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References
Tolar J, Petryk A, Khan K, Bjoraker KJ, Jessurun J, Dolan M et al. Long-term metabolic, endocrine, and neuropsychological outcome of hematopoietic cell transplantation for Wolman disease. Bone Marrow Transplant 2009; 43: 21–27.
Krivit W, Peters C, Dusenbery K, Ben-Yoseph Y, Ramsay NK, Wagner JE et al. Wolman disease successfully treated by bone marrow transplantation. Bone Marrow Transplant 2000; 26: 567–570.
Stein J, Garty BZ, Dror Y, Fenig E, Zeigler M, Yaniv I . Successful treatment of Wolman disease by unrelated umbilical cord blood transplantation. Eur J Pediatr 2007; 166: 663–666.
Bou-Gharios G, Adams G, Pace P, Warden P, Olsen I . Correction of a lysosomal deficiency by contact-mediated enzyme transfer after bone marrow transplantation. Transplantation 1993; 56: 991–996.
Olsen I, Dean MF, Harris G, Muir H . Direct transfer of a lysosomal enzyme from lymphoid cells to deficient fibroblasts. Nature 1981; 291: 244–247.
Hong C, Sutherland DE, Matas AJ, Najarian JS . Bone marrow transplantation for correction of enzyme deficiency disease. Transplant Proc 1979; 11: 498–503.
Rappeport JM, Ginns EI . Bone-marrow transplantation in severe Gaucher's disease. N Engl J Med 1984; 311: 84–88.
Resnick JM, Krivit W, Snover DC, Kersey JH, Ramsay NK, Blazar BR et al. Pathology of the liver in mucopolysaccharidosis: light and electron microscopic assessment before and after bone marrow transplantation. Bone Marrow Transplant 1992; 10: 273–280.
Krivit W, Freese D, Chan KW, Kulkarni R . Wolman's disease: a review of treatment with bone marrow transplantation and considerations for the future. Bone Marrow Transplant 1992; 10 (Suppl 1): 97–101.
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Gramatges, M., Dvorak, C., Regula, D. et al. Pathological evidence of Wolman's disease following hematopoietic stem cell transplantation despite correction of lysosomal acid lipase activity. Bone Marrow Transplant 44, 449–450 (2009). https://doi.org/10.1038/bmt.2009.57
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DOI: https://doi.org/10.1038/bmt.2009.57
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