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Stem Cell Glossary of Terms

Stem cells are amazing cells that have the unique ability to develop into virtually any of the 220 types of cells found in the human body.  Kidney cells, heart cells, liver cells and brain cells are just some of the cell types that scientists have already been able to develop (“differentiate”) from stem cells.  Stem cells are found at all stages of human development and play a vital role in developing, maintaining and repairing the human body. There are two main types of stem cells, embryonic stem cells, obtained from the early embryo, and adult stem cells which can be found in organs and tissues throughout the human body. Embryonic stem cells are capable of differentiating into all the specialized cells in the human body, while adult stem cells are simply immature versions of the cells normally found in the tissue from which they were removed and so are usually only able to differentiate into mature cells of the same or similar type.

Most couples who have surplus frozen embryos after completing their family choose to discard them, but recently many couples have not felt comfortable with this option and instead choose to donate them to stem cell research so that these embryos can be utilized to help others. Fertility centers then provide them to stem cell research laboratories where embryonic stem cells are isolated from embryos that have been grown for approximately a week.  These stem cells continue to grow and divide for long periods of time and are available for further study. For example, a scientist might place the stem cells in conditions that cause them to form neural (brain) cells with the goal of someday using them a source of cells to treat Alzheimer’s disease.

Recently, techniques have been developed that allow scientists to develop stem cells that can be used to better understand specific diseases with the goal of some day using this knowledge to develop better treatments for those diseases.  In this groundbreaking procedure, called “nuclear transfer,” the genetic material (DNA) of an unfertilized egg is removed and replaced with the genetic material from a single skin cell of a patient with a severe disease to permit the generation of stem cells for further study.

Probably, the greatest potential for stem cells is in their use to treat degenerative diseases and major traumatic injuries, which may result in a significant improvement in the quality and length of life for affected patients.  Stem cells could be developed into healthy versions of the cells that have been lost or that are not functioning correctly in that particular disease or condition.  These stem cells would then serve as renewable sources for the cells and tissue needed for transplantation into patients.  The ability to replace defective or damaged cells through cell replacement therapy could allow the treatment of injuries and various genetic and degenerative conditions.  The list of diseases that may be good candidates for stem cell repair include muscular dystrophies, retinal degeneration, Alzheimer's disease, Parkinson's disease, arthritis, diabetes, spinal cord injuries, and blood disorders such as hemophilia, but as can be seen in the table below, many other diseases may turn out to be excellent targets as well.

Human Stem
Cell Overview

Potential Stem Cell Targets

General Overview:

  • Alzheimer’s Disease
  • Birth defects
  • Burns
  • Cardiac Disease
  • Craniofacial (cranium and face) abnormalities
  • Diabetes
  • Infertility Gametogenesis (Spermatogenesis, Ovogenesis)
  • Juvenile Diabetes (type I diabetes
  • Liver Disease
  • Liver disease
  • Lupus
  • Macular degeneration
  • Motor Neuron Disease
  • Multiple Sclerosis
  • Muscular Dystrophy
  • Osteoporosis
  • Parkinson’s Disease
  • Pulmonary disease
  • Pulmonary disease
  • Rheumatoid Arthritis
  • Spinal Cord Injury

Muscular Dystrophies:

  • Duchenne Muscular Dystrophy (DMD)
       (Also known as Pseudohyper
  • Becker Muscular Dystrophy (BMD)
  • Emery-Dreifuss Muscular Dystrophy (EDMD)
  • Limb-Girdle Muscular Dystrophy (LGMD)
  • Facioscapulohumeral Muscular Dystrophy (FSH or FSHD)
       (Also known as Landouzy-Dejerine)
  • Myotonic Dystrophy (MMD)
       (Also known as Steinert's Disease)
  • Oculopharyngeal Muscular Dystrophy (OPMD)
  • Distal Muscular Dystrophy (DD) (Miyoshi)
  • Congenital Muscular Dystrophy (CMD)

Motor Nueron Diseases:

  • Amyotrophic Lateral Sclerosis (ALS)
    (Also known as Lou Gehrig's Disease)
  • Infantile Progressive Spinal Muscular Atrophy (SMA, SMA1 or WH)
    (Also known as SMA Type 1, Werdnig-Hoffman)
  • Intermediate Spinal Muscular Atrophy (SMA or SMA2)
    (Also known as SMA Type 2)
  • Juvenile Spinal Muscular Atrophy (SMA, SMA3 or KW)
    (Also known as SMA Type 3, Kugelberg-Welander)
  • Spinal Bulbar Muscular Atrophy (SBMA)
    (Also known as Kennedy's Disease and X-Linked SBMA)
  • Adult Spinal Muscular Atrophy
    (SMA - Adult)

Inflammatory Myopathies:

  • Dermatomyositis (DM)
  • Polymyositis (PM)
  • Inclusion Body Myositis (IBM)

DISEASES OF THE NEUROMUSCULAR JUNCTION:

  • Myasthenia Gravis (MG)
  • Lambert-Eaton Syndrome (LES)
  • Congenital Myasthenic Syndrome (CMS)

MYOPATHIES DUE TO ENDOCRINE ABNORMALITIES:

  • Hyperthyroid Myopathy (HYPTM)
  • Hypothyroid Myopathy (HYPOTM)

OTHER MYOPATHIES:

  • Myotonia Congenita (MC)
    (Two forms: Thomsen's and Becker's Disease)
  • Myotonia Congenita (MC)
    (Two forms: Thomsen's and Becker's Disease)
    Paramyotonia Congenita (PC)
  • Central Core Disease (CCD)
  • Nemaline Myopathy (NM)
  • Myotubular Myopathy (MTM or MM)
  • Periodic Paralysis (PP)
    (Two forms: Hypokalemic and Hyperkalemic)

 

METABOLIC DISEASES OF MUSCLE:

  • Phosphorylase Deficiency (MPD or PYGM)
    (Also known as McArdle's Disease)
  • Acid Maltase Deficiency (AMD)
    (Also known as Pompe's Disease)
  • Phosphofructokinase Deficiency (PFKM)
    (Also known as Tarui's Disease)
  • Debrancher Enzyme Deficiency (DBD)
    (Also known as Cori's or Forbes' Disease)
  • Mitochondrial Myopathy (MITO)
  • Carnitine Deficiency (CD)
  • Carnitine Palmityl Transferase Deficiency (CPT)
  • Phosphoglycerate Kinase Deficiency (PGK)
  • Phosphoglycerate Mutase Deficiency (PGAM or PGAMM)
  • Lactate Dehydrogenase Deficiency (LDHA)
  • Lactate Dehydrogenase Deficiency (LDHA)

DISEASES OF PERIPHERAL NERVE:

  • Charcot-Marie-Tooth Disease (CMT)
    (Also known as Hereditary Motor and Sensory Neuropathy (HMSN) or Peroneal Muscular Atrophy (PMA))
  • Friedreich's Ataxia (FA)
  • Dejerine-Sottas Disease (DS)
    (Also known as CMT Type 3 or Progressive Hypertrophic Interstitial Neuropathy)

DISEASES OF THE NEUROMUSCULAR JUNCTION:

  • Myasthenia Gravis (MG)
  • Lambert-Eaton Syndrome (LES)
  • Congenital Myasthenic Syndrome (CMS)

Autoimmune Diseases:

  • Autoimmune hemolytic anemia
  • Autoimmune thrombocytopenic purpura
  • Goodpasture's syndrome
  • Glomerulonephritis,
  • Graves' disease
  • Hashimoto's thyroiditis
  • Hypoglycemia
  • Insulin-resistant diabetes
  • Myasthenia gravis
  • Pemphigus vulgaris
  • Epidermal cadherin
  • Pernicious anemia
  • Rheumatic fever
  • Spontaneous infertility

 

T-cell mediated diseases:

  • Experimental autoimmune encephalomyelitis (EAE),
  • Multiple sclerosis (MS)
  • Hashimoto's thyroiditis
  • Insulin-dependent (Type I) diabetes mellitus (IDDM)
  • Rheumatoid arthritis

Immune Complex Diseases:

  • Ankylosing spondylitis
  • Mixed essential cryoglobulinemia
  • Rheumatoid arthritis
  • Systemic lupus erythematosus (SLE)

Myelodysplastic Syndromes:

  • Refractory Anemia (RA)
  • Refractory Anemia with Ringed Sideroblasts (RARS)
  • Refractory Anemia with Excess Blasts (RAEB)
  • Refractory Anemia with Excess Blasts in Transformation (RAEB-T)
  • Chronic Myelomonocytic Leukemia (CMML)

Stem cell disorders:

  • Aplastic Anemia (Severe)
  • Fanconi Anemia
  • Paroxysmal Nocturnal Hemoglobinuria (PNH)
  • Pure Red Cell Aplasia

Myeloproliferative Disorders:

  • Acute Myelofibrosis
  • Agnogenic Myeloid Metaplasia (myelofibrosis)
  • Polycythemia Vera
  • Essential Thrombocythemia

Phagocyte Disorders:

  • Chediak-Higashi Syndrome
  • Chronic Granulomatous Disease
  • Neutrophil Actin Deficiency
  • Reticular Dysgenesis

other inherited disorders:

  • Lesch-Nyhan Syndrome
  • Cartilage-Hair Hypoplasia
  • Glanzmann Thrombasthenia
  • Osteopetrosis
  • Adrenoleukodystrophy

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 (MPS-VII)
  • Adrenoleukodystrophy
  • Mucolipidosis II (I-cell Disease)
  • Krabbe Disease Gaucher's Disease
  • Niemann-Pick Disease
  • Wolman Disease
  • Metachromatic Leukodystrophy

histiocytic disorders:

  • Familial Erythrophagocytic Lymphohistiocytosis
  • Histiocytosis-X
  • Hemophagocytosis

Inherited Platelet Abnormalities:

  • Amegakaryocytosis / Congenital Thrombocytopenia

Inherited Erythrocyte Abnormalities:

  • Beta Thalassemia Major
  • Sickle Cell Disease

Inherited immune system disorders:

  • Ataxia-Telangiectasia
  • Kostmann Syndrome
  • Leukocyte Adhesion Deficiency
  • DiGeorge Syndrome
  • Bare Lymphocyte Syndrome
  • Omenn's Syndrome
  • Severe Combined Immunodeficiency (SCID)
  • 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
    

Leukemias and Lymphomas, including:

acute leukemias:

  • Acute myelogenous leukemia (AML)
  • Acute lymphoblastic leukemia (ALL)
  • Chronic myelogenous leukemia
  • Acute Biphenotypic Leukemia
  • Acute Undifferentiated Leukemia

chronic leukemias:

  • Chronic Myelogenous Leukemia (CML)
  • Chronic Lymphocytic Leukemia (CLL)
  • Juvenile Chronic Myelogenous Leukemia (JCML)
  • Juvenile Myelomonocytic Leukemia (JMML)

Lymphoproliferative Disorders:

  • Hodgkin's lymphoma
  • Non-Hodgkin's lymphoma

Severe aplastic anemia and other marrow failure states, including:

  • Severe aplastic anemia
  • Fanconi anemia
  • Paroxysmal nocturnal hemoglobinuria (PNH)
  • Pure red cell aplasia
  • Amegakaryocytosis / congenital thrombocytopenia

SCID and other inherited immune system disorders, including:

  • Severe combined immunodeficiency (SCID, all sub-types)
  • Wiskott-Aldrich syndrome

Hemoglobinopathies, including:

  • Beta thalassemia major
  • Sickle cell disease

Hurler's syndrome and other inherited metabolic disorders, including:

  • Hurler's syndrome (MPS-IH)
  • Adrenoleukodystrophy
  • Metachromatic leukodystrophy

Myelodysplastic and myeloproliferative disorders, including:

  • Refractory anemia (all types)
  • Chronic myelomonocytic leukemia
  • Agnogenic myeloid metaplasia (myelofibrosis)
 

Familial Erythrophagocytic Lymphohistiocytosis and Other Histiocytic Disorders:

Plasma Cell Disorder:

  • Multiple Myeloma
  • Plasma Cell Leukemia
  • Waldenstrom's Macroglobulinemia
  • Amyloidosis

Other malignancies:

  • Ewing Sarcoma
  • Neuroblastoma
  • Renal Cell Carcinoma
  • Retinoblastoma
  
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