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- W2091796284 abstract "Hematopoietic stem-cell transplantation is currently the most appropriate substitution therapy in the most severe forms of primary immunodeficiency diseases (all the variants of SCID, WA, CID etc.). It can achieve total and permanent immunological reconstitution in 60% of patients, depending on histocompatibility, source of the hematopoietic stem cells and the underlying disease. Stem-cell sources may be bone marrow, umbilical cord blood and the peripheral blood of donors previously treated with colony stimulating factors for the mobilization CD34. We discuss the differences in the results obtained in patients treated at the Hospital Materno-Infantil Vall d'Hebron. Gene therapy opens a new era in the treatment of primary immunodeficiency diseases. The first patient to undergo this treatment in the United States of America had adenosine-deaminase deficiency, even though sustained remodeling has not been achieved. The favorable results obtained in patients with SCID by deficit in the gamma chain of the IL-2 receptor in Paris, with more than a year of follow up, suggest that the near future is promising. We also discuss the differences observed according to the vectors used and the underlying disease." @default.
- W2091796284 created "2016-06-24" @default.
- W2091796284 creator A5078353652 @default.
- W2091796284 date "2001-01-01" @default.
- W2091796284 modified "2023-09-27" @default.
- W2091796284 title "Tratamiento sustitutivo con progenitores hemopoyéticos en las inmunodeficiencias primarias" @default.
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- W2091796284 doi "https://doi.org/10.1016/s0301-0546(01)79030-1" @default.
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