Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome
Author:
Publisher
Springer Science and Business Media LLC
Subject
Transplantation,Hematology
Link
http://www.nature.com/articles/1705934.pdf
Reference31 articles.
1. Bach G, Friedman R, Weissmann B, Neufeld EF . The defect in the Hurler and Scheie syndromes: deficiency of -L-iduronidase. Proc Natl Acad Sci USA 1972; 69: 2048–2051.
2. De Duve C . From cytases to lysosomes. Fed Proc 1964; 23: 1045–1049.
3. Boelens JJ . Trends in haematopoietic cell transplantation for inborn errors of metabolism. J Inherit Metab Dis 2006; 29: 413–420.
4. Fratantoni JC, Hall CW, Neufeld EF . Hurler and Hunter syndromes: mutual correction of the defect in cultured fibroblasts. Science 1968; 162: 570–572.
5. Fratantoni JC, Hall CW, Neufeld EF . The defect in Hurler's and Hunter's syndromes: faulty degradation of mucopolysaccharide. Proc Natl Acad Sci USA 1968; 60: 699–706.
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