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Medical Uses

The following conditions are currently being treated with stem cells. Cord Blood derived stem cells are an area of intense ongoing research. The list of diseases/conditions treated with stem cells is growing daily. Any disease/condition which requires the regeneration of tissue (due to injury or disease) is a potential candidate for stem cell therapy.

Hemoglobinopathies/Blood Disorders:

Sickle-cell disease

b-thalassemia (Cooley’s anemia)

Diamond-Blackfan Anemia

Severe Aplastic Anemia

Pure Red Cell Aplasia

Congenital Cytopenia

Dyskeratosis Congenita

Fanconi Anemia

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Refractory Anemia (RA)

Refractory Anemia with Excess Blasts (RAEB)

Refractory Anemia with Excess Blasts in Transformation (RAEB-T)

Refractory Anemia with Ringed Sideroblasts (RARS)


Metabolic Disorders:


Batten disease (inherited neuronal ceroid lipofuscinosis)

Gunther disease

Hunter syndrome

Hurler syndrome

Krabbe disease (globoid cell leukodystrophy)

Lesch-Nyhan disease

Maroteaux-Lamy syndrome

Tay-Sachs disease

Sanfilippo Syndrome

Metachromatic Leukodystrophy

Alpha Mannosidosis

Gaucher’s Disease

Maroteaux-Lamy Syndrome (MPS-VI)

Morquio Syndrome (MPS-IV)

Mucolipidosis II (I-cell Disease)

Mucopolysaccharidoses (MPS)

Niemann-Pick Disease

Scheie Syndrome (MPS-IS)

Sly Syndrome

Beta-Glucuronidase Deficiency (MPS-VII)

Wolman Disease


Chronic granulomatous disease

Common variable immune deficiency (CVID)

Omenn’s syndrome

Severe combined immune deficiency (SCID and SCID-ADA)

Reticular dysgenesis

Thymic dysplasia

Wiskott-Aldrich syndrome

X-linked lymphoproliferative disease

Bare lymphocyte syndrome (MHC class II deficiency)

Leukocyte adhesion deficiency

Hystiocytic disorders

Familial Erythrophagocytic Lymphohistiocytosis



Langerhans’ Cell Histiocytosis

Chediak-Higashi Syndrome

Chronic Granulomatous Disease

Neutrophil Actin Deficiency

Reticular Dysgenesis

Congenital (Inherited) Immune System Disorders

Absence of T and B Cells SCID

Absence of T Cells, Normal B Cell SCID


Bare Lymphocyte Syndrome

Common Variable Immunodeficiency

DiGeorge Syndrome

Kostmann Syndrome

Leukocyte Adhesion Deficiency

Omenn’s Syndrome

SCID with Adenosine Deaminase Deficiency

X-Linked Lymphoproliferative Disorder



Acute Leukemias: Acute Biphenotypic Leukemia, Acute Lymphocytic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Acute Undifferentiated Leukemia

Chronic Leukemias: Chronic Lymphocytic Leukemia (CLL), Chronic Myelogenous Leukemia (CML), Juvenile Chronic Myelogenous Leukemia (JCML), Juvenile Myelomonocytic Leukemia (JMML)

Myelodysplastic Syndromes


Chronic Myelomonocytic Leukemia (CMML)

Hodgkin’s Disease

Non-Hodgkin’s Lymphoma

Prolymphocytic Leukemia

High risk solid tumors

Brain Tumors

Ewing Sarcoma


Ovarian Cancer

Renal Cell Carcinoma

Small-Cell Lung Cancer

Testicular Cancer


Other Disorders:Cartilage-Hair Hypoplasia, Ceroid Lipofuscinosis, Congenital Erythropoietic Porphyria, Glanzmann Thrombasthenia, Lesch-Nyhan Syndrome, Sandhoff Disease, Amegakaryocytosis / Congenital Thrombocytopenia, Evans syndrome, Familial erythrophagocytic hemophagocytic, Langerhans cell histiocytosis



Emerging Stem Cell Applications*

Alzheimer’s Disease


Heart Disease

Liver Disease

Muscular Dystrophy

Parkinson’s Disease

Spinal Cord Injury


Multiple Sclerosis

Rheumatoid Arthritis

Systemic Lupus Erythematosus


* Chen, Jieli, et al. Expression of neural markers in human umbilical cord blood. Experimental Neurology.2001;171:109-115

Pessina, A., et al. Pancreas developing markers expressed on human mononucleated umbilical cord blood cells. Biochemical and Biophysical Research Communications.2004;323:315-322

Botta, Rosanna, et al. Heart infarct in NOD-SCID mice: Therapeutic vasculogenesis by transplantation of human CD34+ cells and low dose CD34+KDR+cells. The FASEB Journal.2004;18:1392-1394

Perin, E., et al. Transendocardial, autologous bone marrow cell transplantation for severe, chronic ischemic heart failure. Circulation.2003;107:2294-2302

Campli, C., et al. A human umbilical cord stem cell rescue therapy in a murine model of toxic liver injury. Digestive and Liver Disease.2004;36:603-613

Gussoni, E., et al. Long term persistence of donor nuclei in a duchenne muscular dystrophy patient receiving bone marrow transplantation. The Journal of Clinical Investigation.2002;110(6):807-814

Saporta, Samuel, et al. Human umbilical cord blood stem cells infusion in spinal cord injury: Engraftment and beneficial influence on behavior. Journal of Hematotherapy & Stem Cell Research.2003;12:271-278

Vendrame, Martina, et al. Infusion of human umbilical cord blood cells in a rat model of stroke dose-dependently rescues behavioral deficits and reduces infarct volume. Stroke.2004;35:2390-2395.

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