Abstract
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.
Keywords: Hunter's syndrome, glycosaminoglycans degradation
Full text
PDF
Selected References
These references are in PubMed. This may not be the complete list of references from this article.
- Cox R. P., Krauss M. R., Balis M. E., Dancis J. Evidence for transfer of enzyme product as the basis of metabolic cooperation between tissue culture fibroblasts of Lesch-Nyhan disease and normal cells. Proc Natl Acad Sci U S A. 1970 Nov;67(3):1573–1579. doi: 10.1073/pnas.67.3.1573. [DOI] [PMC free article] [PubMed] [Google Scholar]
- Curtis J. E., Hersh E. M., Freireich E. J. Antigen-specific immunity in recipients of leukocyte transfusions from immune donors. Cancer Res. 1970 Dec;30(12):2921–2929. [PubMed] [Google Scholar]
- Danes B. S., Bearn A. G. Hurler's syndrome: a genetic study of clones in cell culture with particular reference to the Lyon hypothesis. J Exp Med. 1967 Sep 1;126(3):509–522. doi: 10.1084/jem.126.3.509. [DOI] [PMC free article] [PubMed] [Google Scholar]
- Di Ferrante N., Nichols B. L., Donnelly P. V., Neri G., Hrgovcic R., Berglund R. K. Induced degradation of glycosaminoglycans in Hurler's and Hunter's syndromes by plasma infusion. Proc Natl Acad Sci U S A. 1971 Feb;68(2):303–307. doi: 10.1073/pnas.68.2.303. [DOI] [PMC free article] [PubMed] [Google Scholar]
- Elves M. W. Comparison of mitomycin C and x-rays for the production of one-way stimulation in mixed leucocyte cultures. Nature. 1969 Jul 5;223(5201):90–91. doi: 10.1038/223090a0. [DOI] [PubMed] [Google Scholar]
- Fratantoni J. C., Hall C. W., Neufeld E. F. The defect in Hurler's and Hunter's syndromes: faulty degradation of mucopolysaccharide. Proc Natl Acad Sci U S A. 1968 Jun;60(2):699–706. doi: 10.1073/pnas.60.2.699. [DOI] [PMC free article] [PubMed] [Google Scholar]
- Freireich E. J., Judson G., Levin R. H. Separation and collection of leukocytes. Cancer Res. 1965 Oct;25(9):1516–1520. [PubMed] [Google Scholar]
- Hors-Cayla M. C., Maroteaux P., de Grouchy J. Fibroblastes en culture au cours de mucopolysaccharidoses: influence du sérum sur la métachromasie. Ann Genet. 1968 Dec;11(4):265–266. [PubMed] [Google Scholar]
- LAGUNOFF D., WARREN G. Determination of 2-deoxy-2-sulfoaminohexose content of mucopolysaccharides. Arch Biochem Biophys. 1962 Dec;99:396–400. doi: 10.1016/0003-9861(62)90285-0. [DOI] [PubMed] [Google Scholar]
- MUIR H., MITTWOCH U., BITTER T. THE DIAGNOSTIC VALUE OF ISOLATED URINARY MUCOPOLYSACCHARIDES AND OF LYMPHOCYTE INCLUSIONS IN GARGOYLISM. Arch Dis Child. 1963 Aug;38:358–363. doi: 10.1136/adc.38.200.358. [DOI] [PMC free article] [PubMed] [Google Scholar]
- Mapes C. A., Anderson R. L., Sweeley C. C., Desnick R. J., Krivit W. Enzyme replacement in Fabry's disease, an inborn error of metabolism. Science. 1970 Sep 4;169(3949):987–989. doi: 10.1126/science.169.3949.987. [DOI] [PubMed] [Google Scholar]
- Neufeld E. F., Fratantoni J. C. Inborn errors of mucopolysaccharide metabolism. Science. 1970 Jul 10;169(3941):141–146. doi: 10.1126/science.169.3941.141. [DOI] [PubMed] [Google Scholar]