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Primary Immunodeficiency Diseases

Pluripotent stem cells could be used in treatment of virtually all primary immunodeficiency diseases. Presently, there are more than 70 different forms of congenital and inherited deficiencies of the immune system that have been recognized. These are among the most complicated diseases to treat with the worst prognoses. Included here are diseases such as severe combined immunodeficiency disease (the “bubble boy” disease), Wiskott-Aldrich Syndrome, and the auto-immune disease lupus. The immune deficiencies suffered as a result of acquired immune deficiency syndrome (AIDS) following infection with the human immunodeficiency virus are also relevant here.http://www.niaid.nih.gov/publications/pid/contents.htm  http://www.nih.gov/grants/guide/1997/97.03.21/rfp-rfa-primary-immu11.html. These diseases are characterized by an unusual susceptibility to infection and often associated with anemia, arthritis, diarrhea, and selected malignancies. However, the transplantation of stem cells reconstituted with the normal gene could result in restoration of immune function and effective normalization of life span and quality of life for these people.

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