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

24 May 2012

Abstract excerpt

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 autoantibody production, including anti-nuclear antibodies (ANAs), frequently occur in ADA-SCID patients after...

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PMID: 22622038
DOI: 10.1172/jci61788

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