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.
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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