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Gene Therapy for Primary Immunodeficiencies

11 Jun 2012

Abstract excerpt

For over 40 years, primary immunodeficiencies (PIDs) have featured prominently in the development and refinement of human allogeneic hematopoietic stem cell transplantation. More recently, ex vivo somatic gene therapy using autologous cells has provided remarkable evidence of clinical efficacy in patients without HLA-matched stem cell donors and in whom toxicity of allogeneic procedures is likely to be high....

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PMID: 22691036
DOI: 10.1089/hum.2012.116

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