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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 engraftment...

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Dettagli Bibliografici
Pubblicato in:J Clin Invest
Autori principali: Hirschhorn, R, Roegner-Maniscalco, V, Kuritsky, L, Rosen, F S
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society for Clinical Investigation 1981
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Accesso online:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC370939/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/7033281/
https://ncbi.nlm.nih.govhttps://doi.org/10.1172/JCI110389
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