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Bone marrow transplantation only partially restores purine metabolites to normal in adenosine deaminase-deficient patients.

To delineate the extent to which bone marrow transplantation provides "enzyme replacement therapy", we have determined metabolite concentrations in two patients with adenosine deaminase (ADA) deficiency treated with bone marrow transplants and rendered immunologically normal. 10 yr after e...

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Autors principals: Hirschhorn, R, Roegner-Maniscalco, V, Kuritsky, L, Rosen, F S
Format: Artigo
Idioma:Inglês
Publicat: 1981
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC370939/
https://ncbi.nlm.nih.gov/pubmed/7033281
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