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Progression & Success of Cell and Gene Based Therapies


Clinical progress in gene therapy over the last several years includes successful treatment of patients with the retinal disease, severe combined immunodeficiency, chronic myelogenous leukemia, Lipoprotein lipase deficiency and Parkinson's disease.

Scientists at the National Institutes of Health successfully treated metastatic melanoma in two patients using killer T cells genetically retargeted to attack the cancer cells. This study constitutes one of the first demonstrations that gene therapy can be effective in treating cancer.

Stem Cells are being used today to help people suffering from dozens of diseases and conditions, including:


  • Brain Cancer
  • Retinoblastoma
  • Ovarian Cancer
  • Skin Cancer: Merkel Cell Carcinoma
  • Testicular Cancer
  • Tumors abdominal organs Lymphoma
  • Non-Hodgkin's lymphoma
  • Hodgkin's Lymphoma
  • Acute Lymphoblastic Leukemia
  • Acute Myelogenous Leukemia
  • Chronic Myelogenous Leukemia
  • Juvenile Myelomonocytic Leukemia
  • Chronic Myelomonocytic Leukemia
  • Cancer of the lymph nodes: Angioimmunoblastic Lymphadenopathy
  • Multiple Myeloma
  • Myelodysplasia
  • Breast Cancer
  • Neuroblastoma
  • Renal Cell Carcinoma
  • Various Solid Tumors
  • Soft Tissue Sarcoma
  • Ewing's Sarcoma
  • Waldenstrom's macroglobulinemia
  • Hemophagocytic lymphohistiocytosis
  • POEMS syndrome
  • Myelofibrosis

Auto-Immune Diseases:

  • Diabetes Type I (Juvenile)
  • Systemic Lupus
  • Sjogren's Syndrome
  • Myasthenia
  • Autoimmune Cytopenia
  • Scleromyxedema
  • Scleroderma
  • Crohn's Disease
  • Behcet's Disease
  • Rheumatoid Arthritis
  • Juvenile Arthritis
  • Multiple Sclerosis
  • Polychondritis
  • Systemic Vasculitis
  • Alopecia Universalis
  • Buerger's Disease


  • Acute Heart Damage
  • Chronic Coronary Artery Disease


  • Corneal regeneration


  • Severe Combined Immunodeficiency Syndrome
  • X-linked Lymphoproliferative Syndrome
  • X-linked Hyper immunoglobulin M Syndrome

Neural Degenerative Diseases and Injuries:

  • Parkinson's Disease
  • Spinal Cord Injury
  • Stroke Damage

Anemias and Other Blood Conditions:

  • Sickle Cell Anemia
  • Sideroblastic Anemia
  • Aplastic Anemia
  • Red Cell Aplasia
  • Amegakaryocytic Thrombocytopenia
  • Thalassemia
  • Primary Amyloidosis
  • Diamond Blackfan Anemia
  • Fanconi's Anemia
  • Chronic Epstein-Barr Infection

Wounds and Injuries:

  • Limb Gangrene
  • Surface Wound Healing
  • Jawbone Replacement
  • Skull Bone Repair

Other Metabolic Disorders:

  • Hurler's Syndrome
  • Osteogenesis Imperfecta
  • Krabbe Leukodystrophy
  • Osteopetrosis
  • Cerebral X-Linked Adrenoleukodystrophy

Liver Disease:

  • Chronic Liver Failure
  • Liver Cirrhosis

Bladder Disease:

  • End-Stage Bladder Disease

Nevertheless, before stem-cell therapeutics can be applied more effectively in the clinical setting, more research is necessary to understand stem-cell behavior upon transplantation as well as the mechanisms of stem-cell interaction with the diseased/injured microenvironment. Sufficient funding of this research will help facilitate treatments and cures.

Help support our mission by clicking here to donate to the Cells Cure Foundation.

The Cells Cure Foundation is a registered 501 (c) (3) not for profit organization.
All donations are tax deductible to the full extent of the law.


Stem Cells

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Thank you for considering a donation to the Cells Cure Foundation. Your support will help us fulfill our mission to fund research and development of cell and gene based therapies to cure diseases and disabilities, with minimal risk of rejection and side effects.

The Cells Cure Foundation is a registered 501 (c) (3) not for profit organization. All donations are tax deductible to the full extent of the law.