adult stem cells are easy to harvest (skin, muscle, marrow, fat) Non-tumorigenic- tend not to form tumors. Noharm done to the donor. Advantages ofAdult StemCell
  • 42. Disadvantages ofAdult stem cells Limited quantity - can sometimes be difficult to obtain in large numbers. Finite - may not live as long as embryonic stem cells in culture. Less flexible - may be more difficult to reprogram to form other tissue types
  • 43. Whyare adult stemcells preferable to embryonic stem cells? • Adult stem cells are naturally exist in our bodies, and provide a natural repair mechanism for many tissues. • They belong in the microenvironment of an adult body, while embryonic stem cells belong in the microenvironment of the early embryo, where they tend to cause tumors and immune system reactions.
  • 44. Induced pluripotent stem cells • Induced pluripotent stem cells (iPSCs) are adult cells that have been genetically reprogrammed to an embryonic stem cell–like state. • Mouse iPSCs were first reported in 2006 • human iPSCs were first reported in late 2007. • Mouse iPSCs demonstrate important characteristics of pluripotent stem cells, including expressing stem cell markers. • Human iPSCs also express stem cell markers and are capable of generating cells characteristic of all three germ layers.
  • 45. (1)Isolate and culture donor cells. (2)Transduce stem cell-associated genes into the cells by viral vectors. Red cells indicate the cells expressing the exogenous genes. (3)Harvest and culture the cells according to ES cell culture. (4)A small subset of the transfected cells become iPS cells and generate ES-like colonies.
  • 46.
  • 47. SourceofStemcells from autologus, • Stem cells may be derived allogeneic or xenogenic sources. • Histocompatability is prerequisite for transplantation of allogeneic stem cells. • Fatal tissue is the best current tissue source for human neural stem cells, however ethical issues are a majorconcern.
  • 48. Potential sources of stem cells Fetal tissue that becomes available after an abortion Excess embryos from assisted reproductive technologies such as commonly used in fertility clinics Embryos created through in vitro fertilization specifically for research purpose Other sources of stem cells are those from umbilical cord blood, & bone marrow. In addition, neural stem cells, haematopoetic stem cells & mesenchymal stem cells can be harvested from fetal blood and fetal tissue.
  • 49. Placenta a Source of Stem Cells Placental stem cells, like umbilical cord blood and bone marrow stem cells, can be used to cure chronic blood-related disorders such as sickle cell disease, Thalasemia, and leukaemia.
  • 50. Umbilical Cord Blood Stem Cell Transplant Umbilical cord blood stem cell transplants are less prone to rejection than either bone marrow or peripheral blood stem cells. This is probably because the cells have not yet developed the features that can be recognized and attacked by the recipient's immune system.
  • 51. Applications • Stemcells can be used to studydevelopment • Stem cells may help us understand how a complex organism develops from a fertilised egg. • In the laboratory, scientists can follow stem cells as they divide and become increasingly specialized, making skin, bone, brain, and other cell types. Identifying the signals and mechanisms that determine whether a stem cell chooses to carry on replicating itself or differentiate into a specialized cell type, and into which cell type, will help us understand what controls normal development. • Some of the most serious medical conditions, such as cancer and birth defects, are due to abnormal cell division anddifferentiation. • 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. This is an important goal of stem cell research.
  • 52. Stem cells have the ability to replace damaged cells and treat disease •treatment of extensive burns, •to restore the blood system in patients with leukaemia and other blooddisorders. •Stem cells may also hold the key to replacing cells lost in many other devastating diseases •Stem cells, offer the possibility of a renewable source of replacement cells and tissues to treat diseases including Parkinson's, stroke, heart disease and diabetes.
  • 53. Stem cells could provide a resource for testing new medical treatments •New medications could be tested for safety on specialized cells. •Other kinds of cell lines are already usedin this way. •used to screen potential anti-tumourdrugs.
  • 54.
  • 55.
  • 56. Celltherapy. • Treatment of neural diseases such as Parkinson's disease, Huntington’s disease andAlzheimer's disease. Stem cells could be used to repair or replace damaged neurons. Repair of damaged organs such as the liver and pancreas. Treatments forAIDS.
  • 57. H o wDoesCellTherapyWork? Stem cells can be used to generate healthy and functioning specialized cells, which can then replace diseased or dysfunctional cells. It is similar to the process of organ transplantation only the treatment consists of transplanting cells instead of organs.
  • 58. How Does Cell TherapyWork? Bone marrow transplants are an example of cell therapy in which the stem cells in a donor's marrow are used to replace the blood cells of the victims of leukemia. Cell therapy is also being used in experiments to graft new skin cells to treat serious burn victims, and to grow newcorneas for the sight-impaired. In all of these uses, the goal is for the healthy cells to become integrated into the body and begin to function like the patient's owncells.