Skip Navigation LinksHome > December 2011 - Volume 11 - Issue 6 > Gene therapy for the Wiskott–Aldrich syndrome
Current Opinion in Allergy & Clinical Immunology:
doi: 10.1097/ACI.0b013e32834c230c
Primary immune deficiency disease: Edited by Amos Etzioni and Ramsey Fuleihan

Gene therapy for the Wiskott–Aldrich syndrome

Galy, Anne; Thrasher, Adrian J.

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Background: Wiskott–Aldrich syndrome (WAS) is a rare X-linked primary immunodeficiency (PID) characterized by micro-thrombocytopenia, recurrent infections, eczema, which is associated with a high incidence of auto-immunity and lymphoreticular malignancy. One of the first diseases to be successfully treated by allogeneic hematopoietic stem cell transplantation, WAS is currently the subject of several phase I/II gene therapy trials for patients without HLA-compatible donors.

Purpose of review: This article reviews the preclinical and clinical data leading to the development of gene therapy of WAS with lentiviral vectors.

Recent findings: A recent clinical trial using a conventional gammaretroviral vector has demonstrated the proof of principle of gene therapy in WAS, but has also highlighted a common limitation of the technology. Encouraging preclinical efficacy and safety results using refined lentiviral vectors, and the development of robust clinical-grade manufacturing processes have supported the initiation of several phase I/II new studies.

Summary: WAS is amenable to hematopoietic stem cell gene therapy. New trials using lentiviral vectors are expected to improve efficacy and safety profiles. Beyond proof of principle, ongoing international efforts to coordinate trials of gene therapy for the WAS may also provide a model for the expedited development of new treatments for other rare diseases.

Copyright © 2011 Wolters Kluwer Health, Inc. All rights reserved.


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