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STEM CELL
Four types 
EMBRYONIC STEM CELLS 
ADULT STEM CELLS 
PLACENTAL STEM CELLS 
(Cord Blood Stem Cells) 
AMNIOTIC FLUID STEM CELLS
Three general properties: 
they are capable of dividing themselves for long periods; 
they are unspecialized; 
and 
they can give rise to specialized cell types.
TOTIPOTENT/PLURIPOTENT 
MULTIPOTENT 
QUADRIPOTENT 
TRIPOTENT 
BIPOTENT 
UNIPOTENT
Multipotent hematopoietic 
Stem cell 
(hemocytoblast) 
Common myeloid 
progenitor 
Common Lymphoid 
progenitor 
Small lymphocyte 
B lymphocyte T lymphocyte 
Natural killer cell 
(Large granular Lymphocyte) 
Plasma cell 
Monocyte 
Macrophage 
Basophil Eosinophil 
Megakaryocyte 
Thrombocyte 
Erythrocyte Mast cell 
Mayeloblast 
Neutrophil
SCNT--CLONE
Fundamental properties of an Embryonic Stem Cell 
Derived from the inner cell mass/epiblast of the blastocyst. 
Capable of undergoing an unlimited number of symmetrical divisions 
without differentiating (long-term self-renewal). 
Exhibit and maintain a stable, full (diploid), 
normal complement of chromosomes (karyotype). 
Pluripotent ES cells can give rise to differentiated cell types 
that are derived from all three primary germ layers of the embryo 
(endoderm, mesoderm, and ectoderm). 
Capable of integrating into all fetal tissues during development. 
……………………
Capable of colonizing the germ line and giving rise to egg or sperm cells. 
Clonogenic, that is a single ES cell can give rise to a colony of genetically identical cells, 
or clones, which have the same properties as the original cell. 
Expresses the transcription factor Oct-4, 
Can be induced to continue proliferating or to differentiate. 
Lacks the G1 checkpoint in the cell cycle. 
ES cells spend most of their time in the S phase of the cell cycle, 
during which they synthesize DNA. Unlike differentiated somatic cells, 
ES cells do not require any external stimulus to initiate DNA replication. 
Do not show X inactivation. 
In every somatic cell of a female mammal, one of the two X chromosomes becomes 
permanently inactivated. X inactivation does not occur in undifferentiated ES cells.
Stem Cell Markers 
c-Kit 
Oct4 (ATGCAAAT) POU Family Protein 
CD34 
CD38 
Cd44 
CD133 
Nestin
Cells in suspension are tagged 
with fluorescent markers specific 
for undifferentiated stem cell 
Labeled cells are sent under 
pressure through a small nozzle 
and pass through an electric field 
A cell generates a negative charge 
if it fluoresces and a positive charge 
if it does not. 
FLUROSCENT 
ACTIVATED 
CELL SORTING 
Laser beam passes 
through one cell 
Stem cell 
SEPARATION OF STEM CELL
Diseases that are treated by stem cells are: 
1) Acute Leukemia 
• Acute Lymphoblast Leukemia (ALL) 
• Acute Myelogenous Leukemia (AML) 
• Acute Biphenotypic Leukemia 
• Acute Undifferentiated Leukemia 
2) Chronic Leukemia 
• Chronic Myelogenous Leukemia (CML) 
• Chronic Lymphocytic Leukemia (CLL) 
• Juvenile Chronic Myelogenous Leukemia (JCML) 
• Juvenile Myelomonocytic Leukemia (JMML) 
Syndromes 
• Myelodysplastic Syndromes 
• Amyloidosis 
• Chronic Myelomonocytic Leukemia (CMML) 
• Refractory Anemia (RA) 
• Refractory Anemia with Excess Blasts (RAEB) 
• Refractory Anemia with Excess Blasts in Transformation 
• (RAEB-T) 
• Refractory Anemia with Ringed Sideroblasts (RARS)
Disorders 
1) Stem Cell Disorders 
• Aplastic Anemia (Severe) 
• Fanconi Anemia 
• Paroxysmal Nocturnal Hemoglobinuria 
• Congenital Cytopenia 
• Dyskeratosis Congenita 
2) Myeloproliferative Disorders 
• Acute Myelofibrosis 
• Agnogenic Myeloid Metaplasia 
• Polycythemia Vera 
• Essential Thrombocythemia 
3) Lymphoproliferative Disorders 
• Non-Hodgkin’s Lymphoma 
• Hodgkin’s disease 
• Prolymphocytic Leukemia 
4) Phagocyte Disorders 
• Chediak-Higashi Syndrome 
• Chronic Granulomatous Disease 
• Neutrophil Actin Deficiency 
• Reticular Dysgenesis 
5) Inherited Metabolic Disorders 
• Mucopolysaccharidoses (MPS) 
• Hurler’s Syndrome (MPS-IH) 
• Scheie Syndrome (MPS-IS) 
• Hunter’s Syndrome (MPS-II) 
• Sanfilippo Syndrome (MPS-III) 
• Morquio Syndrome (MPS-IV) 
• Maroteaux-Lamy Syndrome (MPS-VI) 
• Sly Syndrome, Beta-Glucuronidase Deficiency 
• Adrenoleukodystrophy 
• Mucolipidosis II (I-cell Disease) 
• Krabbe Disease 
• Gaucher’s Disease 
• Niemann-Pick Disease 
• Wolman Disease 
• Metachromatic Leukodystrophy 
6) Histiocytic Disorders 
• Familial Erythrophagocytic Lymphohistiocytosis 
• Histiocytosis-X 
• Hemophagocytosis 
• Langerhans’ Cell Histiocytosis
7) Inherited Immune System Disorders 
• Ataxia-Telangiectasia 
• Kostmann Syndrome 
• Leukocyte Adhesion Deficiency 
• DiGeorge Syndrome 
• Bare Lymphocyte Syndrome 
• Omenn’s Syndrome 
• Severe Combined Immunodeficiency 
• SCID with Adenosine Deaminase Deficiency 
• Absence of T & B Cells SCID 
• Absence of T Cells, Normal B Cell SCID 
• Common Variable Immunodeficiency 
• Wiskott-Aldrich Syndrome 
• X-Linked Lymphoproliferative Disorder 
Other Inherited Disorders 
• Lesch-Nyhan Syndrome 
• Cartilage-Hair Hypoplasia 
• Glanzmann Thrombasthenia 
• Osteopetrosis 
• Adrenoleukodystrophy 
• Ceroid Lipofuscinosis 
• Congenital Erythropoietic Porphyria 
• Sandhoff Disease 
9) Plasma Cell Disorders 
• Multiple Myeloma 
• Plasma Cell Leukemia 
• Waldenstrom’s Macroglobulinemia 
• Amyloidosis 
Abnormalities 
1) Inherited Platelet Abnormalities 
Congenital Thrombocytopenia 
2) Inherited Erythrocyte Abnormalities 
• Beta Thalassemia Major 
• Sickle Cell Disease 
• Blackfan-Diamond Anemia 
• Pure Red Cell Aplasia 
Other Malignancies 
• Ewing Sarcoma 
• Neuroblastoma 
• Renal Cell Carcinoma 
• Retinoblastoma 
• Brain tumor 
• Ovarian Cancer 
• Small Cell Lung Cancer 
• Testicular Cancer

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stem cells

  • 2. Four types EMBRYONIC STEM CELLS ADULT STEM CELLS PLACENTAL STEM CELLS (Cord Blood Stem Cells) AMNIOTIC FLUID STEM CELLS
  • 3. Three general properties: they are capable of dividing themselves for long periods; they are unspecialized; and they can give rise to specialized cell types.
  • 4. TOTIPOTENT/PLURIPOTENT MULTIPOTENT QUADRIPOTENT TRIPOTENT BIPOTENT UNIPOTENT
  • 5. Multipotent hematopoietic Stem cell (hemocytoblast) Common myeloid progenitor Common Lymphoid progenitor Small lymphocyte B lymphocyte T lymphocyte Natural killer cell (Large granular Lymphocyte) Plasma cell Monocyte Macrophage Basophil Eosinophil Megakaryocyte Thrombocyte Erythrocyte Mast cell Mayeloblast Neutrophil
  • 7. Fundamental properties of an Embryonic Stem Cell Derived from the inner cell mass/epiblast of the blastocyst. Capable of undergoing an unlimited number of symmetrical divisions without differentiating (long-term self-renewal). Exhibit and maintain a stable, full (diploid), normal complement of chromosomes (karyotype). Pluripotent ES cells can give rise to differentiated cell types that are derived from all three primary germ layers of the embryo (endoderm, mesoderm, and ectoderm). Capable of integrating into all fetal tissues during development. ……………………
  • 8. Capable of colonizing the germ line and giving rise to egg or sperm cells. Clonogenic, that is a single ES cell can give rise to a colony of genetically identical cells, or clones, which have the same properties as the original cell. Expresses the transcription factor Oct-4, Can be induced to continue proliferating or to differentiate. Lacks the G1 checkpoint in the cell cycle. ES cells spend most of their time in the S phase of the cell cycle, during which they synthesize DNA. Unlike differentiated somatic cells, ES cells do not require any external stimulus to initiate DNA replication. Do not show X inactivation. In every somatic cell of a female mammal, one of the two X chromosomes becomes permanently inactivated. X inactivation does not occur in undifferentiated ES cells.
  • 9. Stem Cell Markers c-Kit Oct4 (ATGCAAAT) POU Family Protein CD34 CD38 Cd44 CD133 Nestin
  • 10. Cells in suspension are tagged with fluorescent markers specific for undifferentiated stem cell Labeled cells are sent under pressure through a small nozzle and pass through an electric field A cell generates a negative charge if it fluoresces and a positive charge if it does not. FLUROSCENT ACTIVATED CELL SORTING Laser beam passes through one cell Stem cell SEPARATION OF STEM CELL
  • 11.
  • 12. Diseases that are treated by stem cells are: 1) Acute Leukemia • Acute Lymphoblast Leukemia (ALL) • Acute Myelogenous Leukemia (AML) • Acute Biphenotypic Leukemia • Acute Undifferentiated Leukemia 2) Chronic Leukemia • Chronic Myelogenous Leukemia (CML) • Chronic Lymphocytic Leukemia (CLL) • Juvenile Chronic Myelogenous Leukemia (JCML) • Juvenile Myelomonocytic Leukemia (JMML) Syndromes • Myelodysplastic Syndromes • Amyloidosis • Chronic Myelomonocytic Leukemia (CMML) • Refractory Anemia (RA) • Refractory Anemia with Excess Blasts (RAEB) • Refractory Anemia with Excess Blasts in Transformation • (RAEB-T) • Refractory Anemia with Ringed Sideroblasts (RARS)
  • 13. Disorders 1) Stem Cell Disorders • Aplastic Anemia (Severe) • Fanconi Anemia • Paroxysmal Nocturnal Hemoglobinuria • Congenital Cytopenia • Dyskeratosis Congenita 2) Myeloproliferative Disorders • Acute Myelofibrosis • Agnogenic Myeloid Metaplasia • Polycythemia Vera • Essential Thrombocythemia 3) Lymphoproliferative Disorders • Non-Hodgkin’s Lymphoma • Hodgkin’s disease • Prolymphocytic Leukemia 4) Phagocyte Disorders • Chediak-Higashi Syndrome • Chronic Granulomatous Disease • Neutrophil Actin Deficiency • Reticular Dysgenesis 5) Inherited Metabolic Disorders • Mucopolysaccharidoses (MPS) • Hurler’s Syndrome (MPS-IH) • Scheie Syndrome (MPS-IS) • Hunter’s Syndrome (MPS-II) • Sanfilippo Syndrome (MPS-III) • Morquio Syndrome (MPS-IV) • Maroteaux-Lamy Syndrome (MPS-VI) • Sly Syndrome, Beta-Glucuronidase Deficiency • Adrenoleukodystrophy • Mucolipidosis II (I-cell Disease) • Krabbe Disease • Gaucher’s Disease • Niemann-Pick Disease • Wolman Disease • Metachromatic Leukodystrophy 6) Histiocytic Disorders • Familial Erythrophagocytic Lymphohistiocytosis • Histiocytosis-X • Hemophagocytosis • Langerhans’ Cell Histiocytosis
  • 14. 7) Inherited Immune System Disorders • Ataxia-Telangiectasia • Kostmann Syndrome • Leukocyte Adhesion Deficiency • DiGeorge Syndrome • Bare Lymphocyte Syndrome • Omenn’s Syndrome • Severe Combined Immunodeficiency • SCID with Adenosine Deaminase Deficiency • Absence of T & B Cells SCID • Absence of T Cells, Normal B Cell SCID • Common Variable Immunodeficiency • Wiskott-Aldrich Syndrome • X-Linked Lymphoproliferative Disorder Other Inherited Disorders • Lesch-Nyhan Syndrome • Cartilage-Hair Hypoplasia • Glanzmann Thrombasthenia • Osteopetrosis • Adrenoleukodystrophy • Ceroid Lipofuscinosis • Congenital Erythropoietic Porphyria • Sandhoff Disease 9) Plasma Cell Disorders • Multiple Myeloma • Plasma Cell Leukemia • Waldenstrom’s Macroglobulinemia • Amyloidosis Abnormalities 1) Inherited Platelet Abnormalities Congenital Thrombocytopenia 2) Inherited Erythrocyte Abnormalities • Beta Thalassemia Major • Sickle Cell Disease • Blackfan-Diamond Anemia • Pure Red Cell Aplasia Other Malignancies • Ewing Sarcoma • Neuroblastoma • Renal Cell Carcinoma • Retinoblastoma • Brain tumor • Ovarian Cancer • Small Cell Lung Cancer • Testicular Cancer