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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...

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Détails bibliographiques
Auteurs principaux: Sauer, Aisha V., Morbach, Henner, Brigida, Immacolata, Ng, Yen-Shing, Aiuti, Alessandro, Meffre, Eric
Format: Artigo
Langue:Inglês
Publié: American Society for Clinical Investigation 2012
Sujets:
Accès en ligne: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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