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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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Detalhes bibliográficos
Main Authors: Sauer, Aisha V., Morbach, Henner, Brigida, Immacolata, Ng, Yen-Shing, Aiuti, Alessandro, Meffre, Eric
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2012
Assuntos:
Acesso em linha: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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