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Defective B cell tolerance in adenosine deaminase deficiency is corrected by gene therapy

Adenosine deaminase (ADA) gene defects are among the most common causes of SCID. Restoration of purine metabolism and immune functions can be achieved by enzyme replacement therapy, or more effectively by bone marrow transplant or HSC gene therapy (HSC-GT). However, autoimmune complications and auto...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: Sauer, Aisha V., Morbach, Henner, Brigida, Immacolata, Ng, Yen-Shing, Aiuti, Alessandro, Meffre, Eric
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society for Clinical Investigation 2012
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3366410/
https://ncbi.nlm.nih.gov/pubmed/22622038
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI61788
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