April 21, 2015

An Emerging Era of Clinical Benefit From Gene Therapy

Author Affiliations
  • 1Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland
  • 2Department of Pediatrics, University of Washington and Seattle Children’s Research Institute, Seattle

Copyright 2015 American Medical Association. All Rights Reserved. Applicable FARS/DFARS Restrictions Apply to Government Use.

JAMA. 2015;313(15):1522-1523. doi:10.1001/jama.2015.2055

The report in this issue of JAMA by Hacein-Bey Abina and colleagues1 provides strong evidence that gene therapy using myeloid/lymphoid conditioning combined with subsequent infusion of lentivector-transduced autologous CD34+ hematopoietic stem cells (HSCs) achieves substantial restoration of immune function associated with prolonged clinical benefit to patients with severe phenotype Wiskott-Aldrich syndrome (WAS). Prior to treatment, 6 of the 7 patients included in the study had the highest WAS severity disease score of 5; all patients had experienced serious clinical events and complications that are the hallmark of classic WAS, including recurrent viral or bacterial infections, bleeding due to microthrombocytopenia, severe eczema, vasculitis, enteropathy, and other autoimmune problems.

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