Effect of Leukocyte Transfusion in a Child with Type II Mucopolysaccharidosis

Treatment of a child affected by type II mucopolysaccharidosis (Hunter's syndrome) with leukocyte transfusions produced dramatic biochemical and clinical changes. The biochemical changes, consisting of greatly increased urinary excretion of glycosaminoglycans and their products of degradation,...

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Veröffentlicht in:Proceedings of the National Academy of Sciences - PNAS 1971-08, Vol.68 (8), p.1738-1741
Hauptverfasser: Knudson, Alfred G., Di Ferrante, Nicola, Curtis, John E.
Format: Artikel
Sprache:eng
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Zusammenfassung:Treatment of a child affected by type II mucopolysaccharidosis (Hunter's syndrome) with leukocyte transfusions produced dramatic biochemical and clinical changes. The biochemical changes, consisting of greatly increased urinary excretion of glycosaminoglycans and their products of degradation, were transient. The clinical changes, on the other hand, were protracted, which suggests that the mobilization and degradation of stored glycosaminoglycans was not followed by their reaccumulation to initial levels. The effects obtained with this treatment are superior to those produced by plasma infusion, and have implications for therapeutic attempts in the treatment of other hereditary diseases.
ISSN:0027-8424
1091-6490
DOI:10.1073/pnas.68.8.1738