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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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Autors principals: Sauer, Aisha V., Morbach, Henner, Brigida, Immacolata, Ng, Yen-Shing, Aiuti, Alessandro, Meffre, Eric
Format: Artigo
Idioma:Inglês
Publicat: American Society for Clinical Investigation 2012
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Accés en línia: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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