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Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

30 Apr 2016

Abstract excerpt

Adenosine deaminase (ADA) deficiency is a rare, autosomal-recessive systemic metabolic disease characterized by severe combined immunodeficiency (SCID). The treatment of choice for ADA-deficient SCID (ADA-SCID) is hematopoietic stem cell transplant from an HLA-matched sibling donor, although <25% of patients have such a donor available. Enzyme replacement therapy (ERT) partially and temporarily relieves...

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PMID: 27129325
DOI: 10.1182/blood-2016-01-688226

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