Successful allogeneic bone marrow transplant for Niemann–Pick disease type C2 is likely to be associ...
Successful allogeneic bone marrow transplant for Niemann–Pick disease type C2 is likely to be associated with a severe ‘graft versus substrate’ effect
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Dordrecht: Springer Netherlands
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Language
English
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Dordrecht: Springer Netherlands
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Contents
Niemann–Pick disease type C2 (NPC2) is caused by the inherited deficiency of a lysosomal cholesterol transport protein, NPC2 protein. Many cases of NPC2 present in early infancy with inflammatory lung disease, with subsequent severe neurological disease and death in early childhood. This disease is theoretically correctable by bone marrow transplan...
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Full title
Successful allogeneic bone marrow transplant for Niemann–Pick disease type C2 is likely to be associated with a severe ‘graft versus substrate’ effect
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TN_cdi_proquest_journals_1461754112
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_1461754112
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ISSN
1573-2665,0141-8955
E-ISSN
1573-2665
DOI
10.1007/s10545-010-9060-3
