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Article

Clinical Applications of Gene Therapy for Primary Immunodeficiencies

1 Apr 2015

Abstract excerpt

Primary immunodeficiencies (PIDs) have represented a paradigmatic model for successes and pitfalls of hematopoietic stem cells gene therapy. First clinical trials performed with gamma retroviral vectors (γ-RV) for adenosine deaminase severe combined immunodeficiency (ADA-SCID), X-linked SCID (SCID-X1), and Wiskott-Aldrich syndrome (WAS) showed that gene therapy is a valid therapeutic option in patients lacking an...

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PMID: 25860576
DOI: 10.1089/hum.2015.047

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