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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Bibliografiske detaljer
Main Authors: Sauer, Aisha V., Morbach, Henner, Brigida, Immacolata, Ng, Yen-Shing, Aiuti, Alessandro, Meffre, Eric
Format: Artigo
Sprog:Inglês
Udgivet: American Society for Clinical Investigation 2012
Fag:
Research Article
Online adgang: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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