Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines
Author:
Publisher
Springer Science and Business Media LLC
Subject
Transplantation,Hematology
Link
http://www.nature.com/articles/1703839.pdf
Reference131 articles.
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2. Di Ferrante N, Nichols BL, Donnelly PV et al. Induced degradation of glycosaminoglycans in Hurler's and Hunter's syndromes by plasma infusion. Proc Natl Acad Sci USA 1971; 68: 303–307.
3. Knudson Jr AG, Di Ferrante N, Curtis JE . Effect of leukocyte transfusion in a child with type II mucopolysaccharidosis. Proc Natl Acad Sci USA 1971; 68: 1738–1741.
4. Hobbs JR, Hugh-Jones K, Barrett AJ et al. Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation. Lancet 1981; 2: 709–712.
5. Krivit W, Shapiro EG, Peters C et al. Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy. N Engl J Med 1998; 338: 1119–1126.
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